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Global perspectives on vital diagnostic technology and precision medicine for blood diseases P6-7 THE FIRST VIRALLY INACTIVATED HUMAN PLATELET LYSATE
EFFICIENT SAFER* STANDARDISED
alternative to FBS to expand cell therapy products www.platelet-lysate.macopharma.com
* Than their predecessors MultiPL’30 and MultiPL’100. MultiPL’ contains human source material and must be treated as potentially infectious. Despite all testing, proper safety precautions for potentially infectious agents must be taken. For research use and ex vivo cell culture processing only and not intended for human in vivo administration. This document is for the exclusive use of the healthcare and cell culture professionals. XB2RA05A - 04/2018
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IN THIS ISSUE
“For me, having myeloma doesn’t mean life stops,” says Bob Munro. Read his inspiring story
CAR-T and blood cancer awareness. What you need to know.
“How haematologists are revolutionising how we treat and manage disease.” Royal College of Pathologists
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Global collaboration leads to better healthcare for blood disease patients
Blood diseases exert a huge toll on low- and middle-income countries. By providing local medics with access to global experts, the benefits to patient healthcare can be improved.
H
a e m a to lo gi ca l diseases comprise a broad spectrum of disorders that affect the blood or the blood-forming tissues, such as the bone marrow, and may be either malignant or non-malignant in origin. Among the blood cancers, leukaemia, lymphoma and myeloma are among the most prevalent, whereas non-malignant blood disorders encompass various types of anaemia and a range of haemorrhagic conditions. In the UK and other developed countries, patients with blood disorders have access to sophisticated diagnostic facilities and the latest treatments. Unfortunately, the same cannot be said of lower- and middle-income countries, which
require support from the global community to ensure that patients with haemetological conditions receive the proper care they need.
most likely to be impacted by it.
Global approach key to healthcare
A lack of diagnostic tools in developing countries Countries with developing economies often possess limited screening facilities and diagnostic tools, while access to the latest treatments is thwarted by a scarcity of resources and a limited healthcare infrastructure. The cost of essential technology used for diagnosing and monitoring blood diseases means that low- and middle-income countries are frequently without access to these major diagnostic tools, resulting in patients not being treated as adequately as they might. Non-malignant blood disorders exert a huge health toll, too. In developing countries, conditions such as
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Professor Adrian Newland CBE Immediate Past President and Council member, the International Society of Hematology
sickle cell anaemia, thalassemia and haemophilia are expensive to treat. Even anaemia, the most common of all blood disorders, carries a huge health burden, especially when children and pregnant women are the ones in the community
The International Society of Hematology is a global organisation which facilitates collaborations, teaching and training through its bi-annual World Congress and journal, Hematology. Working in partnership with national societies and the World Health Organization (WHO) to provide world-wide education and advice on healthcare to lower- and middle-income countries. Collaborations at a global level help to provide the poorest countries with solutions to some of their most pressing problems when it comes to treating blood disorders. Developing diagnostic tests in the community and advising on what healthcare facilities should look like at a district level forms a large part of the advice offered by
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WHO and its international partners. In the absence of the latest treatments, support is also given to lowand middle-income countries in the form of expert-led training and digital resources that focus on providing best practice to clinicians with reference to what is available to them. At the same time, WHO produces country-level report recommendations aimed at health ministers and funding bodies who can use them to assess the health gaps in their countries. By working together with local clinicians and providing them with links to haematological experts at both a national and global level, it is hoped that the healthcare benefits of those living in developed countries can be shared with those suffering from blood disorders in more disadvantaged areas of the world. Victoria Briggs PLEASE RECYCLE AFTER READING
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New collaboration to improve outcomes for myeloma patients SPONSORED
Pioneering new myeloma database that measures treatment impact and tracks patient feedback shows how collaboration can mean a winwin for patients, government and industry.
In a UK first, NHS Wales and Welsh Government have partnered with Janssen UK, a pharmaceutical company of Johnson & Johnson, to pilot a new healthcare database for people with myeloma – an incurable blood cancer. In this personalised database, a much wider set of data will be collected from myeloma patients and prescribers, which will build on existing
clinical data with real world evidence that measures the effectiveness of current treatment pathways. According to Lee-Ann Farrell, Janssen’s Government Affairs Manager for Wales and Northern Ireland, “this important initiative will ensure that key information from registered myeloma patients can be collected at every step of a patient’s journey – from diagnosis, to treatment response and overall survival.” “Crucially, it will seek rich qualitative data from patients to better understand what they actually want from their treatment in terms of clinical outcomes and life-quality. By collaborating with NHS Wales and Welsh Government, we hope to build an infrastructure that supports more informed treatment decisions; helping to ensure patients receive the right treatment, at the right time, in the right way – and for the right reasons,” she says. Myeloma was chosen as the first blood cancer of focus for the new data collection system due to the
Lee-Ann Farrell Government Affairs Manager, Wales and Northern Ireland, Janssen UK
“There has been rapid therapeutic advancement in the treatment of myeloma in recent years.”
