Research Australia grassROOTS winter 2015

ISSUE ELEVEN 02 A Message From The CEO 03 Blood Group Gene Discovery Solves Newborn Puzzle

04 Research Grant To Discover The Link

RESEARCH AUSTRALIA

AN ALLIANCE FOR DISCOVERIES IN HEALTH

Between Cardiovascular Disease And Neurodegeneration 05 Clinical Trials Lead To Malaria Breath Discovery 06 Preventing Preterm Birth: New Pharmacological Strategies 07 MS Researchers Help Kiss Goodbye To Multiple Sclerosis 08 $1.6m Helps Researchers Translate Evidence Into Action 10 Participation In Research Sheds New Light On Brain Health And Exercise 11 Looking Forward, Looking Back: 30 Years Of Passion And Results For Indigenous Health 12 Griffith Dental Trial Goes Back To Roots 14 First Australian Type 1 Diabetes Clinical Research Resource Map 15 Ingham Institute ‘Strength 2 Strength’ Injury Program For Carers 16 Mitochondrial Donation And Its Future In Australia 17 Rapid Assessment Of Cardiac Chest Pain Research 18 $8.9 Million Injection Boosts Bowel Cancer Research 19 Further $4 Million Investment In Quest To Cure Blood Cancer 20 Targeting Unhealthy Listening Habits 21 The Mobile Application Rating Scale (MARS): A New Tool For Assessing The Quality Of Health Apps 22 Philanthropy And Research – Aligning Expectations 23 HMRI Chosen As Test Site For Revolutionary Stroke Detector 24 Lipid Pathways In Multiple System Atrophy 25 International Collaboration Seeks To Make Childbirth Safer In Developing Countries 26 Rare Voices Australia Hosts A Rare Disease Summit 27 Bringing Medicine, Engineering, Science And Industry Together 28 Value Of Medical Technology: Saving Lives And Healthcare Costs 29 Compounds Australia Facilitating New Spinal Cord Research 30 A SURE Thing 31 Amalgamating 15 Medical Research Foundations Into One 32 Board of Directors 32 Editor’s Corner

WINTER 2015

Winter 2015

A Message From The CEO This week I had the privilege of speaking at a meeting of the Canberra branch of the MS Angels. MS Angels is a group of female business leaders and future leaders in current management positions, each of whom has made a personal commitment to supporting multiple sclerosis (MS) research in Australia.

Each group consists of about 20 dynamic female executives, some whom have a personal connection to MS. MS is a disease that commonly impacts women in the prime of their life and the MS Angels believe it is a cause that warrants support from well-connected women to both fund research and raise awareness amongst their colleagues, family and friends.

The MS Angels have contributed over $700,000 in seed finding towards cutting-edge MS research projects since they began in 2009.

The evening was designed for these remarkable women, including some with MS, to hear how the research they were seed funding was progressing and to discuss the importance of health and medical research in Australia. Dr Holly Cate, Head of the Myelin Repair Laboratory at Melbourne University, gave a really engaging presentation on the research her team is undertaking. Dr Cate is looking at the cell process for how the body does or does not repair the myelin sheath damaged by MS. The level of interest and quality of questioning by the MS Angels was as fascinating as the research itself.

One of the women I met was an up and coming lawyer who had been diagnosed with MS about four years ago. Her message to me was very powerful; fund raisers for various diseases need to consider the impact they have on the community’s perception of the people with the condition. She is concerned that people’s perception of her career potential could be adversely impacted if ‘doom and gloom’ was the theme of campaigns. Her work colleagues do not know she has the condition as she considers the knowledge may adversely impact how they treat her, the types of work she is given, and therefore her career prospects. She was very positive about the ‘Kiss MS Goodbye’ campaign, because it has a very positive message. That really got me thinking about the importance of having patient input not only to the research process, but also into our fundraising messages.

This event confirmed for me the value of the Research Australia Philanthropy for Health and Medical Research Conference, where we bring givers, researchers, fund-raisers and consumers together to share their ideas. We are currently in the process of creating a really interesting speaker program I hope to share we you soon. So please put a placeholder in your diary for 19 August, Double Bay Sydney, at the new Intercontinental Hotel. I look forward to seeing many of you there.

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Research Australia grassROOTS WINTER 2015

SAVE THE DATES Research Australia Events for 2015 •R esearch Australia Philanthropy for Health & Medical Research Conference Wed 19 August 2015 Intercontinental Double Bay, Sydney •R esearch Australia Awards Dinner Wed 18 November 2015 Park Hyatt, Melbourne see our website for more details www.researchaustralia.org

Winter 2015

Blood Group Gene Discovery Solves Newborn Puzzle Your blood type is more complicated than you think, with hundreds of possible variations apart from the commonly known ABO and Rhesus blood groups. Genetic techniques allow blood variants to be characterised with pinpoint accuracy, and researchers at the Australian Red Cross Blood Service have used them to solve the medical mystery of a Canadian baby who nearly died at birth.

In 1990, researchers at the Blood Service found a novel antigen on the red cells of a single, very dedicated blood donor. This donor was named Sarah, and her new antigen was named after her: ‘SARA’.

SARA didn’t fit into any of the known families of blood group antigens, and a vial of Sarah’s unusual blood was stored, frozen, in a blood type archive.

Around 20 years later, the Blood Service received blood samples from collaborators at the Canadian Blood Services which came from a family whose baby had needed a massive blood transfusion at birth. The Canadians thought that this case could be related to the rare SARA antigen and sent samples to Australia for confirmation.

The mother’s plasma was tested against a panel of over 50 archived rare cell types,

and reacted only with a sample of SARA red cells. Rhiannon McBean, a doctoral candidate in Research and Development at the Blood Service and the University of Sydney, explained the results: “The father was SARA-positive and the mother was SARA-negative. She had an antibody that reacted strongly with SARA-positive cells. The baby needed a double volume exchange transfusion to recover.”

With the problem identified as SARA incompatibility, a Blood Service research team, led by Associate Professor Catherine Hyland, set out to identify the gene responsible. Blood and DNA samples were taken from both the Canadian and Australian families, including members from several generations of each family. Rhiannon noted that the Australian family played a vital

Rhiannon McBean at work in Research and Development at the Australian Red Cross Blood Service

role in her research, saying, “This family has a lot of respect for the Blood Service they couldn’t do enough to help me”.

To identify the gene, researchers first identified the family members who carried the antigen by checking whose blood reacted with the antibody in the Canadian mother’s plasma.

The next step was to find a gene sequence shared only by those family members.

“We used whole exome sequencing, which reads the entire coding region of a person’s DNA. It’s a leading-edge technology that wasn’t widely available to the research world even five years ago,” said Rhiannon.

The analysis found nearly 500,000 single nucleotide variations in the Australian family, so finding the one variation responsible for the SARA antigen was an incredibly detailed and painstaking task.

“The technology is really powerful and yet generates so much data that you can feel like you’ve hit a brick wall,” said Assoc.

Professor Hyland. Despite the painstaking nature of the task, Rhiannon systematically refined the data: “Because SARA is so rare, we knew we were looking for something that hadn’t been discovered before (and) we also had to look for something which fit the inheritance pattern,” she explained.

When the final analysis was complete, they discovered that in SARA-positive individuals a single nucleotide had changed in the DNA sequence which encodes glycophorin A, one of the main proteins on the surface of the red blood cell. Rhiannon was ecstatic: “I couldn’t believe my eyes.

I didn’t want to believe my luck.”

The SARA antigen has now been officially recognised by the International Society for Blood Transfusion as a new blood group, and, for the young SARA-positive males in the families, this discovery means that they and their families will be able to successfully manage future pregnancies where the SARA antigen may be inherited.

As Assoc. Professor Hyland concluded, “One of the advantages of working at the Blood Service is that we are involved in these clinical cases that are important.

The family, including Sarah, were dedicated donors, and their generosity has led to this outcome which can help patients.” www.donateblood.com.au

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Winter 2015

Research Grant To Discover The Link Between Cardiovascular Disease And Neurodegeneration Earlier this year, the Alzheimer’s Australia Dementia Research Foundation (AADRF) awarded a postdoctoral ‘half-funded’ scholarship to Dr Scott Ayton from the Florey Institute for Neuroscience and Mental Health to further investigate the link between high blood pressure, cardiovascular disease and neurodegeneration.

Dr Ayton was one of twenty five researchers who shared in a total of $2.4 million, thanks to the efforts of community donors and fundraisers who helped fund the 2014 AADRF dementia research grants program.

With more than one million Australians touched by dementia in some way, it is imperative we continue to make research breakthroughs in the cause, care, prevention and potential treatments for dementia. If not, it is possible that more than three million of us could develop dementia over the next 36 years.

Dr Ayton hypothesises a strong link between heart health and brain health, and his research aims to investigate and clarify the cardiovascular aspects of Alzheimer’s disease, using a variety of innovative techniques such as tissue culture and animal and human analysis.

“Current research tells us that individuals who have elevated blood pressure during the middle portion of their lives (that is 50-65) have a greater risk of developing Alzheimer’s disease later in their life,” Dr Ayton said.

“While the reason for this is not fully understood, new preventative therapeutics might be possible if we discover what causes the association between mid-life elevation of blood pressure and risk for Alzheimer’s disease.”

Dr Ayton’s recent findings in animal studies have shown that a protein called tau, which is known to be involved in Alzheimer’s disease, rises with elevated blood pressure.

It is known that when tau proteins malfunction they form protein tangles inside the cell which lead to a breakdown in the brain cell’s ability to communicate with other brain cells and eventually to cell death. This has led to Dr Ayton’s belief that the tau protein is an important link between heart health and Alzheimer’s disease.

Scientia Professor Henry Brodaty, Chair of the Alzheimer’s Australia Dementia Research Foundation said these research grants and fellowships are supporting the next generation of Australian dementia researchers who will be among those

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making the breakthroughs in understanding and treatment of dementia in the future.

“Dr Ayton is just one example of many early career Australian dementia researchers who are committed to seeing the number of people affected by the disease decreasing in the future,” said Professor Brodaty.

The Alzheimer’s Australia Dementia Research Foundation is the research arm of Alzheimer’s Australia, and runs an annual competitive research funding program that provides scholarships, project grants and postdoctoral fellowships to students and early career dementia researchers.

Research Australia grassROOTS WINTER 2015

r Ayton plans to investigate the link D between cardiovascular disease and Alzheimer’s disease using a variety of innovative techniques.

The 2015 Alzheimer’s Australia Dementia Research Foundation grants program is currently open for applicants. The foundation welcomes donations and philanthropic support to achieve its objective of increasing Australia’s dementia research capacity. www.fightdementia.org.au

Winter 2015

Clinical Trials Lead To Malaria Breath Discovery When Professor James McCarthy began testing new malaria medicines, he never anticipated the clinical trials would result in the potential for a breath test to diagnose the deadly disease.

Professor McCarthy – who heads Tropical Medicine at QIMR Berghofer Medical Research Institute – has been giving healthy volunteers a controlled malaria infection in clinical testing of new antimalarial treatments.

His work came to the attention of the CSIRO and the Australian National University – and the resulting ‘side project’ could provide a key to global efforts to eradicate a disease which kills hundreds of thousands of people every year.

More than a dozen participants from Professor McCarthy’s clinical trials in Brisbane were asked to provide a breath sample for the collaborative study.

The scientists found distinctive chemicals present in the breath samples well before malaria parasites could be spotted in the subjects’ blood.

They found the levels of these chemicals increased in line with the severity of the infection and disappeared when the malaria was cured.

Professor McCarthy said if a simple breath test could be developed from this discovery it would save lives and make diagnosis less invasive.

“Malaria is still detected by looking for parasites in patients’ blood,” Professor McCarthy said.

“These chemicals are present in the breath four days before the parasites show up, meaning patients can be treated before they get really unwell.

“Quicker diagnosis would not only save lives but could also help reduce the spread of malaria because people can help transmit the infection before the symptoms become evident.”

Dr Stephen Trowell from the CSIRO said the team is now collaborating with researchers in Africa and parts of Asia – where malaria is endemic – to test whether the same chemicals can be found in the breath of patients there.

“We are also working with colleagues to develop very specific, sensitive and cheap ‘biosensors’ that could be used in the clinic and the field to breath test for malaria,” Dr Trowell said.

The World Health Organisation estimated 584 000 deaths were caused by malaria in 2013, from almost 200 million cases – many of them children in developing countries.