prevalence of the condition in the UK and the new treatment advances seen in this area. Historically, data capture for cancer patients in Wales has been through the Cancer Network Information System Cymru (CaNISC), however this was designed for solid tumours, and is therefore illequipped to capture the nuances of haematological cancers. CaNISC also struggles to capture quality-of-life data or treatment response – a scrutiny gap that leads to wasted resource and ineffective treatment for some patients. Dr Ceri Bygrave, Consultant Haematologist, Cardiff & Vale Health Board, NHS Wales, said: “We know that there has been rapid therapeutic advancement in the treatment of myeloma in recent years, however there is more still to be done. Through this partnership we hope to develop an advanced database which will measure the impact of treatment, ultimately benefiting outcomes for patients in Wales.” The first data collection is
expected to start before the end of the year, and the hope is that data collections for other haematolgocial malignancies will follow. For the Welsh Government, which has a significant resource investment in this project, the opportunity is exciting. Ms Farrell says: “This is an opportunity for the NHS in Wales to ensure that funding for myeloma is appropriately allocated for the processes and treatments that are effective and, that patients want and need as early as possible.” Partnerships following this innovative model can provide major benefits to patients, the NHS, government and pharmaceutical companies alike. All parties are looking forward to seeing the benefits in the months and years to come. Alisa Colquhoun For more about Janssen: janssen.com/uk @JanssenUK
Date of preparation: May 2018 Date of preparation: May 2018 PHGB/HEM/0518/0014 PHGB/HEM/0518/0014
Haematologists promote ‘inclusion over exclusion’ for children SPONSORED
Having a bleeding disorder no longer means children will be consigned to watching from the sidelines. Instead, exercise is helping to build physical strength and selfesteem as treatment promotes further inclusion.
Physical activity is important for us all, regardless of whether we have a bleeding disorder or not. But, as recently as the 1980s, children living with haemophilia could be left with damaging bleeds into the joints and muscles if they were to engage in sport, so often had to sit on the sidelines. Thanks to significant advances in treatment, however, children with haemophilia can now engage in sport and exercise, improving both their physical strength and self-confidence. For parents of children with
Dr Dan Hart Consultant Haematologist, The Royal London Hospital Haemophilia Centre
Dr Brian Colvin Formerly Director, The Haemophilia Centre, The Royal London Hospital (1977 - 2007)
haemophilia or other bleeding disorders, the desire to protect their child from harm can see them shielded from physical activity in early life.
when they’re older,” Dr Hart said. “As long as we encourage wearing a helmet, cycling is a fantastic way to keep children with bleeding disorders active. Otherwise, they can feel isolated from their friends.”
Cycling and swimming are great for building strength Dr Dan Hart, Consultant Haematologist at The Royal London Hospital, says learning how to swim and ride a bike at a young age can be safer in the long run. “Both of these things are life skills, it’s better and safer to let children learn with their friends rather than
The pro-cyclist with a bleeding disorder One boy stands out as an example of why having a bleeding disorder doesn’t mean you can’t achieve extraordinary things. Alex Dowsett’s story shows that children with bleeding disorders can still reach the pinnacle of a sport.
Alex was diagnosed with haemophilia at 18 months, when a bleed in his mouth didn’t stop. That day, a long journey of learning how to manage the disorder began. Alex learned to inject a clotting factor – which allows him to participate in sport – into his arm by the age of nine. He’s now a professional cyclist, riding his first Giro d’Italia in 2012 and is the Chairman of his own charity Little Bleeders which supports young people with haemophilia. Little Bleeders message to children living with bleeding disorders is “move more, be more’ and that they should have the confidence to pursue their sporting aspirations. For parents, managing a child’s expectations is key.
Advice to parents: Set achievable goals “Not every child will achieve what Alex has achieved, but our goal has to be minimising marginalisation of children with bleeding disorders. The goal is inclusion, not exclusion,” Dr Hart says. Dr Brian Colvin, former Director of the Haemophilia Centre at the Royal London Hospital, echoes the view that empowering children with bleeding disorders to join in is
essential. “There has to be a commitment from the parents that their child can lead a nearly normal life,” he said. “Some sports, like rugby will be out of the question, but parents can confidently encourage their child to swim or cycle. Not every child will be an elite athlete, but that’s true for all of us.”
Competence in a sport is very confidence-building Dr Colvin also pointed to both the physical and mental benefits of helping children with bleeding disorders move more. “If you have better control of the body’s moving parts, you’re more confident that you’re not going to have that accident that could lead to a bleed,” he said. Bleeding disorders shouldn’t stop children from enjoying their early life and being active. Managing their treatment and expectations allows them to flourish and increase selfesteem through being involved. James Alder Read more on littlebleeders.com @Littlebleeders
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Myeloma drug access is a ‘postcode lottery’
Rosemarie Finley Chief Executive, Myeloma UK
Effective myeloma drugs are available, but they are expensive. Equality of access to treatment across the UK simply must be a priority, according to Myeloma UK’s CEO, Rosemarie Finley.
Myeloma is one of the rarest forms of cancer, yet it carries the sixth highest death rate of them all. Just 5,700 myeloma cases are recorded in the UK each year. Its rarity means early diagnoses can be difficult, yet early diagnosis is vital for improving patient outcomes and quality of life. Rosemarie Finley believes educating GPs to spot warning signs earlier, and improving diagnostics, are a key focus for research. “We need a flagging system for GPs. Something that will alert them to consider myeloma if a patient shows specific symptoms.” Symptoms of myeloma are normally vague to start with (anaemia, fatigue, back pain, musculoskeletal non-specific pain, bruising, weight loss), which makes it tricky to detect or even suspect myeloma in the first place. Once there are more specific, yet more serious symptoms (such as bone disease symptoms, fractures and renal failure) - usually discovered via A&E route - the patient can have a much worse prognosis. It is a challenge to educate GPs and introduce a ‘flagging system’ of the right kind exactly, because key symptoms are so vague. Better diagnostics would also mean treatments could be far more focused and cost-effective in the long run, ending the ‘pick and mix’ approach often employed while correct diagnosis is achieved. Where you live impacts your quality of care In the UK, the quality of care for myeloma patients is directly linked to where they live, something Finley says must change. “Eligibility for drugs trials is based on your location, and equality of access to drugs simply has to happen,” Finley said. Finley did cite the vast improvement in treatment over the last 10–15 years. Treating patients with MGUS (monoclonal gammopathy of undetermined significance – a pre-condition of myeloma) is an important consideration for the future, so we are treating people sooner to have improved outcomes. Myeloma UK focuses heavily on improving public and professional awareness of the disease, as well as the support networks available to people affected by myeloma. “People often confuse it with the skin cancer, ‘melanoma’. Making people aware that myeloma is a blood cancer is as important as improving awareness of common symptoms,” Finley said. “There are many support networks available, with over one hundred support groups for myeloma and other related diseases, where people can meet and learn from others. We are very involved in health research and working with partners such as the Institute of Cancer Research and the University of Leeds, to advance the discovery and development of new treatments.
James Alder
Takeda Oncology and
Myeloma doesn’t stop me leading a great life For me, having myeloma doesn’t mean life stops, says Bob Munro. His story is one of pragmatism, intelligence and a will to contribute to raising myeloma’s profile.
B
ob Munro was 52, active, the owner of a relatively young business and was about to escape to the slopes to ski with friends. He went, despite feeling extraordinarily unwell. He had a token beer at the airport to show willing, but sat out the first day’s skiing and tried to recuperate ahead of day two. Having hauled his way to the top of the mountain, a friend asked whether he’d realised he had blood pouring from his nose.
“I’d never had nose bleeds before,” he says. Bob was unaware his thinned blood was being caused by a disease he hadn’t yet heard of - myeloma. “I was sweating profusely at night, and all an online advice website said, was: ‘go to the doctor’.” He deteriorated further still and was eventually rushed to hospital back in the UK with severe pneumonia. Further tests revealed the myeloma diagnosis that would change his life. A CT scan unearthed ‘lesions on the bones’ all over his chest and pelvis, but Bob didn’t immediately make the link with cancer.
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Bob remembers being told it was myeloma, along with phrases like ‘incurable’ and ‘improved treatments’ but the bombshell was ’tumour’. Bob and his family had to confront the disease he had likely had for two years or more. However, confront it he did. “The consultant said ’my firm advice is, don’t google it’. The survival stats are shocking. My wife was there and it was all a bit tearful.” But Bob still considers himself “lucky” having caught the disease at a relatively early stage. “I call myself ‘lucky man’ because I got pneumonia and discovered it
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before I had major bone or kidney damage. Some people don’t find out until they pick up their grandchildren and their ribs shatter.” His treatment didn’t start as he’d hoped, but he ensured he recorded what did or didn’t work. “I wanted to do all I could to survive. That meant being responsible for my own treatment and taking an interest in it.” When Bob’s treatment stopped working in 2016, he did his own research. He suggested a new treatment pathway to his consultant, which his private insurance was able to cover.
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Bob’s treatment started to work again. “I’m very fortunate that I’m able to have a fantastic, active life, even with myeloma. But it’s vital to engage with your treatment and consultant.” A summer spent recovering and watching the 2012 Tour de France sparked something in Bob. His new obsession with cycling provided an outlet for his competitive spirit and it has become a huge part of his own efforts to help Myeloma UK raise awareness of a disease 97% of people still haven’t heard of. “As a relatively unknown cancer, Myeloma UK needed funds to help
Bob Munro Patient and Volunteer for Myeloma UK
develop treatments and raise the awareness of GPs, who might only see four or five cases in their whole career. I thought, ‘what can I do?’” The answer: organise the first Myeloma UK cycle from London to Paris. The event raised £330,000 in its first year, in 2016. “Spotting myeloma early is incredibly difficult for GPs. Raising awareness and introducing screening, along with more personalised treatments, will be vital for improving outcomes and survival”. “Myeloma UK has an active drug development programme and acts as a powerful patient advocate to
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ensure newly-approved drugs are made available through the NHS, so I know the funds we raise go directly to benefit patients”. For now, he has the 2018 cycle ride to focus on, with Paris again firmly in his sights. “As the police cleared the route to around the Arc de Triomphe for us that first year, it was such a wonderful moment in my life. I can’t wait to do it again.” James Alder Find out more on myeloma.org.uk
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The role of flow cytometry in diagnosing blood cancers
Flow cytometry plays an integral role in helping doctors to detect disease and determine the best treatment pathways for patients. But what is flow cytometry and how does it work?