Professor McCarthy’s clinical trials are conducted in partnership with the Genevabased Medicines for Malaria Venture (MMV), which is committed to developing anti-malarial drugs which are affordable and appropriate for regions where the disease is endemic. www.qimrberghofer.edu.au

alaria kills more than half a million people each year, many of them children in developing M countries. Image courtesy of Medicines for Malaria Venture.

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Winter 2015

Preventing Preterm Birth: New Pharmacological Strategies Preterm birth is now officially the leading cause of death in children under the age of 5 years. In Australia our rates of preterm birth are around 7 – 8%, resulting in around 25,000 preterm deliveries annually at a cost to the healthcare system of around half a billion dollars a year.

A new initiative was launched in WA in 2014 with the aim of reducing the rate of preterm birth in the state over the next five years: The Western Australian Preterm Birth Prevention Initiative (www.thewholeninemonths.com.au).

Central to this initiative is a programme of on ongoing research into prediction, treatment & prevention of prematurity, combined with monitoring of preterm birth rates over the course of the initiative.

Babies born more than two months preterm have the greatest risks of adverse outcomes, and the majority of such deliveries arise as the result of intrauterine infection and inflammation. In these pregnancies, bacteria residing in the vagina ascend and breach the cervical barrier, colonise and invade the fetal membranes and amniotic fluid, and elicit an intraamniotic inflammatory reaction (manifested as histologic chorioamnionitis) that triggers preterm labour. This can also result in associated morbidities such as cerebral palsy. The bacteria most commonly isolated from the amniotic cavity in preterm deliveries are the Ureaplasma species, commensal organisms found in the reproductive tracts of around 50% of pregnant Australian women.

A related bacterium, Mycoplasma hominis, is also a relatively common isolate from infected amniotic cavities. Collectively, these ‘genital mycoplasmas’ are a major cause of early

preterm birth, although a wide variety of other bacteria can also trigger inflammation-driven preterm birth.

In order to develop effective therapies to treat and prevent infection-associated preterm birth we must overcome two main hurdles: 1) how to identify women at risk who would benefit from treatment, and 2) how to treat intraamniotic and fetal infection and inflammation effectively and safely. To address the first issue, we are currently undertaking a large NHMRCfunded study to employ microbiological, biochemical and metagenomic approaches to identify asymptomatic women who are at high risk of preterm birth arising from intrauterine infection.

With respect to the second hurdle, one might presume that antibiotic therapy would be a simple solution. Somewhat surprisingly, however, attempts to date to prevent preterm birth using antibiotics have been largely unsuccessful, due in part to deficiencies in the pharmacokinetics and pharmacodynamics of currently available antibiotics. We recently made a major discovery that could change the antimicrobial therapeutic landscape in pregnancy. Using our pregnant sheep model we discovered that a new fluoroketolide antibiotic called solithromycin is capable of crossing the placenta and reaching effective concentrations in

Pharmacological strategies for the treatment and prevention of preterm birth

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Research Australia grassROOTS WINTER 2015

the fetal circulation and amniotic fluid within 4 hours of a single maternal dose.

Solithromycin is the first macrolide-like antibiotic that is active against all the major microorganisms known to be associated with preterm birth and able to effectively treat intrauterine/fetal infections via oral maternal administration (other macrolides do not readily cross the placenta).

Importantly, it is highly effective against a wide variety of antibiotic-resistant bacteria and is particularly potent against all genital mycoplasma species and strains. Its high oral bioavailability (~70%), ease of administration (400 mg p.o. once per day), potency, ability to overcome antibiotic resistance and favourable tolerability/ side-effect profile make it a convenient and extremely attractive therapeutic option.

In this regard, solithromycin is markedly superior to widely used antibiotics such as azithromycin, erythromycin, clindamycin and metronidazole for this indication in pregnancy.

Not only is it highly effective, but solithromycin is also an effective antiinflammatory agent, significantly more potent than existing macrolides in terms of suppressing macrophage/monocyte activation. Studies are also ongoing on the use of more potent agents that block cytokine signalling in the amniotic cavity in response to microbial triggers. It is now widely accepted that the fetal inflammation which occurs as a consequence of intrauterine infection must be prevented in order to protect the fetus and maximise the benefits of antenatal/perinatal antimicrobial treatment. Cytokine-blocking agents can be given intra-amniotically to ensure that amniotic and fetal inflammation is inhibited without compromising the maternal immune system and risking persistence of infection.

Solithromycin is not currently available; it is expected to be licensed for use in 2016.

In the meantime, its pharmacokinetics and potential use in pregnancy will be investigated in a series of clinical trials planned for later this year. www.uwa.edu.au

Winter 2015

MS Researchers Help Kiss Goodbye To Multiple Sclerosis On 1 May, multiple sclerosis (MS) researchers around Australia replaced their white lab coats with red ones to show their support of the ‘Kiss Goodbye to MS’ campaign.

An initiative of MS Research Australia, Kiss Goodbye to MS is a national fundraising campaign, which aims to empower those with MS, their friends, family and colleagues to raise vital funds towards MS research.

Taking place throughout May each year, Kiss Goodbye to MS also aims to highlight the important role of talented MS researchers and the incredible advances seen as a result of their work.

This year, teams of researchers around Australia were invited to trade in their white lab coats for red ones, take a photo and post it on social media. Photos were then shared, retweeted and reposted by the MS community to great effect.

Associate Professor Michael Buckland, MS Research Australia Brain Bank CoDirector, and Head of Neuropathology at Royal Prince Alfred Hospital says, “Our team were more than happy to wear our red lab coats on the 1st May! It’s a great way to raise awareness about the exciting

MS research happening in Australia.”

Also embracing the campaign is Associate Professor Mark Slee, a neurologist based at Flinders University, who captains the ‘Miles for Myelin’ cycling team. Earlier this year, the team, made up of MS neurologists and researchers, their colleagues and friends successfully raised over $15,000 for MS research through their participation in the challenging Tour Down Under cycling event.

‘I am part of an amazing research community in Australia and New Zealand that is accelerating our progress towards a cure. Significant advances have happened in MS research in recent years, and a lot of it is due to Australian and New Zealand researchers. Therefore, it is my hope that in my lifetime I will be able to help kiss goodbye to MS,’ said Associate Professor Mark Slee.

With a combination of expert MS researchers, amazing fundraisers and financial donors, the outlook for people with MS is improving at a significant rate.

Just twenty years ago when a person was diagnosed with MS, the treatment options were extremely limited and there were no prevention strategies available for the disease. This meant that the prospect of needing to use a wheelchair or walking aid within just ten years of diagnosis was a very confronting reality for many MS patients.

In recent years, cutting-edge MS research has led to increasingly positive outcomes for those affected. The first injectable immunotherapy was released in 1993 and the first oral treatment was approved in 2011. In 2015, there are a total of 11 therapies available for the treatment of relapsing-remitting MS in Australia and many people with MS are able to achieve a good quality of life for much longer.

However, there are still people with MS who do not benefit from current treatments, particularly those with the more severe forms of the disease and those diagnosed with progressive forms of MS. It is therefore essential that the momentum in MS research continues until a cure for MS becomes a reality.

Established in 2012, the Kiss Goodbye to MS campaign has attracted a new generation of fundraisers to the world of MS, primarily young women – the demographic most commonly diagnosed with MS – to participate in the cause, and has raised an impressive $2.5 million to date. All funds raised are directed towards vital research into the cause, treatment and a cure for MS. The ultimate aim is that we will one day ‘Kiss Goodbye to MS’ once and for all. www.kissgoodbyetoms.org

r Scott Byrne and his research team D at the University of Sydney.

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Winter 2015

$1.6m Helps Researchers Translate Evidence Into Action Bupa Health Foundation announces Health Awards 2015 recipients.

Leading Australian researchers have received significant funding support with the announcement of the Bupa Health Foundation 2015 Health Awards.

The Bupa Health Foundation, which is celebrating its 10th anniversary this year, awarded approximately $1.6 million between five winning research projects to improve health and care outcomes for Australians.

This year’s successful research proposals look to: • Help reduce vision loss across the population with a technology-driven, user-friendly screening system which identifies people with eye disease; • Improve clinical-decision making and patient care by using specific patient information to identify ways to use angiograms more appropriately;

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• Reduce system cost and hospital stays for people who have had hip or knee joint replacement surgery; • Decrease the number of hospitalisations and amputations by better managing foot ulcers in people with diabetes living in remote areas of Australia with information technology (‘telemedicine’); • Predict response to memory-enhancing drugs for people with dementia using brain imaging techniques, delivering individualised care to improve outcomes for people with Alzheimer’s disease.

Bupa Health Foundation Executive Leader Annette Schmiede said high quality health research is a critical driver of innovation to improve the effectiveness of Australia’s health services.

Since its inception in 2005, the Bupa Health Foundation has contributed more than $26 million to fund more than 100 projects, with this year’s winners selected from a pool of 584 applications.

“This year’s Health Award winners are dynamic projects embedded in the healthcare system, allowing for rapid translation of evidence into action and impact on clinical practice and patient care,” Ms Schmiede said.

Research Australia grassROOTS WINTER 2015

“Australians have access to some of the best healthcare in the world and continued investment in research is essential to sustaining our health system and delivering better health outcomes.

“The Foundation is very proud to support each of these award winners, and we look forward to supporting each team to translate discovery into health benefits in the years to come.”

The grant recipients were recognised at the annual Bupa Health Foundation Health Awards dinner held in Melbourne on Wednesday 29 April.

The Bupa Health Foundation is one of the country’s leading private charitable organisations dedicated to improving the health and wellbeing of Australians across its five key focus areas: wellbeing, managing chronic disease, healthy ageing, empowering people about their health and promoting affordable healthcare.

2015 Bupa Health Foundation Health Award Winners Development of an automated webbased screening system for eye diseases University of Melbourne, Centre for Eye Research Australia Professor Mingguang He Half of the eye diseases such as glaucoma, retinal damage caused by diabetes, and agerelated macular degeneration are undetected in Australia. Retinal imaging is a powerful screening method for these diseases but currently interpreting the screening results mainly depends on clinicians. This project will develop a web-based screening system incorporating artificial intelligence and automated grading. The system can be used at optometry or diabetes clinics. It will help improve access, reduce cost and waiting times and potentially maximise the vision outcome of patients.

Clinical decision-making and the appropriate use of coronary artery angiography University of Melbourne Associate Professor Vijaya Sundararajan An angiogram is a common procedure recommended for diagnosing narrowing of the blood vessels which supply oxygen to the heart and is recommended in all patients with a heart attack but in only a select group of patients with stable heart disease. However, research suggests that angiograms are underused in heart attacks and potentially overused in stable disease. This study will investigate how angiograms could be used more appropriately using specific patient information (including age, gender, risk factors and medical history) to improve clinical decision-making and patient care.

The project, if successful, will provide insights into how to change clinical practice so that a projected 1000 lives in Australia each year could be saved in heart attack patients without increased resources or cost to the health system.

STEP – St Vincent’s Early Mobilisation Pathway for total joint arthroplasty University of Melbourne Dr Trisha Peel Researchers will conduct a clinical trial comparing two different care plans in people who have had hip or knee joint replacement surgery. The study will compare the length of time patients stay in hospital after surgery with an Early Mobilisation Plan compared with Standard Care. The results will help to improve health outcomes and the costeffectiveness of care for these patients.

Telemedicine: New horizon in managing diabetic food ulcers in remote/rural Australia James Cook University, Translational Research on Endocrinology and Diabetes [TREAD], College of Medicine and Dentistry, Townsville Associate Professor Usman Malabu Information technology (‘telemedicine’) will be used to help nurses manage foot ulcers in people with diabetes living in remote areas of Australia. Foot ulcers are a serious complication of diabetes, causing more than 80 per cent of lower limb amputations in people with diabetes. Yet, people in remote areas have limited access to wound care specialists. If this model is successful, and rolled out to rural facilities, it will provide greater access to specialist wound care, and reduce hospitalisations and amputations for people with diabetes.

Predicting response to cholinesterase inhibitors in people with dementia CSIRO – Digital Productivity Flagship, The Australian e-Health Research Centre Associate Professor Stephen Rose This study will investigate whether measuring brain function associated with memory in people with early dementia can predict their response to cholinesterase inhibitors, medicines that may enhance cognitive function and stabilise memory.