Flow cytometry is a vital technology used in the diagnosis and monitoring of a range of health disorders, particularly haematological cancers, immune deficiencies and HIV infection. The technology works by detecting the type of cells present in body fluids, such as blood, bone marrow and cerebrospinal fluid, or wherever a single-cell suspension can be made from cell culture or tissue samples. On entering the flow cytometer machine, the cells in the sample
Dr Katherina Psarra President, ESCCA
marked with detectable fluorescent dyes coupled to antibodies that are directed against appropriate cell structures has a laser beam directed at it. A series of electronic detectors then measure the degree of fluorescence emitted by the cells to determine its various properties. The process enables medical staff to recognise specific diseases and to distinguish between the different types of haematological malignancies that exist, such as leukemia and lymphoma, enabling treatment to be tailored to the type and stage of a patient’s disease.
A global diagnostic tool Flow cytometry is an essential diagnostic tool for haematologists and immunologists all over the world. Not only does the technology enable
precise diagnoses, it also allows residual levels of disease to be detected following a course of treatment. Before flow cytometry was developed, physical samples had to be analysed under a microscope—a process that only allows a counting of a few hundred cells and determining one or two structures on a cell at any one time. For a haematologist looking for evidence of any residual cancer cells in a patient’s body after treatment, it was not possible to measure residual aberrant cells. Faster computers, smarter software, more powerful lasers and better electronic detector apparatus mean it’s now possible to count millions of cells, thanks to flow cytometry. What’s more, it enables the data from a sample to be analysed very quickly,
so patients can find out in a very short space of time whether their treatment has been successful or not. As well as cell diagnostics, flow cytometry also plays an integral role in genetics, immunology and biomedical research. While flow cytometry training is not a standard undergraduate requirement in medical or biology degrees, certified postgraduate courses are available to take for those who already possess medical or biological sciences experience. The European Society for Clinical Cell Analysis (ESCCA) offers such education at ESCCA courses and conferences as well as the opportunity of becoming a certified cytometrist through ESCCA exams. Victoria Briggs
Understanding and treating blood diseases From life-long blood disorders, like sickle cell disease and hemophilia to sudden, life-altering blood cancer diagnoses, like leukemia or multiple myeloma, blood diseases can be the source of so much uncertainty for patients and their caregivers.
Fortunately, there is much to be hopeful for in the understanding and treatment of these disorders, and the American Society of Hematology (ASH) is working with scientists, research institutions, pharmaceutical companies, and policymakers to accelerate scientific discovery, drug development and deployment of new therapies to conquer blood diseases.
Harnessing our immune system to defeat cancer In 2017, a revolutionary new therapy made the leap from the research
Dr Alexis A. Thompson President, American Society of Hematology
bench to the bedside when the U.S. Food and Drug Administration (FDA) approved a groundbreaking CAR T-cell therapy to treat certain leukemia patients. In a process that once would have sounded like science fiction, this therapy involves taking a person’s immune cells from their bone marrow, reprogramming those cells to
target their cancer, and returning them to the patient. CAR T-cell therapy is a prime example of precision medicine’s potential to revolutionize the care we deliver to our patients. CAR T-cell therapy has also been approved for some patients with another blood cancer called lymphoma. Almost every day, clinical trials are reporting further success in treating patients with other types of blood cancers, such as multiple myeloma, using CAR T-cells. ASH is committed to improving the safety, effectiveness, and availability of revolutionary cancer therapies, and we are actively promoting research to hasten their delivery.
Sickle cell disease— from the first discovered to the first conquered molecular disease Sickle cell disease (SCD) is an
inherited blood disorder that affects nearly 100,000 Americans, primarily of African, Mediterranean, and Middle Eastern descent. SCD causes red blood cells to become rigid and sickle-shaped, leading to reduced oxygen flow to almost every organ, causing crises of severe pain, stroke, organ damage, and even death. While treatment options are currently limited for those with SCD, several research teams from around the globe are using the latest advancements in precision medicine to make cures in SCD possible in the near future. This includes calling on gene therapies and genome editing techniques to correct the genes responsible for this disease. While it’s still too early to deliver many of these therapies and cures to people, scientists are hard at work making them a reality.
Today, tomorrow, and beyond In the 1960s, the first successful trials in chemotherapy were reported in people with leukemia. Today, with scientific breakthroughs in precision medicine, hematologists are mapping the frontiers of medicine, and ASH is honored to play a key role in fostering this groundbreaking work.
Get involved
For those interested in learning more about blood health, the importance of funding biomedical research, and how you can get involved, please visit: hematology.org/patients If you would like to donate to our efforts to conquer blood diseases, please visit: hematology.org/foundation.
Complete blood count (CBC) test: What is it and when is it ordered? The complete blood count (CBC) is a very common test. Many people have a CBC performed when they have a routine health examination. If a person is healthy and has results that are within normal limits, then that person may not require another CBC until they become ill, their health status changes or until their healthcare provider feels that it is necessary.
A CBC may be ordered when a person has any number of signs and
symptoms that may be related to disorders that affect blood cells, including white blood cells (WBCs) that protect against infections, red blood cells that carry oxygen throughout the body and platelets that help control bleeding. When an individual has fatigue or weakness or has an infection, inflammation, bruising, or bleeding, a health practitioner may order a CBC to help diagnose the cause and/or determine its severity. When a person has been diagnosed with a disease known to affect blood cells, a CBC will often be ordered on a regular basis to monitor their
condition. Likewise, if someone is receiving treatment for a blood-related disorder, then a CBC may be performed frequently to determine if the treatment is effective. Some therapies, such as chemotherapy, can affect bone marrow production of cells. Some medications can decrease WBC counts overall. A CBC may be ordered on a regular basis to monitor these drug treatments. Significantly abnormal results in one or more of the different types of blood cells can indicate the presence of one or more conditions. Typically, other tests are performed
to help determine the cause of abnormal results. Often, this requires visual confirmation by examining a blood smear under a microscope. A trained laboratorian can evaluate the appearance and physical characteristics of the blood cells, such as size, shape and color, noting any abnormalities that may be present. Any additional information is noted and reported to the healthcare practitioner. This information gives the health practitioner additional clues as to the cause of abnormal CBC results. When you have a CBC performed, you can talk to your healthcare
provider about the meaning of results, whether additional tests are necessary, and why. Source: AACC Lab Tests Online, republished from Lab Tests Online.
For answers to more questions like What is being tested? How to prepare for a CBC? or What do the CBC results mean? as well as information on 300+ other tests visit: (US and Canada) labtestsonline.org/tests/cbc or (UK) labtestsonline.org/tests/fbc
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How laboratories can benefit from off-the-shelf flow cytometry testing How off-the-shelf antibody testing has revolutionized flow cytometry testing in the hematology laboratory.
O
Dr Véronique Saada PharmSCiD, Specialist in Hematology, Institut Gustave Roussy, Paris, France
Dr Benjamin Hedley Senior Research Fellow, London Health Sciences Centre, Ontario, Canada
Mike Keeney Associate Scientist, at the Lawson Health Research Institute, London Health Sciences Centre, Ontario, Canada SPONSORED
ff-the-shelf flow cytometry testing can enable smaller hematology laboratories to maximize their workforce capacity, save time and money, and achieve faster, more detailed and more accurate immunophenotyping for their clients, according to international experts in this increasingly important field of hematological study. Flow cytometry is a powerful tool used in immunophenotyping for hematological malignancies, such as chronic lymphocytic leukemia (CLL), as well as in basic research and in clinical trials. Flow cytometry is held to have several advantages over immunohistochemistry (IHC) in cancer including: the ability to define distinct cell populations by their size and granularity; the capacity to gate out dead cells detection of weakly expressed surface antigens; and multi-colour analysis to measure several antigens simultaneously. At the heart of the test is a panel of antibodies that detect markers or antigens on the cells, aiding laboratorians in the identification of cell lineage. Although widely used, to date, flow cytometry testing has not fulfilled its potential to provide laboratories with maximum return on the high capital cost associated with the purchase of a flow cytometer: methodology and data interpretation, clinical data sharing and education have all been hindered by the complexity and variability of test set-up and results analysis. For manually prepared and designed panels, each of the required number of antibodies must be titrated and validated, and then revalidated when combined with other antibodies – a process which itself may require further technical adjustment in order to achieve accurate results. Quality assurance of the reagent cocktail demands rigorous documentation (dating, etc) at each step, including regular, ongoing testing to assess the quality of each
batch. Lot variations, as well as stability issues, converge to add to the administrative burden. Manual antibody cocktail preparation is a process that is inherently fraught with the potential for errors. Dr Véronique Saada is a specialist practitioner in hematology at the leading cancer research centre, the Institut Gustave Roussy in Paris. She says: “There can be missing markers, and the wrong antibodies can be added,” raising the spectre of potential misinterpretation of the patient sample and an incorrect clinical conclusion that can have serious consequences for the patient. In words that will resonate with lab technicians throughout the world, Dr Saada says: “Cocktail preparation is a complex, time consuming process involving individual pipetting of antibodies into the batch.” For the laboratory, the complexity of test preparation has important commercial ramifications. In some labs, technicians are employed solely to prepare flow cytometry analysis panels, says Mike Keeney, associate scientist at the Lawson Health Research Institute, London Health Sciences Centre, Ontario, Canada. And, with antibodies costing up to $1,000 each, errors and waste can be costly.