Imaging techniques (PET and MRI) will be used to measure the function of specific (cholinergic) nerves in the brain, to help doctors decide whether to prescribe the cholinesterase inhibitors. This individualised treatment could improve outcomes for people with Alzheimer’s disease. www.bupa.com.au/about-us/bupahealth-foundation/about

-R: Prof Christine Bennett AO, L John Conde AO, Annette Schmiede, A/Prof Stephen Rose, A/Prof Vijaya Sundrarajan, A/Prof Usman Malabu, Dr Trisha Peel, Dr Paul Bates.

Absent from photo: Prof Mingguang He.

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Participation In Research Winter 2015 Sheds New Light On Brain Health And Exercise Body health and brain health both need one vital thing – physical exercise. National guidelines recommend older people need 150 minutes of physical activity each week to enjoy the physical health benefits. people were performing sufficient regular physical activity.

The first project, known as the INDIGO study, is investigating whether setting personal exercise goals and receiving encouragement from a volunteer mentor could be effective in overcoming a sedentary lifestyle. The study is funded by the National Health and Medical Research Council.

Lautenschlager’s team is working with research participants in Melbourne and surrounding areas. Participants take part in a six-month, home-based physical activity program with the amount of exercise gradually increased to 150 minutes weekly.

“Changing sedentary behaviour is not easy and many people find it challenging to develop the confidence to get started,” she said.

Research shows that physical activity helps brain health such as supporting memory and other cognitive functions.

The National Ageing Research Institute is working on three different projects to investigate the role of physical activity in preventing the onset of Alzheimer’s as well as depression.

Central to the success of the programs is the involvement of older people.

Associate Professor Briony Dow, Director, Health Promotions said: “Our vision is to bring research to life to improve health outcomes for older people. Our research guides aged care practice as well as policy to ensure positive ageing for Australia’s older people.

“One of the best ways we can do our work is by involving older people in our research so that they learn about the power of research in answering important questions.

The benefit of participants is that, generally, their health improves along the way.”

At least 20 per cent of cases of Alzheimer’s disease, the most common form of dementia, could potentially be prevented if

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Initial findings suggest that getting involved in the program is paying dividends.

Participant Mrs Cooper is now several steps closer to getting a dog and has started to swim again thanks to being involved in an innovative research project run by the National Ageing Research Institute (NARI).

Before joining, 70-year-old Mrs Cooper was not particularly physically active, tended to drive more than she walked and, in her own words, ‘needed to get moving.’

She had been caring for her mother and had not been focusing on her own health.

She knew that she needed to change her ways and being involved in a program that was properly supervised helped her to do that.

Today, she is walking three times a week for up to 50 minutes a time and she belongs to a walking group.

The team is looking to recruit more participants, specifically people aged 60-85, living in Melbourne or the Greater Melbourne area, who do less than 60 minutes of leisure-time physical activity weekly and who have noticed changes to their memory, are invited to join the study.

The second study, IMPACCT, is a randomised controlled trial evaluating the effectiveness of a personalised physical activity intervention to improve mental health and minimise functional decline among

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carers and the people they are caring for.

The study is funded through the National Health and Medical Research Council.

Lautenschlager says the reality is that most care is provided by informal carers, many of whom are over 60 years.

“Often these carers are at high risk of depression and they also face barriers when it comes to participating in health promoting activities,” she says.

The study, based on the Otago program, a home-based training program is running over six months with assessments at six and twelve months to determine how the participant is going.

The program begins with a baseline assessment to measure depression, burden, and current physical activity.

Participants are then placed into one of three groups: a home exercise program, social control to look for the potential improvement in depression and social contact and usual care, where people will receive minimal contact.

The study is currently looking for volunteers within a two-hour drive of central Melbourne. Volunteers have to be over 55, living at home with somebody they care for who is aged over 60, so you might be eligible to participate. All assessments and interventions take place in the home and over the telephone so no travel is required.

Stand Up and Go is the most recent study which is recruiting for volunteers. A sitting time reduction and physical activity program, Stand Up and Go is about improving physical and brain health through exercise.

NARI researchers are looking for people who are aged between 50 and 85, have Type 2 diabetes and do less than 1 hour of exercise per week. The program is 12-weeks and is based at home.

Participants will have two assessments (memory, fitness, brain imaging and blood tests) during the study.

People interested in taking part in any of the studies should contact NARI at reception@nari.unimelb.edu.au www.nari.unimelb.edu.au

Winter 2015 Looking Forward, Looking Back: 30 Years Of Passion And Results For Indigenous Health Professor Alan Cass has great passion for the Menzies School of Health Research.

Research’s past and present: its incredible legacy of improving health outcomes for Aboriginal and Torres Strait Islander people for over 30 years, and its vital work in the field today.

Menzies began as a bold vision to understand and address the unique health problems faced by Aboriginal communities in the north of Australia. Today, it is an internationally recognised medical research institute with a proud history of scientific discovery and public health achievement.

Prof Cass, a former Menzies PhD student and today one of Australia’s leading kidney specialists, points to the anniversary year as providing a key national platform for the organisation to broadcast the impact of its research and to celebrate the contribution of Menzies researchers, working in partnership with Aboriginal communities.

Beginning as a bold new vision to establish a dedicated health research centre in the Northern Territory to deal with health problems unique to this region, Menzies began its research efforts in January 1985.

Early areas of research focus included heart disease, trachoma, hepatitis B, alcohol-related diseases, nutrition and infectious diseases.

Demonstrating the international standing of its research outputs, Menzies received a ‘5 out of 5’ Australian Government Ranking for Excellence in Research with its work being described as “well above world standard”.

In an environment where fewer than 1 in 6 applications for national competitive research funding are successful, Menzies was awarded federal funding for more than 1 in 3 of its submitted competitive grants and fellowships by Australia’s peak body for supporting health and medical research, the National Health and Medical Research Council (NHMRC).

A major success for 2014 was the announcement that both of its submissions for Centres of Research Excellence (CRE) were successful. The CREs in Indigenous ear and hearing health and improving Indigenous primary health care, were some of a select few announced nationally, and have added to Menzies’ stable of Indigenously focussed CRE’s which include cancer and lung health centres of excellence.

Despite clear and sustained health improvements for Aboriginal and Torres Strait Islander children, outcomes are

nowhere near those for non-Aboriginal children, nor are they where they should be.

“Improving health outcomes requires more organisations respectfully working together, partnering with and growing capacity within communities, and having leading researchers on the ground who are truly passionate to make a difference,” Prof Cass said.

“Throughout our history we have honed respectful, culturally sensitive ways of working. Our collective and considerable wisdom, acquired in communities across Australia, positions Menzies uniquely to lead other agencies in a redoubled effort to ‘close the gap’ between Indigenous and non-Indigenous health and wellbeing.’

Visit the 30th anniversary website to learn more about Menzies’ history, achievements and a wide range of celebratory events planned for the year. menzies.edu.au/30years

Jonas & Elias Bonson from Maningrida

Fast forward to 2015, some 30 years later, Menzies research continues to grow and diversify.

“At Menzies, our research teams seek to work hand-in-hand with communities to ensure our research and education agenda addresses fundamental health and developmental priorities. This represents a foundation pillar of our work,” Prof Cass explains.

“These same teams are making discoveries that will shape the future of Indigenous and tropical health, which reach beyond the laboratory and will benefit all Australians and many others in our region.

“Complementing our laboratory-based biomedical research, is our work to boost the capacity of health service providers, community based researchers and health services – to deliver better care based on evidence about what works, and what doesn’t.”

Menzies’ current full-time workforce comprises of more than 200 researchers, many of which are award-winning researchers from around Australia and the Asia-Pacific region.

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Winter 2015

Griffith Dental Trial Goes Back To Roots For decades dentists have been advocating prevention in the fight against tooth decay and other oral diseases, but these messages require regular reinforcement. This is something that remote (mostly Aboriginal) communities miss out on due to over-stretched primary services.

A recent National Health and Medical Research Council (NHMRC) funded project is examining whether large scale, intervention-based dental care could provide lasting results to remote community members who struggle to retain consistent professional services or maintain the healthy lifestyles required for adequate prevention.

Professor Newell Johnson

Led by Professor Newell Johnson at Menzies Health Institute Queensland, the team will spend three months in the Cape York Aboriginal community of Bamaga delivering an intensive “four-prong” treatment and education (intervention) program they hope will stand the communities’ children in better shape than treating tooth decay after it has happened.

“The theory we’re running with is that if you explain everything we know about tooth decay and apply established preventative treatments, people will benefit for years and this will be cheaper than finding a dentist or therapist to live in a remote community. Even when dental clinics in places like Bamaga are staffed, the dental professional spends most of his/her time treating people with pain or broken dentures and there is insufficient time to apply prevention to the whole community.

Children are especially in need of intensive prevention,” said Professor Johnson.

“So in a one-on-one treatment we deal with any existing disease first and then apply prevention; that means disinfecting the mouth, applying topical fluoride and then a fissure sealant so the patient is made dentally perfect. Then we train them in the best way to take care of their teeth and maintain a healthy lifestyle.”

Professor Johnson has been treating, researching and writing about tooth decay for over 40 years, including a 20 year stint at the Royal College of Surgeons of England, London and remains one of Australia’s top oral health researchers.

While tooth decay can be painful and lead to more serious infections, Professor Johnson is also worried about how the rate of decay is indicative of a poor diet and greater health problems in remote communities.

“I have to say, after all these years I’m very surprised to find myself testing this

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hypothesis. I’m not an interventionist at all: I believe in basic, primary prevention; tooth decay is a disease caused by sugar so it goes against my grain, but we know that getting the population to severely restrict sugars and to clean their teeth well and regularly is impossible”.

“We have to concede that all the work we’ve done in remote Australian communities hasn’t achieved much, we are really struggling to get the people in these communities to adopt healthier lifestyles, so we’re essentially going for a big bang interventionist approach,” said Professor Johnson.

The approach adopted by Professor Johnson’s team is modelled on the World Health Organisation’s anti-smoking guidelines, which found a single one-onone direction to cease smoking from a doctor had the most dramatic and costeffective impact on individuals.

Research Australia grassROOTS WINTER 2015

Professor Johnson and his team are hoping that combined with a thorough treatment of existing decay in those with cavities [about 60% of the children based on their earlier survey], then intensive preventative measures with all children may have greater, and more cost-effective, longterm preventative impact on community members who take up their appointments.

“We’ll then go back to the community in two or three years and measure what we’ve found. But it’s going to take a pretty big change in the diets of children, as well as taking up better brushing habits to have complete success.”

Professor Johnson’s study is in the detailed planning stage and begins on the ground in winter [the dry season in Cape York] 2015. www.griffith.edu.au/health/menzieshealth-institute-queensland

Winter 2015

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Winter 2015

First Australian Type 1 Diabetes Clinical Research Resource Map The Australian-first Type 1 Diabetes Clinical Research Resource Map, published by JDRF Australia, is a snapshot analysis of the type 1 diabetes clinical research landscape in Australia. Incorporating data collected in interviews with leading Australian clinical researchers, the Resource Map enhances future researcher access to available clinical research resources by increasing their visibility and providing a directory of existing databases, biobanks and networks.

The Resource Map has identified three areas of need in the research development pipeline that if addressed, could accelerate, expand, and increase the potential of Australian type 1 diabetes clinical research.

• Translational research which is currently underfunded, meaning that many potential therapies do not make it from the laboratory to human clinical trials • Clinical trials which can be high-cost at both early-phase and late-phase trials, and therefore can make it difficult to attract commercial investment • Clinical research accelerators and enablers such as infrastructure, databases and bio-resources are currently fragmented and not always accessible to the wider scientific community Through the Resource Map, JDRF Australia has highlighted a number of opportunities to address these areas of need and accelerate the progress of Australian type 1 diabetes clinical research, further increase its global impact, and increase patient access to new therapies and treatments with targeted funding, programs, and connections.

Including: Invest in new ideas and new researchers • Fund targeted grants for transformative projects that have the potential for high benefit.

• Invest in career-support for emerging clinician researchers.

Accelerate research translation • Establish targeted funding schemes for bridging the ‘valley of death’ between innovative treatments developed in the laboratory and human clinical trials.

• Increase infrastructure that facilitates connection between basic and clinical researchers.

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Type 1 diabetes is an autoimmune disease with a complex origin that is caused by destruction of the insulin-producing islet cells in the pancreas, and at present there is no cure. It results in lifelong dependence on injected insulin and has a strong association with serious long-term health complications. The disease imposes a considerable medical, financial and emotional burden on individuals and families in Australia, as well as incurring substantial costs to the Australian health system.

Establish targeted clinical research funding schemes • Increase duration of clinical research grants.

• Increase the maximum amount of funding available for clinical research projects.

Connect Australian clinical research resources and infrastructure • Develop a national database of type 1 diabetes patients willing to participate in clinical research.

• Develop a ‘living biobank’.

Build targeted partnerships and collaborations • Invest in developing global collaborations.

• Build partnerships with industry to aid in commercialisation.

The publication of the Resource Map also underscores the necessity of coordinating our research priorities and consolidation of our resources. The Resource Map identified the need for a concerted, national effort to encourage Australian researchers to share and

Research Australia grassROOTS WINTER 2015

access the resources that are available in Australia, including databases, biobanks, and patient pools.

To access a copy of the Resource Map and to learn more about Australian clinical trials in type 1 diabetes, please visit www.t1dcrn.org.au.

The research and publication of the Resource Map was supported by the Macquarie Group Foundation.

Ingham Institute ‘Strength 2 Strength’ Injury Program For Carers

Winter 2015

To minimise the impact of injuries on carers of family members with traumatic brain injuries, Ingham Institute researchers have launched the ‘Strength 2 Strength’ care program introducing better coping mechanisms and overall improvement in quality of life for patients and carers across NSW. For many years brain injury research has predominately focused on the physical impact of injury on patients with little research devoted to evidence of its impact on carers and families, prompting Ingham Institute Brain Injury Rehabilitation Group Leader Associate Professor Grahame Simpson to lead ‘Strength 2 Strength’. This is a unique trauma program conducted across various hospital sites around Australia designed to help build resilience amongst families and carers of patients with brain and spinal injury which he first presented to the community at the Ingham Institute Luncheon on Friday 6 March.

“Prior research relating to chronic illness and child disability fields suggested that building resilience is a critical factor that modulates the quality of family adjustment,” explained Association Professor Simpson.

“However, there was no research investigating resilience amongst families and carers with Spinal Cord Injury (SCI) or Traumatic Brain Injury (TBI) – instigating the need to investigate the link between resilience and these particular areas of injury.”

Rachel and Emily James

The Ingham Institute Strength 2 Strength program is a world-first psychoeducational group program that aims to support families and carers to cope with caring for patients with TBI or SCI. It gives participants guidance on coping emotionally with the impact of injury, becoming more proactive in their communication with health professionals and identifying the strengths that can act as coping mechanisms.

“The immediate reaction after injury is to focus on the patient’s physical and psychosocial health, which often leads to the feelings of chronic hopelessness and sometimes suicide – however these are things which often impact the carer as well.

In fact, the impact on families and carers often bears more of a brunt emotionally and psychologically in terms of their ability to process the impact of the injury and what it means for them long-term,” explained Association Professor Simpson.

“This can include things like making modifications to homes and registering the fact that they are responsible for providing full-time care for their son, daughter, mother or relative for the rest of their lives – a daunting and traumatising revelation for many people.”

The Strength 2 Strength program was evaluated in 2012 via a multi-centre controlled clinical trial involving 49 family members that were recruited from seven rehabilitation centres across NSW. Due to the success of the trial, it was rolled out throughout NSW and is now a standard program conducted at Liverpool Hospital, with South Australia and Victoria to follow.

One family that is living proof of the benefits that a program like Strength 2 Strength are Rachel and Emily James from Mosman in Sydney. Mother (Rachel), a registered nurse, suddenly became her 22 year old daughter Emily’s full-time carer after Emily had a skiing accident which rendered her quadriplegic. Rachel became a participant of the Strength 2 Strength program at Royal Rehab in Ryde to help improve her ability to cope with her new role as a fulltime carer.

“No-one is prepared for spinal cord injury and coping isn’t innate,” explained Rachel James.

“Suddenly I was an everyday carer within the family home. I draw from my professional background to meet some of the practical demands of care giving in a domestic household: caring is only sustainable with resources, knowledge, experience and health,” she continued.

“I thought I was managing my daughter, Emily’s trauma but Strength 2 Strength taught me that coping and caring can be enhanced by peer group sharing and it initiated a friendship support group which became vital when we left rehabilitation and had to live independently in a challenging new world,” said Rachel.

Despite the extent of Emily’s injuries she has continued to thrive both socially and academically and is now completing a Bachelor of Psychology at the University of Sydney in NSW. And as a result of completing the Strength 2 Strength program, Rachel now hosts a blog www.everydaycaring.wordpress.com committed to online communication focused on sharing caring, coping and addressing disability. www.inghaminstitute.org.au

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Winter 2015

Mitochondrial Donation And Its Future In Australia On 24 February 2015 the United Kingdom House of Lords passed legislation to allow for the use of mitochondrial donation in the IVF process for women at risk of having a child with mitochondrial DNA (mtDNA) disease.

This landmark decision follows eight years of research and lobbying plus three separate expert reviews, and was passed by a majority of 232 votes. The UK Human Fertilisation and Embryology Authority is now charged with issuing a licence for these techniques to be used therapeutically on a case by case basis.

The two new mitochondrial donation techniques being developed for IVF are maternal spindle transfer and pronuclear transfer. They involve transferring nuclear genetic material from the mother’s egg into a donor egg that has had its nuclear DNA removed and retains only its healthy mtDNA; the resulting child therefore does not inherit the mitochondrial disease.

Children born from a mitochondrial donor pregnancy will have DNA from three people. However, it is important to note that the donated mtDNA replaces only 37 mtDNA genes (contributing about 0.1 per cent of a person’s genetic make-up), compared with approximately 20,000 genes in the nucleus. The mtDNA contribution is important for converting food into energy but appears to make no significant contribution to appearance, behaviour or other features, which are determined by the nuclear genes and environment.

Some groups have made sensationalist claims about the techniques being inappropriate because the children could

Nuclear DNA transferred into egg with healthy mitochondria

Donor’s egg containing healthy mitochondria Healthy embryo with no mitochondrial disease

Donor’s nuclear DNA removed

Mitochondrial Donation Maternal spindle transfer uses unfertilised eggs (oocytes), while pronuclear transfer uses eggs already fertilised by the father (one-cell embryos or zygotes). Unlike other IVF methods, both techniques could allow any woman carrying mitochondrial DNA disease to have healthy children who are genetically related to both parents.

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This is a simplified diagram outlining two methods for mitochondrial donation – maternal spindle transfer (prior to fertilisation) and pronuclear transfer (after fertilisation).

be said to have three parents. However, this is “misleading, inappropriate and unhelpful” according to the Nuffield Council on Bioethics in London, which in 2012 reviewed the procedures from an ethical standpoint.

Australian legislation currently prohibits research involving human embryos containing genetic material (including

Research Australia grassROOTS WINTER 2015

A recent publication in the New England Journal of Medicine – Mitochondrial Donation: How Many Women Could Benefit – estimates that “the average number of births per year among women at risk for transmitting mtDNA disease is 152 in the United Kingdom”, which would correspond to approximately 56 births per year in Australia.

The Australian Mitochondrial Disease Foundation (AMDF) strives to enact change in the Australian legislation concerning research on human embryos and to make mitochondrial donation available to Australian patients. Without such change, Australian families will be left with the choice of passing on potentially deadly genetic mutations, or foregoing bearing their own genetically related children.

Nuclear DNA removed from egg with unhealthy mitochondria Egg containing unhealthy mitochondria

mtDNA) from more than two persons. In 2010, an independent committee was appointed to review the Prohibition of Human Cloning for Reproduction Act 2002 and the Research Involving Human Embryos Act 2002. The report, released on 7 July 2011, recommended the legislation remain unchanged.

The AMDF has recently set up the Mitochondrial Donation Working Group, comprised of AMDF Board Members and members of its Scientific and Medical Advisory Panel. This group will take on the role of coordinating education for both politicians and the public on mitochondrial donation and its implications. From here, the working group will set out to lobby the government for the relevant changes to legislation that need to be made in order to bring these techniques to Australian women. So far, the working group has already held a useful meeting with politicians and ethicists, and has begun engaging more closely with Australian embryology and IVF researchers.

The AMDF will continue to pursue what it believes to be a promising step for women at risk of passing mitochondrial disease on to their children. While this process took eight years to be passed in the UK, the AMDF is hopeful it will not be nearly as long a wait for Australian patients. www.amdf.org.au

Winter 2015

Rapid Assessment Of Cardiac Chest Pain Research About one in every ten patients who present to an Emergency Department (ED) have symptoms that could be attributable to heart disease, making up an estimated 500,000 ED visits across Australia each year.

A significant challenge is safely and efficiently identifying the 80% or more who do not have heart disease. The current National guidelines recommend a lengthy assessment process for nearly all of these patients. Establishing a diagnosis of heart-related conditions is challenging and resource-intensive, with the process often taking between six and 24 hours.

Queensland Emergency Medicine Research Foundation (QEMRF) has funded a program of cardiac chest pain research led by Associate Professor Cullen, Senior Staff Specialist at the Department of Emergency Medicine, Royal Brisbane and Women’s Hospital.

to present the research outcomes at national and international conferences and meetings. She is now aiming to make Queensland a world leader in testing new cardiac biomarkers, novel strategies for risk assessment in the ED and translational research.

QEMRF’s investment in this program of research demonstrates a significant return on investment that has allowed emergency medicine professionals to respond effectively to significant challenges faced in the Emergency Department.

As the busiest frontline healthcare field, emergency medicine is also the most

under-funded medical research specialty in Australia. QEMRF support has enabled efforts for emergency medicine clinicians to conduct high quality, robust clinical research that may directly translate to improved patient care. Emergency medicine requires continued and expanded support of evidence-based and high-quality research to enable health system improvements and to address the challenges of growing demands on Australia’s health system. www.qemrf.org.au Associate Professor Louise Cullen

This program of research aims to identify those patients at low risk of heart disease within 2 hours after arrival in the ED. The rapid assessment process uses clinical information, electrocardiographs (ECGs) and blood tests.

This method of earlier diagnosis: • allows serious heart disease to be ruled out quickly in a large proportion of cases • moves patients out of emergency departments and acute care beds more quickly • reduces unnecessary hospital admissions by up to 20% thereby decreasing hospital crowding • contributes to significant potential financial savings across Queensland Health • is more satisfying for patients to be either discharged or admitted to hospital more quickly.

Associate Professor Cullen’s exceptional research has received financial support from QEMRF since the organisation’s first grant round in 2008, allowing her team to expand its trials and test new theories.

QEMRF also awarded her the Noel Stevenson Research Fellowship in 2012 in support of her attaining a PhD. Research findings have been published in the Lancet and Journal of the American College of Cardiology. Associate Professor Cullen is now recognised as an international leader in chest pain research and is frequently invited

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Winter 2015

$8.9 Million Injection Boosts Bowel Cancer Research Researching a cure for Australia’s second biggest cancer killer is to receive a major boost with $8.9 million to establish a professorial chair in bowel cancer research at the University of Sydney.

The majority of funding comes from national charity Bowel Cancer Australia which has committed $5.9 million to establish the position and will be supported by additional funds of $3 million available to the University to advance research into bowel cancer.

Latest figures from Cancer Australia reveal that 2009-2011 funding for bowel cancer specific research was $47.8 million, making the $8.9 million a significant injection of funding into the research mix.

Funded largely by the charity’s community fundraisers, the announcement comes as government funding for medical research is under review and the NHMRC chief has highlighted the limited number of grants available to medical researchers.

Bowel Cancer Australia chief executive Mr Julien Wiggins said the size of the funding commitment will enable the chair and ancillary support to continue in perpetuity.

“It’s good news in tough times. Our funding will expand Australia’s research capacity and we hope the Chair will be a game changer for bowel cancer research in this country,” he added.

Bowel cancer is the second most commonly diagnosed cancer and the second leading cause of cancer deaths in Australia.

“Changing that fact requires significant, long-term funding for dedicated bowel cancer research and that’s what we’ve provided.”

“Screening can help with early detection but it won’t eliminate bowel cancer.

Research is the only way to discover a cure,” said Mr Wiggins.

The Chair, to be named the Lawrence Penn Chair in Bowel Cancer Research after one of Australia’s oldest bowel cancer survivors, will be based at the University of Sydney’s Northern Clinical School Campus.