Technological development to detect cancers Since June 2017, labs have benefited from US Food and Drug Administration authorized technology, designed to detect a range of leukemias and lymphomas, including chronic leukemia, acute leukemia, non-Hodgkin lymphoma, myeloma, myelodysplastic syndrome (MDS) and myeloproliferative neoplasms (MPN). Announcing the market approval of this first-in-type test, Alberto Gutierrez, director of the FDA’s office of in-vitro diagnostics and radiological health, said: “Laboratories and healthcare professionals now have access to an FDA-validated test that provides consistent results to aid in the diagnoses of these
serious cancers. This represents a major step forward for the hematology-oncology community.” The new technology, Beckman Coulter ClearLLab Reagents, have been authorized through the de novo premarket review pathway, a regulatory pathway for novel, low- to moderate-risk devices that are not substantially equivalent to an already marketed device. The authorization was supported by a study designed to demonstrate the test’s performance, which was conducted on 279 samples at four independent clinical sites. The study compared the test’s results to alternative detection methods used by the clinical sites. The study showed that the results of the test aligned with the study site’s final diagnosis 93.4% of the time and correctly detected that there was a cancer presence (i.e., cancer abnormality) 84.2% of the time.
Aligned with 2006 Bethesda International Consensus recommendations A key factor in the success of the test on the global stage is its alignment with the world-famous 2006 Bethesda International Consensus recommendations on the immunophenotypic analysis of hematolymphoid neoplasia by flow cytometry. These recommendations set out the reagents and reporting for the flow cytometric diagnosis of hematopoietic neoplasia - and for hematology laboratories the world over represents a successful attempt to define a set of consensus reagents suitable for the initial evaluation of hematopoietic neoplasia. The Bethesda committee included laboratory professionals from private, public, and university hospitals as well as large reference laboratories that routinely operate clinical flow cytometry laboratories with an emphasis on lymphoma and leukemia immunophenotyping. A survey of participants successfully identified the cell lineage(s) to be evaluated for each of a variety of specific medical indications and
defined a set of consensus reagents suitable for the initial evaluation of each cell lineage.
Lower costs, less time and greater patient and professional satisfaction For labs around the world, the availability of FDA and Bethesda International Consensus recommendations compliant panels, containing premixed and optimized multi colour antibodies assay, has important implications. The Lawson Health Research Institute’s Mike Keeney estimates there can be 12-15% time and cost savings for an average hematological lab. Savings from reduced manual input, reduced waste and errors, and inventory savings, can run into thousands of dollars. An additional benefit is improved work flow and a higher quality of the work experience for technicians. Senior research fellow, Dr Benjamin Hedley, also of the London Health Sciences Centre in Ontario, says: “We can move our highly qualified technicians away from essential but tedious manual tasks into more professionally satisfying data analysis.” For patients, quicker and more accurate laboratory testing has obvious benefits for health. Less directly, the advent of standardized technology and procedure across the global stage has important ramifications for the advancement of clinical education and research: research can now be shared across laboratories in confidence that results are not confounded by variables in the assembly and interpretation process, and that they conform with international guidelines. In short, says Mike Keeney, “this technology is a significant step forward on the path to standardization.” To download the free ClearLLab e-Casebook and receive copies of the data files please visit info.beckmancoulter. com/casebook-usa
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A diagnosis of chronic myeloid leukaemia is no longer fatal Twenty years ago, someone diagnosed with chronic myeloid leukaemia could expect to live just a few years. Now, thanks to huge treatment innovations, many are completely cured.
Each year, around 750 individuals in the UK are diagnosed with chronic myeloid leukaemia (CML), a cancer that causes the bone marrow to produce too many white cells, called granulocytes. Twenty years ago, life expectancy for someone diagnosed with CML was just six years but treatment developed over the last two decades
Professor Jane Apperley Chair of the Department of Haematology, Imperial College London
one thing
has changed all that. 2018 marks 20 years since the first group of CML patients began clinical trials for new drugs, called tyrosine kinase inhibitors (TKIs). “The leukaemia is characterised by a single chromosomal abnormality, known as the ‘Philadelphia chromosome’. This single abnormality creates a new gene that is present in the leukaemic cells but not in any other cells in the body,” explains Professor Jane Apperley, Chair of the Centre for Haematology at Imperial College. This new gene makes a new protein that is different to any other in the body, so researchers developed TKIs to target those specific
proteins and stop the cells from growing and dividing.
Patients fight for drug approval The efficacy of the TKIs was immediately apparent. From 1998 onwards, drug companies have continued to develop even more effective treatments. “A recent study from Scandinavia shows that survival rates for people with CML are now well over 90% and many have the same life expectancy as they would have had before they were diagnosed with the condition,” says Apperley. There are now a whole range of
TKI drugs that are taken as a daily tablet. And, because they are focused on tackling a specific gene, the damage that you get with other forms of chemotherapy is not so widespread. “The patient and medical communities worked together and fought to get all the drugs approved by NICE and to be able to prescribe them in a logical fashion, so now, if you fail to respond to one, you can move to the next,” says Apperley.