“We are extraordinarily grateful to Bowel Cancer Australia for their support,” said the

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awrence Penn (whom the position will be named after) and his son Richard Penn L (a patron of Bowel Cancer Australia).

Dean of Sydney Medical School, Professor Bruce Robinson.

“In times when national research funding is increasingly difficult to secure, we rely more than ever on funds from community groups and individuals to undertake the research which is essential if we are to better prevent and treat bowel cancer.”

“The University of Sydney has many world class cancer researchers and clinicians.

This philanthropic support for bowel cancer means we can build on our existing programs and make a greater contribution to improving cancer outcomes,” said Professor Robinson.

Mr Wiggins said that establishing the Chair will start with an international search in the first half of this year to attract a world-class research leader.

Bowel Cancer Australia chairman Brian McFadyen said that the charity’s agreement with the University of Sydney was the culmination of many years of effort by the Bowel Cancer Australia board.

To support Bowel Cancer Australia’s ongoing research effort, please call 1800 555 494 or visit www.bowelcanceraustralia.org

Research Australia grassROOTS WINTER 2015

Bowel cancer – fast facts • Bowel cancer, also known as colorectal cancer, is a cancer in the lining of the colon or rectum.

•M ost bowel cancers develop from pre-cancerous growths called polyps.

• 1 in 12 Australians will develop bowel cancer in their lifetime.

• About 15,000 Australians are diagnosed with bowel cancer each year and some 1,000 are under the age of 50.

• Around 77 Australians die each week from bowel cancer • Symptoms include any persistent change in bowel habits, blood in the bowel movement, bloating and cramping, unexplained weight loss, and severe abdominal pain.

• If detected early, around 90 per cent of bowel cancer cases can be successfully treated.

• Major risk factors include a personal or family history of the disease; being 50+ years old; a history of polyps; ulcerative colitis or Crohn’s disease.

Winter 2015

Further $4 Million Investment In Quest To Cure Blood Cancer

-R: Jo Denley, Director of Corporate Services & Risk Management at Bridgestone Australia, Dr Colm Keane, University of Queensland Diamantina L Institute (Recipient of Bridgestone Career Establishment Grant), Dr Anna Williamson, Leukaemia Foundation Head of Research & Advocacy, Professor Maher Gandhi, Leukaemia Foundation of Qld Chair in Blood Cancer Research at University of Queensland Diamantina Institute, Andrew Moffatt, Managing Director, Bridgestone Australia.

Blood cancer – the third biggest cause of cancer death in Australia – can develop in anyone, of any age, at any time.

In 2011, 3978 Australians died of blood cancer which claims more lives each year than well-known cancers such as prostate cancer (3294 deaths), breast cancer (2864 deaths) and melanoma (1452 deaths). In the next 12 months, an estimated 12,000 Australians will be newly diagnosed with leukaemia, lymphoma, myeloma, MDS, MPN or a related blood disorder, and an estimated 60,000 Australians are currently living with a blood cancer diagnosis.

In its quest to cure blood cancer, Australia’s peak body for blood cancer, the Leukaemia Foundation, invests in leading research to improve treatments and find cures, thereby reducing the impact of these diseases on Australians.

Since 2005, the Foundation has invested $38 million of donated funds in blood cancer research through its National Research Program, which currently has 49 research projects underway at leading research institutions across Australia.

Leukaemia Foundation Head of Research & Advocacy, Dr Anna Williamson, said three key projects, which the Leukaemia Foundation had previously seed funded, announced significant achievements in 2014. These new therapies are: • ABT-199 (for acute myeloid leukaemia [AML], myeloma, non-Hodgkin lymphoma and chronic lymphocytic leukaemia [CLL]); • CX5461 (for AML and myeloma); and

• CSL362 (for AML).

“We had provided vital seed funding for the basic biology that saw these important new therapies emerge, and we anticipate similar results to eventuate from our 2015 round of new research awards,” Dr Williamson said.

This year, 14 new awards, totalling almost $4 million were granted to researchers, students, a national co-operative trial group and institutions in Melbourne, Sydney, Brisbane and Adelaide. They comprise three post doctoral fellowships, two clinical PhD scholarships, five PhD scholarships, a career establishment grant, a supportive care grant, a clinical trial (for CLL), and a $550,000 contribution to the Australasian Leukaemia and Lymphoma Group Discovery Centre (formerly the ALLG Tissue Bank).

“What I find particularly exciting this year is the Foundation’s provision of funding across the whole blood cancer spectrum – lymphoma, myeloma and the chronic and acute leukaemias,” Dr Williamson explained.

“There also are studies in some of the most important and influential areas of research – epigenetics (the non-DNA component of the cell), CAR T-cells and immunotherapeutic mechanisms.

“All our latest grants are in fields that are the hottest areas of research in haematology at the moment. This means our researchers and students are working

at the frontiers of medicine. And this puts us right on the mark with what’s happening internationally, as highlighted in presentations at the world’s largest annual conference of the American Society of Hematology last December.

“The Foundation is achieving its aim to invest in the best research projects by Australia’s best researchers who have the best ideas, and this reflects our ongoing Vision to Cure,” said Dr Williamson.

She acknowledged the extraordinary support the Foundation’s research program receives from the community and major sponsors.

“The career establishment grant is sponsored by Bridgestone Australia Ltd, a clinical PhD scholarship is sponsored by the Pinter Trust and four awards are supported by individuals who each raised more than $10,000 in last year’s Light the Night event,” she said. www.leukaemia.org.au

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Winter 2015

Targeting Unhealthy Listening Habits Late last year, the HEARing Cooperative Research Centre (HEARing CRC) launched HEARsmart™ a new initiative to promote smarter hearing habits, aiming to reduce preventable noise-induced hearing loss associated with loud leisure noise.

According to the World Health Organisation (WHO), hearing loss now affects around 360 million people worldwide, making it the most common global sensory disability.

WHO’s recent international campaign “Make Listening Safe” stresses that many individuals could avoid hearing loss by adopting healthier hearing habits.

HEARsmart is a broad initiative that targets research and promotional campaigns towards specific groups engaged in highrisk behaviours, as well as venues and legislative authorities involved in creating or regulating environments where excessive leisure noise occurs.

Drawing on HEARing CRC research, HEARsmart is targeting specific groups identified at risk of acquiring a noiseinduced hearing loss and/or tinnitus in the longer term. HEARing CRC Chief Executive Officer, Prof Robert Cowan said “young people frequently develop a keen interest in music – this can increase their risk of acquiring a hearing loss through prolonged exposure to amplified sound, while playing instruments or listening to music at home, at gigs or at nightclubs, whether for work or for pleasure.

“Our aim is to develop practical solutions to help individuals and organisations alike to be aware of the risks and to protect their hearing, or the hearing of others, and make listening safe as well as enjoyable for all,” Prof Cowan explained.

In 2014, Know Your Noise (knowyournoise.nal.gov.au) was the first HEARsmart promotional campaign.

It was driven primarily by social media and through the distribution of postcards Australia-wide (via Avant Card); it promoted the Know Your Noise website, and its two main features: • a hearing test to help visitors determine their ability to hear speech in noise – this does not replace the need for clinical hearing assessments, but can recommend clinical follow up • an online noise risk calculator that enables users to discover their personal risk of developing hearing loss as a consequence of current listening habits, such as going to nightclubs, concerts or using personal music devices (taking into account duration as well as intensity of sound); it also offers simple suggestions on how to optimally manage these risks.

Dr Elizabeth Beach, a principal researcher from the National Acoustic Laboratories and one of the Project Leaders for HEARsmart noted that “one of the positive outcomes from the Know Your Noise Campaign was our ability to successfully engage our target audience by using different but complementary approaches. Getting our messages out through traditional media, social media and websites that young people hold in high regard, can create an ongoing awareness that we hope, given time, will result in a change in attitude and behaviour around loud sound.”

The HEARing CRC recently announced a second HEARsmart campaign, co-funded by the Deafness Foundation of Victoria – a pilot study that will help determine how live music venues in Australia can become more ‘hearing friendly’. The study will work with a small number of live music venues to develop practical and cost-effective solutions to help reduce noise exposure for musicians, patrons and venue staff while maintaining or improving the fidelity of the music being heard.

“In the coming months, HEARing CRC researchers, will carefully measure noise levels at gigs and gain input from patrons and staff on their experience of sound and their exposure,” Meghan Stewart HEARsmart Venue Pilot Manager explained. “Using this information and in consultation with venue owners, we will develop a practical noise-reduction package. While the package will be venuespecific, we hope it will be adaptable so that, in time, it can be implemented in other similar music venues across Australia.”

Through HEARsmart, the HEARing CRC is working closely with other organisations active in the hearing and music industry arena, including: Australian Hearing, the National Acoustic Laboratories, Australian Deafness Foundation, The Deafness Forum Australia, Vic Deaf and Music Victoria.

More information about HEARsmart™ www.hearsmart.org EARsmart: Know Your Noise campaign H advertising postcard developed and distributed via Avant Card.

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Research Australia grassROOTS WINTER 2015

Winter 2015 The Mobile Application Rating Scale (MARS): A New Tool For Assessing The Quality Of Health Apps More than 1 million mobile applications (apps) are available to smartphone users, with the ‘health & fitness’ and ‘medical’ app categories in the Apple app store ranked at 11 and 18 in popularity respectively. But with the rapid proliferation of smartphone apps, and little to no research to indicate their effectiveness, how can we as users, health professionals, and researchers trust the quality of information and guidance they are providing?

The Young and Well Cooperative Research Centre (CRC), in partnership with Queensland University of Technology (QUT), has developed the first multidimensional tool for assessing the quality of mobile health apps. The Mobile Application Rating Scale (MARS) was developed by a team of psychologists, web/app developers and designers at QUT, and tested on 60 randomly selected wellbeing apps.

Results indicated the MARS provides a comprehensive, reliable and objective tool for app developers, researchers and health professionals to rate apps against four quality scales: engagement, functionality, aesthetics, and information quality. It also provides a measure of the perceived level of user satisfaction and utility of the app.

VicHealth has already acknowledged the MARS as one the most outstanding projects and campaigns involved in improving the health and wellbeing of Victorians, winning a 2014 Victorian Health Promotion Foundation Award.

The subjective and un-regulated nature of star-ratings in app stores means that they can be unreliable, and previous attempts to create mobile app evaluations have been too technical or specific. The MARS is an easy-to-use (with appropriate training), simple, objective, reliable and widely applicable tool for measuring app quality. A simpler ‘app user’ version of the MARS has also been developed to allow researchers and app-developers to conduct user-acceptability studies.

This version of the MARS uses simpler language and does not require health expertise or training.

The MARS will allow people at all levels of app-interaction to confidently create and

use reliable apps, to ensure app users receive the best help and support possible.

“The Mobile Application Rating Scale is an excellent addition to the new body of evidence and practical resources we are creating at the Young and Well CRC, which explores how new and emerging technologies can improve the mental heath and wellbeing of young people,” said Young and Well CRC CEO, Associate Professor Jane Burns.

“The MARS will be a comfort to parents and professionals in making informed choices about the use of apps.”

Associate Professor Leanne Hides, who led the project, said, “The MARS is a game-changer for our modern, app-centric lives. Researchers and app developers will benefit from having a reliable tool to help them create high quality apps. It will also increase the confidence of young people and their parents’ of the reliability and accuracy of the apps they are using.”

The free Mobile Application Rating Scale online training modules are available on the Young and Well CRC YouTube page – www.youtube.com/yawcrc

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Winter 2015

Philanthropy And Research – Aligning Expectations Knowledge Translation Australia has identified the need for researchers to develop specific skills to successfully obtain funding from alternative sources.

Health and medical research funding is getting increasingly competitive. The 2014 funding round from the National Health and Medical Research Council saw overall success rates at just 15%. This low rate, and the increasing number of graduating researchers, has resulted in a push toward alternative funding opportunities from both government and private sectors. One such funding opportunity is that of philanthropy.

Philanthropic funders and charitable foundations are motivated by outcomes.

They want to see benefits delivered to society, particular groups or afflictions and the overall health system in general.

It is a much more personalised funding source with investors often attached to a particular cause in some way, which creates a desire for tangible research outcomes and measurable impact. The successful realisation and demonstration of research impact requires a good understanding and solid implementation of knowledge translation.

Knowledge translation (KT) in health and medical research is often narrowly viewed as commercialisation, such as advances in drug discovery and medical technologies.

But, KT is a system of processes aimed at closing the research to practice gap, be it through commercialisation, policy development or changes to service delivery.

KT requires tailored communication, multi-disciplinary collaboration and implementation of research knowledge.

Furthermore, it is based on creating tangible outcomes and research impact, both of which align with the motivations of philanthropic funders. By tapping into these motivational triggers, the opportunity exists for researchers to capitalise on the available funding that these organisations have to offer. Due to the high numbers of grant seekers, philanthropic funding is still competitive, but perhaps the bigger challenge for researchers is in understanding the expectations that come with this type of funding.

A survey of several funders and funding groups revealed three areas where researchers can improve their efforts to meet the funder’s needs. These are communication, application of findings, and big picture thinking.

Communication deficiencies covered three distinct areas. Firstly, the ability of

the researcher to communicate to a nonacademic funder or audience through a succinct, easy to understand “elevator pitch” was in high regard. Ideally, researchers should be prepared to present themselves in this way to possible funders and networks.

Secondly, all funders discussed the desire to be kept in the loop, and highlighted this as a rare occurrence rather than the norm. More frequent and even informal communication is highly desirable. Finally, there is a desire that researchers communicate not only what has been achieved with the current funding but the direction of the research moving forward.

Having a research focus with an immediate application of findings, or tangible outcome, was considered attractive amongst those surveyed, particularly when requesting health service funding. One funder highlighted the need to examine quality of life and services for people suffering with certain conditions rather than always focusing on a cure, although both are obviously important.

Finally, there was a desire for researchers to broaden the focus when describing their research. Being able to articulate your current work in the context of a long term pathway to impact was highlighted both as valuable information for the potential funder and also creates an ability to communicate more broadly the big picture value of your work to diverse audiences.

Furthermore, presenting the next step creates an opportunity to secure additional funding without the usual competitive grant application process.

Researchers need to be the “salesperson” of their ideas; it’s not enough to know what you are doing, you must convince the funder that you can meet their needs and expectations throughout the process.

Access to philanthropic funding demands a different approach than gaining traditional government grants. It requires a different communication strategy and a solid understanding of the broader KT principles. You must align your research with the motivation of the funder, sell your idea and articulate the possible impact of the research. www.ktaustralia.com

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Winter 2015

HMRI Chosen As Test Site For Revolutionary Stroke Detector Clinicians working at the frontline of acute stroke care have a simple creed: “Time is brain”. In other words, the clock starts ticking from the moment a stroke strikes, and the sooner that treatment begins the greater the chance of minimising neurological damage.

Distance can be a decisive factor where regional-based health districts cover a broad geographical expanse, but a new portable imaging device called the Strokefinder MD100 is set to assist in saving time and lives.

The device holds promise of early stroke diagnosis – one day, it may allow treatment to commence en route to hospital.

Swedish firm Medfield Diagnostics has just signed an agreement with the Hunter Medical Research Institute (HMRI) to conduct the first international testing and further explore the diagnostic possibilities.

Medfield turned to HMRI because of its international standing in stroke treatment and research.

The Strokefinder employs microwave technology spawned from research at Chalmers University of Technology and Sahlgrenska University Hospital in Gothenburg. Having been adapted from mobile phone and defence applications, it is compact, powerful and has no known side effects.

Unlike with a large CT scanner, the patient’s head is placed directly on the

device and sequentially scanned by antennas. These low-energy pulses “scatter” in brain matter and can then differentiate bleeding patterns.

Pilot studies at Sahlgrenska, comprising over 200 stroke patients, have shown promising results and HMRI-aligned researchers from Hunter New England Health now plan to involve patients at John Hunter Hospital. They’re also set to hold discussions with long-term partner NSW Ambulance to explore potential collaborations in field-testing the device.

Professor Chris Levi, Senior Staff Neurologist at John Hunter Hospital, says that time-saving measures are critical when an ischemic stroke occurs, particularly in remote areas where travel to hospital often takes longer.

“The faster we begin thrombolysis treatment to dissolve the clot, the more brain can be salvaged,” Professor Levi said. “However, we must be absolutely sure that it’s not a haemorrhagic stroke, which occurs around 20% of the time, because the treatment paths are vastly different.”

HMRI Director Professor Michael Nilsson said that if the Strokefinder’s microwave platform could reach the level of CT scanning when it comes to diagnostic safety, clot-busting treatment should be able to start in the field.

“We are expecting the first Strokefinder to arrive later this year. There’s a long road ahead but the time-saving benefits are certainly worth pursuing as our region has a relatively high rate of stroke,” Professor Nilsson said.

“Our long-term ambition is to have the device fitted in a number of ambulances – we have a very progressive ambulance service that works closely with the stroke unit at John Hunter Hospital, and hopefully the Strokfinder can do for stroke patients what the “Packer Whacker” defibrillator has done for heart attack victims.” www.hmri.com.au MRI Director, Prof Michael Nilsson H with Strokefiner.

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Winter 2015

Lipid Pathways In Multiple System Atrophy Multiple system atrophy (MSA) is a degenerative brain disorder that impairs the body’s involuntary functions, like blood pressure, heart rate, and bladder function.

These processes are regulated by the autonomic nervous system. MSA also causes problems with muscular coordination and movement, like those seen in patients with Parkinson’s disease, and this is why MSA is often misdiagnosed as Parkinson’s disease. While the cause of MSA is largely unknown, studies point to a possible genetic component, as well as environmental factors capable of increasing susceptibility.

MSA affects men and women equally and begins primarily in the 50s, although it can strike as early as in the 30s. The progression of disease is rapid and patients are confined to bed within five years of onset of symptoms. Death results within about nine years. There is no cure or effective treatment.

Diseased support cells in the brain To tackle the problem of identifying what goes wrong in the brains of people with MSA, researchers from NeuRA study post-mortem brains from donors. The main finding so far is that there is a buildup of a protein called α-synuclein.

Interestingly, this protein also builds up

in other neurodegenerative illnesses like Parkinson’s disease and dementia with Lewy bodies; however, in MSA, the α-synuclein is deposited in cells called oligodendrocytes, rather than in neurons.

This build-up can lead to serious problems within these cells.

What is different about oligodendrocytes?

If neurons are the wires, oligodendrocytes are the plastic covering around them: they are support cells that help to insulate signals between neurons.

Oligodendrocytes produce myelin, a fatty membrane that wraps around the axon fibres of all neurons in the brain. Without myelin, neurons eventually die. Thus there is a need to identify the molecular pathways that specifically lead to disease in oligodendrocytes. Recent progress in the study of how oligodendrocytes handle fats and manufacture myelin heralds a promising advance.

Striking a balance between fats Research has found that before the α-synuclein aggregates are visible in the brain of MSA patients, myelin becomes structurally unstable. This provides a clue

to the series of events that lead to the eventual brain damage in the disease.

Myelin stability relies on a balance between the fats, or lipids, that it contains, particularly sphingolipids and cholesterol.

Our team has recently found that the lipid transporter ABCA8 is increased in MSA brain and is likely to have a role in myelin processing and maintenance. These data provide strong impetus for investigation of myelin lipid dysregulation in MSA pathology. The next line of investigation is to see whether lipid dysregulation causes buildup in the ‘culprit’ α-synuclein.

New findings in the MSA brain 78% of myelin is made of lipids. However, we haven’t previously been able to measure whether there are changes in lipid levels or distribution in the MSA brain. Our team at NeuRA recently undertook a comprehensive analysis of MSA myelin. We measured three groups of lipids, sphingomyelin, sulfatide and galactosylceramide, which are all important in myelin integrity and function, in a region of the brain affected in MSA, the motor cortex. We also looked in a control region unaffected in MSA, the visual cortex.

For all three groups of lipids, most of the species were severely decreased (by 40– 69%) in affected but not unaffected MSA brain regions. Based on this and other data we conclude that dysregulation of myelin lipids in the course of MSA pathogenesis may trigger myelin instability.

Lipid balance is increasingly recognised as a crucial factor for normal brain function and our data is among growing evidence that altered lipid levels and/ or distribution contribute to a number of neurodegenerative diseases. Future studies into the unique lipid-related processes underlying MSA pathology could reveal molecular targets that could be exploited to regulate α-synuclein aggregation in oligodendrocytes, providing potential therapeutic avenues for the treatment of MSA. www.neura.edu.au Dr Scott Kim.

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Winter 2015

International Collaboration Seeks To Make Childbirth Safer In Developing Countries A global alliance led by GSK and Monash University promises to fast-track delivery of oxytocin to mothers in developing countries.

Every year, thousands of mothers die from a preventable cause of death: post-partum haemorrhaging, the excessive loss of blood after childbirth. Despite advances in maternal health, haemorrhage remains the leading direct cause of maternal death worldwide.

Between 2003 and 2009, haemorrhage accounted for about 661,000 reported maternal deaths. Over 99% of those deaths occurred in developing countries.

Post-partum haemorrhage can be treated by a simple injection of oxytocin immediately after childbirth. Oxytocin has long been considered the gold standard therapy; the World Health Organization recommends that the hormone should be considered the “first choice” treatment of medical practitioners in preventing haemorrhaging during labour.

While synthetic oxytocin is readily available in birthing clinics in developed nations including Australia, it is a different story elsewhere. In developing countries, access to the medicine is impeded by logistics and the fact that many women give birth at home: oxytocin requires refrigerated transport and storage, as well as skilled health workers to administer the injection.

The conditions in these often underresourced countries make it difficult for mothers to receive oxytocin injections in a safe and timely manner.

A new global collaboration involving GSK, the Monash Institute of Pharmaceutical Sciences (MIPS), the McCall MacBain Foundation, the Planet Wheeler Foundation and Grand Challenges Canada may soon offer a solution. With GSK and the MIPS

In Australia, GSK continues its commitment to R&D through the GSK Award for Research Excellence and accompanying $80,000 grant.

Nominations close, 3 July 2015 Visit www.gsk.com.au

at the helm, these organisations have committed to accelerating the development of a new dry-powder inhaled form of oxytocin.

The technology was first developed by Dr Michelle McIntosh and her team at the MIPS, and is now being co-developed with GSK. Dr McIntosh explains the marriage: “At Monash, we bring the product concept, pre-clinical research and scientific development to the table but we don’t bring manufacturing, commercialisation and distribution – that’s why we needed to form a partnership.”

The new formulation will allow mothers to inhale oxytocin immediately after delivery.

Importantly, it will be heat-stable and not require costly and logistically complicated cold-chain transport systems. The inhaler device would facilitate administration by minimally-skilled birthing attendants.

As part of the collaborative agreement, GSK has been licensed to co-develop, register and distribute the product in regions of high maternal mortality.

McCall McBain has provided $1.5m, a figure matched by other donors. Grand Challenges Canada has contributed

roughly $1m in partnership with the Canadian Government’s Department of Foreign Affairs, Trade and Development. A further $500,000 has been provided by the Planet Wheeler Foundation, and GSK has contributed funds and in-kind contributions valued at $16m. The pooling will enable collaborative teams from Monash and GSK to conduct an early-phase development programme over the next few years.

The programme will be comprehensive, spanning across pre-clinical research, early-stage clinical trials, product optimisation, manufacturing and research into local markets.

Allan Pamba, Vice-President for GSK in East Africa and co-chair of the UN Every Woman, Every Child Innovation Working Group, said: “Pregnancy and childbirth should be an incredible time.

But for thousands of women, often in the world’s poorest countries, it puts their life in jeopardy. For the sake of new mums and their babies – a child’s health and prospects are better if they have their mother – this needs to change. In GSK’s Maternal and Neonatal Health Unit, R&D, we are constantly challenging ourselves to find new interventions that improve the life chances of mothers and their newborns.

Collaborating with others is fundamental to these efforts and we are delighted to partner with Monash. By combining their technical expertise with our experience in drug development and respiratory knowhow, we have the potential to give new mothers a fighting chance.”

Pre-clinical trials have so far shown that the hormone is effectively taken up by inhalation. According to Dr McIntosh, in the best-case scenario, the product could be ready for use in 2019. Thanks to the alliance, the global vision to improve maternal mortality rates in the developing world may soon become a reality.

There are no plans to market inhaled oxytocin in Australia in the foreseeable future. www.gsk.com.au r Michelle McIntosh (front, fifth from left) D and her team at the Monash Institute of Pharmaceutical Sciences are developing inhaled oxytocin with the help of an international collaboration.