Treatment-free remission Because CML is a single gene disorder, doctors can measure the amount of disease that’s left in the patient very precisely. A few years ago, a number
my doctor can do… + give me peace of
faster CML monito
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What is the true cost of innovation in cancer research?
Professor Stephen G O’Brien Professor of Haematology, Newcastle University and Chair, NICE Technology Appraisal Committee C The huge advances in treatment for patients with chronic myeloid leukemia has transformed patients’ lives, but the challenge is balancing innovation and affordability.
of brave patients who clearly showed a very positive response to the treatment came off the drugs altogether, with very promising results. “For people with very deep sustained responses it looks like about 40% could stay off the drugs and may have been cured simply by the tablets, which is phenomenal. We call this treatment-free remission,” says Apperley.
Unexpectedly high success rates While the current drugs are hugely effective, there are a number of chronic side effects that mean many patients want to come off them altogether. Three years ago, Professor Richard
mind with oring results*
Clark (University of Liverpool) led a study to see what would happen if patients were first given half the dosage for one year and then stopped completely. Yet, again, the results were unprecedented. “Our results for the proportion of patients who can stop was much higher – at 70% – for reasons we don’t entirely understand,” says Apperley. The findings will be presented at the European Haematology Association congress in June and could spark the next wave of research into finding a cure. Patients who are effectively able to stop treatment have to achieve such deep remissions that there are virtually no traces of the cancer in their blood
cells. They will need to undergo regular blood tests to check there is no relapse, but the importance of this development cannot be understated. Stopping treatment will save the NHS hundreds of thousands of pounds each year and, most importantly, it will give patients their life back. The success seen by CML patients has sparked a whole range of research into cancer treatments and, while there is always more to be done, it’s clear to see why many are hailing CML drugs as the poster child for cancer treatment. Kate Sharma Read more on healthawareness.co.uk
“Improvements in the treatment of chronic myeloid leukaemia (CML) is one of the greatest success stories in cancer treatment,” says Professor Stephen O’Brien, Consultant Haematologist at the Newcastle Hospitals NHS Foundation Trust. Just 20 years ago, patients diagnosed with the condition could expect to live for around five years. Today, some are completely cured and others can live a normal life on medication. Not only are survival rates impressive, but the fact that the drug can be taken in tablet form at home has revolutionised the way that cancer patients are treated. “Follow-up calls can be taken at home, patients don’t have to take time out of their lives to come to an institutional setting and we even have one patient, who’s a farmer, who receives his delivery of medication when he’s out on his tractor,” says O’Brien. Of course, the benefits all come at a cost and, when they first arrived on the market, treatments for CML cost in excess of £20,000 per patient per year. Considering the majority of patients are on the tablets for the rest of their lives, the cost was significant. However, the patent expired in 2017, which means the drugs are now available for hundreds of pounds a year – a fraction of the cost. This is clearly a positive step forward, but the tension between innovation and affordability remains ever-present. “As a clinician and scientist, this is a really exciting time,” says O’Brien. “But as a citizen and Chair of a NICE Technology Appraisal Committee, I’m acutely aware of the costs. We have to be stringent in evaluating new technology and drugs to ensure the best outcomes for patients and ensure affordability for the NHS.” Kate Sharma
Learn more by visiting www.cepheid.com/bcrabl-ultra * For monitoring of Chronic Myeloid Leukaemia patients with BCR-ABL major breakpoint (p210 transcripts) In Vitro Diagnostic Medical Device
Not all tests available in all countries. Not available in the U.S.
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Industry leads research into new CAR-T therapies for myeloma care
Around
240,000 people are living with a blood cancer or related disorder in the UK
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There are over
100 different types of blood cancer
Over
15,000 people die from a blood cancer or related disorder each year in the UK
SOURCE: BLOODWISE
Someone is diagnosed with blood cancer every 14 seconds
Dr Martin Pule Clinical Senior Lecturer Honorary Consultant in Haematology, Cancer Institute, University College London
Industry partnerships are fuelling the way for new CAR-T therapies for myeloma care.
Traditional treatments for cancer – chemotherapy or bone marrow transplantation in patients with myeloma (blood cancer arising from plasma cells) are horrible, invasive procedures, says Dr Martin Pule, who is the Clinical Senior Lecturer in the Department of Haematology at UCL Cancer Institute and Honorary Consultant in Haematology at University College London Hospital. “Because of the toxicity they come with a significant chance of long- term problems, even death. If we can replace that with another effective therapy, then that is a big deal for patients – and their families.” For years, the foundations of cancer treatment have been surgery, chemotherapy, and radiation therapy. Over the last two decades, drugs that target specific cancer cells, for example, in breast cancer, have also forged a role as standard treatments. However, more recently, immunotherapy – therapies that enlist and strengthen the power of a patient’s immune system to attack tumours – has emerged. By far, the most exciting of these in terms of clinical development is CAR-T cell therapy. Due to the inherent advantages for researchers of blood over solid cancers, for example access to samples, blood cancers lend themselves more easily to pioneering research. Among the most promising areas for clinicians today is the use of CAR-T therapy in multiple myeloma. Thanks to the backing of some big players within the pharmaceutical industry, it is possible there will now be rapid commercial development of products within this therapy area. Once these become available, they give clinicians an important new treatment for myeloma. Dr Pule says: “Just five or six years ago, CAR-T therapy was only an academic discipline, but once the commercial pharma companies took an interest, this made people think this new technology could really take off.” Encouraged by the evidence of survival and remission rates of between 40-60%, UK medicines watchdog, the National Institute of Health and Care (NICE), has also expressed support for these therapies; it describes CAR-T as having “potentially very substantial patient benefits”. Approval of CAR-T therapies for use in the NHS could see this therapy in wide-spread use in the NHS within a few months. “In resistant disease, the result being generated by the clinical trials are amazing,” says Dr Pule. “This is the most important advance in the treatment of haematological cancer in a generation… their ability to induce long-term remission is remarkable.” Ailsa Colquhoun
Blood cancer is the third biggest cancer killer, and yet awareness of the condition remains dangerously low.
Blood cancer claims more lives than prostate cancer or breast cancer each year, yet awareness levels are worrying low. Part of the problem is that blood cancers are often referred to by their individual names, rather than as a collective group, so people just don’t realise quite how prevalent they are. Categorising conditions in this way means that the public, patients, health services and the government are failing to recognise the devastating, cumulative impact of blood cancer. “The government and other organisations don’t recognise blood cancers together, which means the weight put behind resourcing it is lower,” says Gemma Peters, CEO of Bloodwise.
Blood cancer behaves differently from other cancers Blood cancers don’t behave like other tumours and so the treatment pathway is very different. Rather than being referred to oncology units, blood cancer patients often find themselves in haematology departments where they may see doctors who are specialists in a range of blood conditions, but not necessarily blood cancer. “There is a huge variation in the quality and consistency of care provided to
medication immediately. Instead, they have to ‘watch and wait’ to see how their condition progresses.
Improved diagnosis and treatment pathways
Gemma Peters CEO, Bloodwise
“We need to keep blood cancer in the forefront of people’s minds.” blood cancer patients. They are also much less likely to have access to a clinical nurse specialist than some other cancers,” says Peters. “The government’s Cancer Strategy talks a lot about living beyond cancer, but it doesn’t talk as much about living with cancer, which is the reality for many blood cancer patients who will have to take medication for the rest of their lives.” For some patients who have a very slow onset of cancer, the side effects of treatment are considered much worse than living with the cancer itself, so they are not put on
The physical and psychological impact this has on a patient should not be underestimated, and was one of the points highlighted in a report released by the All Party Parliamentary Group (APPG) on Blood Cancer earlier this year. The report made a number of recommendations calling on government, the NHS and individual trusts and health commissioners to improve the care and treatment of people with blood cancer. At the heart of the report is a renewed effort to raise awareness of blood cancer and to provide better joined up working between primary and secondary health services, and between oncology and haematology teams. The report recommends that this encompasses not just the medical care but also psychological support, particularly for those who fall into the ‘watch and wait’ category or ‘watch and worry’ as many patients refer to it. While there is much more that can be done to improve care for blood cancer patients, Peters is keen to point out that there are strong foundations to build on. “We are optimistic about the future, but need renewed pressure to keep blood cancer in the forefront of people’s minds,” concludes Peters. Kate Sharma
Every 13 minutes someone is diagnosed with a blood cancer or related disorder in the UK SOURCE : BLOODWISE
How is CAR-T therapy given? CAR-T therapy is a completely new type of treatment that modifies a patient’s T cells (a type of white blood cell) to boost their ability to recognise and kill specific cancer cells. If CAR-T therapy is a treatment option for someone, they will discuss this with their doctor and have tests to see if CAR-T could work for them.
1
2
T cells are taken from the patient’s blood
T cells are turned into CAR-T cells
During a visit to hospital, blood is taken from the patient. The T cells (a type of white blood cell) are removed. The remaining blood is given back to the patient.
In the laboratory, the T cells are genetically modified so they can recognise and kill specific cancer cells. They are now called CAR-T cells. The CAR-T cells are multiplied to make enough for the treatment.
3
4
CAR-T cells are given to the patient
CAR-T cells attack cancer cells
The patient has chemotherapy, which reduces the number of normal T cells in the blood. This makes room for the CAR-T cells. After chemotherapy, the CAR-T cells are put back into the patient’s blood through a drip.
The CAR-T cells continue to multiply in the bloodstream, finding and killing cancer cells. CAR-T cells may remain in the bloodstream for some time to help prevent cancer from coming back.
CAR-T therapy is designed to be a one-off treatment. The patient is closely monitored with regular tests and check-ups for the next 30 days. If they experience side effects, they may need to go back to hospital. The doctor continues to monitor the patient for around 2–3 months, to check their response, overall condition, and watch for side effects. If you or anyone you know has been affected by blood cancer, you can get in touch with our support line on (freephone) 0808 2080 888 or email support@bloodwise.org.uk
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Get in touch for the next edition. + 44 (0) 203 642 07 37 georgia.gerstein@mediaplanet.com @mediaplanetUK