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Winter 2015

Rare Voices Australia Hosts A Rare Disease Summit Rare Voices Australia (RVA) hosted a Rare Disease Summit 27 – 28 March 2015 in Melbourne, titled A National Rare Disease Plan – Driving Collaboration, Driving Action.

The Rare Disease Summit brought together opinion leaders from patient, healthcare, research, government and industry organisations to share knowledge and insights for future action. Over 120 delegates from across the country, with international representation from New Zealand, Switzerland, United Kingdom and Singapore. The two day summit allowed participants to join the conversation and bring consolidated input to the dialogue driving A National Plan for Rare Diseases.

RVA is encouraged to see many voices now working together to drive collaboration and action, and is calling for the Federal Government to adopt a national approach to rare diseases. “In Australia there is no rare disease policy, national strategy or plan. Whilst RVA has received bipartisan support in Federal parliament, the Government are yet to endorse and support a National Rare Disease Plan” says Megan Fookes, Executive Director of Rare Voices Australia. “There is much at stake.

We need a rare disease policy to help people suffering from rare diseases to gain access to co-ordinated care, services and treatments which can extend and improve their quality of life.”

Senator Richard Di Natale gave an opening address at the Summit, urging RVA to continue its advocacy, and praised its activities to ensure the collective voices are heard. He acknowledged the constant uphill struggle of thousands of Australians who live with a rare disease. “We’ve got to make sure rare diseases are recognised as a priority ... that people get access to the best available treatment. We’ve got to fix or improve what is generally a good system when it comes to providing

people with access to new drugs, but we’ve got to make it easier for people who are diagnosed with one of these rare conditions. It’s no longer acceptable that somebody who is unlucky enough to be born with a rare disease has to fight every step of the way.... to get diagnosed and access to the treatment we know works,” Senator Di Natale said.

The Summit attendees unanimously agreed that a National Plan for rare disease needs to be adopted by the Australian Government. People with rare diseases deserve attention and their needs are very specific. At the networking function, guest speaker Parliamentary Secretary to the Treasurer Kelly O’Dwyer highlighted the importance of investment towards innovation and research.

A draft Communique outlining the key Principles and Objectives for a National Plan was proposed for endorsement from organisations from around the country asking for Australia to please listen.

These key principles of the Summit Communique include:

1. Rare diseases need to be recognised as a national health priority

2. More equitable and timely access to diagnostics, treatments, services and coordinated care for people living with a rare disease.

3. Incentives are required to drive a coordinated and collaborative action on rare diseases. A national approach should be established, backed by the Australian Government.

4. Leaders from patient, healthcare, research, and government and industry organisations need to work collaboratively to champion a National Plan.

5. The National Plan should identify a set of objectives and enabling strategies to drive action.

Six key themes of the objectives are:

i.

national coordinated and A collaborative approach.

ii. Data collection and use

iii. Coordinated care

iv. Equitable access to services

v. Equitable access to diagnostics and treatment and,

vi. Nationally coordinated research

People living with a rare disease need better support and coordination of care.

A national co-ordinated approach which provides equitable access to diagnosis, care and quality services in the area of rare disease is long overdue.

For more information on the Rare Voices Australia – Rare Disease Summit, filmed sessions, presentations and a copy of the Summit Communique, please go to www.rarevoices.org.au are Disease Summit Steering Committee R Members; (left to right); Professor Jeff Szer (Professor/Director, Department of Clinical Haematology and Bone Marrow Transplant Service, The Royal Melbourne Hospital), Nicole Millis (Mucopolysaccharide & Related Diseases Society Australia), Professor Jack Goldblatt (featured on iPad image Director, Genetic Services of WA and Familial Cancer Programme), Megan Fookes (Executive Director; Rare Voices Australia), Cameron Milliner (Shire Australia), Shelley Evans (Genzyme), Jenny Sturrock (Paroxysmal Nocturnal Haemoglobinuria Support Association Australia & RVA Board of Directors), Lisa Adams (Independent Facilitator).

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Winter 2015

Bringing Medicine, Engineering, Science And Industry Together Biomedical research in Australia stands on the threshold of a new era of growth and discovery but requires leaders with vision to help make it happen.

The planned Aikenhead Centre for Medical Discovery (ACMD) in Melbourne will be Australia’s first biomedical engineering research and education centre.

of chronic disease in the community and create new, world-leading industries in device creation, tissue engineering and drug design and delivery.

Bringing together research and training from hospitals, high-tech manufacturing industries, universities and medical research institutes in a purpose-built centre, the ACMD will drive medical innovation and position Australia as a global leader in the fast growth industry of biomedical engineering.

The second is to co-locate tertiary students alongside experts in the new facility.

Support for the project has been strong.

The ACMD’s partners – St Vincent’s Health Australia, the University of Melbourne, Swinburne University of Technology, the Bionics Institute, the O’Brien Institute, Australian Catholic University, RMIT University, the Centre for Eye Research Australia, the University of Wollongong, and St Vincent’s Institute of Medical Research – have put up $60m of their own funds to get the project off the ground.

The Victorian Government led by Premier Andrews has matched the sum and is championing its development. The final piece in the puzzle is the Commonwealth Government which has been asked to invest the remaining $60m to turn the ACMD from a blueprint into a reality.

The opportunities associated with the ACMD make such an investment rich with reward.

In the space of a decade we expect the ACMD to deliver $4.8 billion in total economic benefits, kick-starting a host of new manufacturing and export opportunities. We estimate the centre will create up to 10,000 new jobs in the biomedical engineering industry. A further 1,000 construction jobs will be created from the capital works. It’s also projected that the ACMD will save Australia’s healthcare system $550 million over 10 years and extend the working life of Australians by up to five.

The ACMD model has two distinct features: the first is to assemble leading Australian experts in bioengineering and medical science – with a proven capability – to solve complex health problems in a hospital setting.

Through concurrent research and development collaborative work practices, the ACMD’s goals are to reduce the burden

We will bring together skills in medicine, medical research, nursing, allied health, biomedical engineering (tissues, drug design and bionic devices), engineering (electronic, electrical, material and software), biotechnical platforms and education.

By co-locating experts from various disciplines in the new building at St Vincent’s Hospital in Melbourne, it will allow clinicians to be exposed to the latest advances in bioengineering and material science, and engineers and scientists to be exposed to clinical research and care.

This will produce a new generation of highly skilled professionals who understand each other’s disciplines and will work collaboratively to make new research discoveries that can be fast-tracked to clinical use.

support, IT infrastructure, technical support, platform technologies and flexible laboratory designs all contributing to efficiencies.

Already, commercialisation of research underpins an industry that employs over 40,000 people and exports $3.8 billion of goods in this country. The ACMD will build on that and will secure Australia’s place as world leaders in this market into the future.

This is a once-in-a-generation opportunity.

The Aikenhead Centre will be unique in Australia and rare internationally, but if we don’t act now we’ll end up exporting our brightest minds overseas instead of exporting better care, better treatments and income-generating better health technology to the rest of the world. www.svha.org.au Proposed ACMD building

A major focus of the ACMD will be the fabrication of medical devices which have a much shorter lead time from development to market than pharmaceuticals. Central to that success will be our partnerships with industry.

We believe the ACMD’s biomedical engineering focus will attract commercial research contracts and new philanthropic funding resulting in less dependence on government.

Economic benefits will also be delivered to our partner organisations.

The ACMD will reduce operational expenditure and increase staff and capital efficiencies with shared laboratory

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Winter 2015

Value Of Medical Technology: Saving Lives And Healthcare Costs The Medical Technology Association of Australia (MTAA) Value of Technology (VOT) research improves the understanding of the impact of advances in medical technology on healthcare expenditure in Australia, and the associated costs and benefits for the Australian healthcare system and community. VOT research provides support for advocacy for funding of a range of technologies that might not have strong Australian evidence to date and/or lack funding.

Insulin pump therapy delivers fast acting insulin continuously through a small needle or cannula inserted and left in place under the skin. It is costeffective, and has several clinical advantages over conventional multiple daily injection (MDI) therapy.

These include: • better glycaemic control and prevention of complications • a reduction in the insulin dose per day, and • improved quality of life for the patient and their family, including a reduction in chronic fear of severe hypoglycaemia, and more flexibility of lifestyle.

In 2010 there were 1,310 Australians on the kidney transplant waiting list and only 548 kidney transplants, therefore most individuals with end stage kidney disease rely on renal dialysis for survival. Home dialysis has been shown to be the most cost-effective form of dialysis; however, only around 30% of individuals dialyse at home.

An increase in the use of home dialysis over the next 10 years is estimated to result in net savings of $378 - $430 million for the Australian healthcare system.

In Australia, there are approximately 700 – 800 new cochlear implant recipients each year; however, as many as 84,000 more people would benefit from a cochlear implant. Improved patient access to cochlear implants has flow on effects, including improved employment opportunities, socioeconomic status and health-related quality of life. The cochlear implant is extremely cost-effective, generating important health benefits at reasonable direct costs and providing net savings to society if benefits translate into reduced educational costs and increased earnings.

Atrial fibrillation (AF) is the most common cardiac arrhythmia, and can lead to serious complications such as heart failure and stroke (cause of 15-20% of strokes).

The estimated annual cost of AF is at least $1.25 billion per annum. Catheter ablation is a proven treatment for patients with AF and arrhythmias, and is associated with a high rate of acute procedural success and low follow-up rate. Treatment of AF using catheter ablation is associated with substantial cost savings gained through: • reduced hospitalisations and GP visits • reduced medication use and carer’s duties • improved quality of life and quicker recovery and return to work.

Remote monitoring of chronic heart failure (CHF) patients with implantable cardiac devices may result in decreased direct and indirect health costs. For example, the implementation of remote monitoring of implantable cardiac devices in Australia has the potential to deliver cost savings of approximately $115 million per year as a result of a 21% reduction in CHF and CHF-related hospital admissions.

Australia has one of the highest rates of overweight and obesity among developed countries. Bariatric surgery, including a range of procedures such as laparoscopic gastric banding, sleeve gastrectomy and Roux-en-Y gastric bypass, has proven to be a successful method in treating individuals who are morbidly obese. In Australia, surgically-induced weight loss has been shown to be a cost-effective intervention for managing obese patients with Type 2 diabetes, with mean medication costs 1.5 times higher for conventionally treated patients compared with patients who underwent bariatric surgery.

Imaging technologies play a key role in cancer diagnosis. For example, mammography is generally the first imaging modality used in breast cancer screening, and may be supplemented with ultrasound, particularly in young women with dense breast tissue. While magnetic resonance imaging is used mainly in women identified as being high risk for breast cancer.

Osteoarthritis is a major contributor to the musculoskeletal disease burden in Australia, and is one of the most common causes of chronic knee pain. Total knee replacement surgery has been shown to be highly cost-effective ($12,000 per disability-adjusted life-year), improving the quality of life of patients with osteoarthritis.

Radiotherapy is an effective treatment for primary and advanced cancers, and can be used either alone or in combination with surgery and/or chemotherapy to reduce the size of cancers and alleviate symptoms.

More than $2.6 billion per year is spent by the Australian healthcare system on chronic wounds (greater than $60,000 annually per individual with a wound). The high cost of chronic wounds, which includes venous leg ulcers, diabetic foot ulcers and pressure ulcers, is often due to the increased length of stay in hospital and number of hospitalisations.

Modern wound care devices offer considerable clinical and economic benefits over traditional wound care treatment, including a reduction in the: • number of dressing changes required • healing time of the wound • clinician and nursing time for assessment and treatment • cost and frequency of complicating infections, and

In vitro diagnostic tests are used in cervical cancer screening to detect pre-cancerous cellular changes in epithelial tissue of the cervix that could develop into invasive cancer.

In Australia, at least 18,000 healthcare professionals suffer from needlestick and sharps injuries (NSIs) every year. Safetyengineered medical devices (SEMDs) can be used in place of most conventional needles and sharps devices, and are very effective in reducing the number of NSIs. Implementing the use of SEMDs in all Australian hospitals would result in cost savings of at least $18.6 million per year for the Australian healthcare system.

Chronic pain puts considerable strain on the economy through lost productivity, disability and healthcare utilisation. The use of continuous drug infusion pumps post-surgery has been shown to reduce the need for analgesics, the risk of surgical site infection and the length of hospital stay by between one and three days.

Potential cost savings of approximately $600 million per year could be achieved if pain infusion pumps were used in Australian hospitals following the most common surgical procedures.

• number and length of hospital stays.

MTAA would like to acknowledge the contribution of its members and the following stakeholder organisations:

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For further information on MTAA VOT research: www.mtaa.org.au/about-the-industry/value-of-technology

Winter 2015

Compounds Australia Facilitating New Spinal Cord Research Compounds Australia, a sophisticated compound storage and management facility housed within Griffith University, is making a major contribution to advanced medical research.

Based at Griffith’s Eskitis Institute for Drug Discovery, Compounds Australia maintains compound libraries submitted

by Australian-based chemists and ensures compounds are readily available for screening by biologists.

Among recent projects, Compounds Australia has been pivotal to research analysing the therapeutic efficacy of the natural compound curcumin for spinal cord repair.

A team led by the Eskitis Institute’s Dr James St John is working on processes through which glial cells from the olfactory mucosa – located in the upper region of the nasal cavity – are relocated to the damaged spinal cord. Glial cells are the supporting cells of the nervous system and can help nerve fibres to regenerate.

Curcumin comes from the spice turmeric and has been discovered to stimulate the cells that are used for transplantation into the spinal cord.

Compounds Australia obtained and screened numerous analogues of curcumin to identify those with higher biostability and efficacy. This process supported work by one of Dr St John’s team, researcher Ms Rebecca Yao, as she completed her Bachelor of Biomedical Sciences Honours degree.

obtained or synthesised many analogues of curcumin.

“Within 2-4 weeks, this resulted in another 75 analogues being supplied by collaborators directly to the researcher.”

Dr St John agrees Compounds Australia’s role was crucial to the project.

“As well as organising the storage, preparation, dilution and plating of the compounds for the assays, it supplied repeat preparations of the plates as needed for additional assays,” he says.

“With the central involvement of Compounds Australia, this project has now identified several structural analogues of curcumin that will be further investigated for their improved therapeutic efficacy.”

Compounds Australia began life in 2008 as the Queensland Compound Library, starting after the receipt of 150,000 samples from Cancer Therapeutics CRC, a collaborative partnership of leading research institutes, universities and biotechnology companies.

Relaunched late last year as Compounds Australia to better reflect the facility’s expanding role in enabling vital scientific research in Australia, it has since embarked

on a concerted program of growth and diversification to boost its collection of compounds and extracts and to enhance benefits for industry and academic research.

As well, Compounds Australia has joined with EU-OPENSCREEN and Therapeutic Innovation Australia in signing a Memorandum of Understanding to allow the sharing of compound collections between Australian and European researchers.

“This demonstrates that while we are bringing together compounds from the Australian chemistry research community, we are also looking internationally. The relaunched facility exemplifies that broader capability and vision,” says the Chair of Compounds Australia, Professor Sally-Ann Poulsen.

As Australia’s only dedicated compound management facility, Compounds Australia offers industry standard storage conditions, high end laboratory automation, access to compounds in assay-ready microplate formats and a dedicated team with years of experience in compound management. www.griffith.edu.au/science-aviation/ compounds-australia

“Using chemical fingerprint and tanimoto similarity, Compounds Australia searched the Open Access Compound collections of 55,000 unique compounds for chemical structures similar to curcumin,” says the Manager of Compounds Australia, Ms Moana Simpson.

“We also capitalised on our ever increasing network of Australian chemists by issuing an email requesting further information and possible submissions of analogues to Ms Yao. “While Compounds Australia immediately identified 10 structural analogues held within our collection, the highlight was the response from Australian researchers providing information among themselves and identifying two other researchers, not included in the original email, who have Professor Sally-Ann Poulsen

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Winter 2015

A SURE Thing Researchers are now able to securely access highly sensitive data from anywhere in Australia through SURE: the Secured Unified Research Environment, Australia’s first remote-access data research laboratory developed by the Sax Institute.

When Dr Anna Kemp started her research into the therapies used by Australian women diagnosed with early stage breast cancer four years ago, she had to pack her bags and relocate from Perth to Sydney in order to access crucial, secure Medicare data.

Dr Kemp, Research Fellow at the Centre for Health Services Research at the University of Western Australia, spent 16 months based at the Sax Institute in Sydney in order to access linked population data from the 45 and Up Study, Pharmaceutical Benefits Scheme and Medicare Benefits Schedule.

But the launch of SURE: the Secured Unified Research Environment, in 2012, meant Dr Kemp has since been able continue her research back in Perth, and more recently, from her new home on the NSW south coast.

SURE, developed by the Sax Institute and launched in 2012, is Australia’s first and only secure, high-powered computing environment that researchers can access remotely in order to analyse the large

volumes of sensitive data contained in registries, cohorts, routinely collected administrative data and the linkages between them.

To date, 130 researchers have made use of SURE, and there are now 40 research projects underway using the network with another 20 project applications pending.

Dr Kemp and fellow researchers from the universities of South Australia, Sydney and Notre Dame are using the SURE facility to continue their analysis of de-identified hospital, cancer registry, medication and Medicare records of 1500 Australian women with newly diagnosed breast cancer participating in the 45 and Up Study.

“What is so exciting about SURE is that really important, sensitive health records can now be securely accessed by researchers from all over the country, and this provides more opportunities and scope for people working in regional universities to carry out great work,” Dr Kemp says.

She says other secure data models that were under consideration before SURE was established would have seen researchers having to travel to secure sites in capital cities to access data, or perhaps having to submit requests for data analysis rather than being able to access the datasets and undertake their own analyses.

“There are so many stages of analysis, it would have been unworkable to have data like this,” she says.

She said the launch of SURE enabled her team to undertake the most comprehensive research to date into what actually happens to women with breast cancer in the Australian community, whereas previous studies had relied on self-reported data.

Three papers with important implications for future policy and clinical practice have already been published from the research project, including research that shows more than half of Australian women with breast cancer stop their hormone treatment within five years, despite strong Dr Anna Kemp

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evidence they should continue endocrine therapy for much longer to minimise the risk of cancer recurrence.

A further two papers are close to completion: one focusing on recurrence of cancer and the other looking at health service usage patterns among women who discontinue therapy earlier than recommended.

SURE manager Joanna Khoo says the SURE project aims to promote “Big Science” in health and social science research by boosting Australian researchers’ capacity to conduct largescale, collaborative research projects that tackle major health and social issues.

Each accredited researcher is allocated a virtual computer, which runs entirely on hardware physically located and controlled

by SURE. The researchers see a facsimile of the remote virtual computer screen on their local screen, eliminating the need for them to use their local computing environments, which may have technical and security limitations.

Because SURE can be accessed remotely by researchers from different institutions at different sites across Australia or even overseas, it offers the opportunity for collaboration on important, large-scale research projects in the national interest.

“The opportunity SURE presents is for researchers to collaborate and analyse data that was previously very hard to access,” Ms Khoo says. “For researchers, the benefit is access to data, and for data custodians it allows access with better protection of data.”

Dr Kemp agrees: “I think SURE is securing the future of linked health research in this country,” she says. “It’s an absolute game changer.” www.saxinstitute.org.au

Winter 2015

Amalgamating 15 Medical Research Foundations Into One The organisation uniquely had 15 internal medical research foundations whose objectives were to increase the resources of the organisation to support identified areas of medical research, education and scholarship.

Each foundation had its own constitution, council, staff and passionate donors, many of whom were prominent members of the Sydney community. Some had been operating for over 50 years. During the period 2009 to mid-2014 my role was to strategically implement a standard constitution and ensure compliance with legal and governance standards within an overarching organisational strategy whilst ensuring the important relationships that had been developed were not lost in the change process.

I developed a strategy to amalgamate these internal foundations as a part of an over-arching strategy to provide professional fundraising and administrative support to increase the effectiveness of the resources available whilst maintaining the passion and support for the broad range of medical research been undertaken. To date 12 of these foundations have amalgamated into one focused medical research foundation and the results show that the income over the consolidation period has increased – $6.5mil to $22.5mil.

been considered – no actual or perceived conflicts – just a decision of peers.

I was also able to call upon leaders in the organisation to articulate the strategy to their peers and provide leadership in embracing the changes.

By introducing current governance standards, and implementing them – fixed terms and nominations committees, declarations of external interests, ensuring council members had philanthropic networks, not research skills, ensuring clear financial reporting and then budgeting were a part of the meeting agenda, it become easier to attract council members who had fresh networks, new ideas and enable the researchers to

spend more time on their research, not fund raising.

Advocating for those issues, their implementation, the politics involved, the constant conversations, the many stakeholders who need to be consulted and the influencing of those who made final decisions made the role challenging and the outcome to date worthwhile.

And what remains continues to reflect the history of the organisation, its place in the community and the passion of its many donors and supporters. www.sydney.edu.au hauna Jarrett was an Assistant Group S Secretary and Provost’s Nominee on each of Sydney Medical School foundations at the University of Sydney.

How was this done in the passionate and competitive world of medical research?

I firstly gained the trust of the council members; provided clear and frank advice as to the strategy and the desired outcome. At times these discussions were robust and I had to call on my skills as an advocate and litigator, defending my side of the argument. I listened to the concerns raised and encouraged council members to address the issue of sustainability of their foundation, what were the skills and networks they needed to have and how the organisation could provide on-going support for their particular area of research.

The reality of the shrinking income for each foundation could be counteracted by the benefit of shared support.

I also had a clear alternative model that was to replace the former way of doing things. The model was able to demonstrate how the sharing of administration and the use of professional fundraisers increased the income for their objectives. There was also recognition of the transparency needed around grant making with the establishment of a Scientific Advisory Committee which called upon experts in a particular area when grants were

Research Australia grassROOTS WINTER 2015

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Board of Directors Chair Professor Christine Bennett AO Dean, School of Medicine, Sydney University of Notre Dame Australia

Deputy Chair Peter Wills AC

Elizabeth Foley CEO & Managing Director Research Australia Dr Alison Butt Director Research Investment National Breast Cancer Foundation Shelley Evans Patient Advocacy Director Genzyme Associate Professor Mary Haines Director Strategic Research Investment Cancer Institute NSW Professor Janet Hiller Dean, School of Health Swinburne University of Technology Geoffrey Joyce Executive Director Macquarie Capital Dr Anna Lavelle CEO AusBiotech Ltd Professor Brendan Crabb AC Director & CEO Burnet Institute

Professor Alexandra McManus Director Centre of Excellence Science Seafood & Health Curtin University Barry Thomas Vice-President, Director Asia Pacific Cook Medical Dr Andrew Nash Senior Vice President, Research CSL Limited Professor Richard Head Deputy Vice Chancellor, Research & Innovation University of South Australia Professor John McGrath Executive Director Queensland Centre for Mental Health Research Andrew Giles CEO Garvan Research Foundation Associate Professor Greg Kaplan Chief Operating Officer Ingham Institute

Editor’s Corner Who can put an article in grassROOTS? grassROOTS is for and by members of Research Australia and is designed to showcase the activity in philanthropy for health and medical research, through either fund raising activity, awards, or the results of actual research funded by philanthropy. It is also a vehicle for the broader community to understand the importance of philanthropic funding and how they can contribute to the expansion and improvement in health and medical research in Australia.

When will I need to get my article in by if I want to be featured in the next edition?

The article submission deadline is 22 July, 2015. Due to high demand for editorial space and to ensure the magazine maintains its readability and audience engagement levels the article word count has been reduced to 600 – 650, and please provide one or more photos (with subtitles) and logo to accompanythe article.

Please submit articles via email to: danijela.krha@researchaustralia.org Research Australia does not warrant or guarantee the accuracy, quality, completeness, currency, or validity of any information on its website or newsletter. Some of the materials in classified ads, press releases, and newsletters are also provided by other organisations. Research Australia does not edit or control the financial information it receives. Due to the possibility of human and mechanical error, neither Research Australia nor any of the contributors to this newsletter are responsible for any errors or omissions. All information is provided “as is” without warranty of any kind. Neither Research Australia nor the contributors to this newsletter make any representations as to the accuracy or integrity of the information. They disclaim all express, implied, and statutory warranties of any kind, including warranties as to accuracy, timeliness, completeness, merchantability, or fitness for any particular purpose. Neither Research Australia nor its contributors will be liable for any damages of any kind incurred as a result of the information contained within this newsletter or on this site.