Research news

Promising results for new PAH drug

A new medication, that could be approved for use next year, has produced promising results in its latest clinical trial.

Using six-minute walk tests, the STELLAR trial showed that when sotatercept is given in addition to other therapies, it significantly improves exercise capacity in people with pulmonary arterial hypertension.

The drug, which is given under the skin as a subcutaneous injection, is the first of its kind to rebalance what’s known as the ‘bone morphogenic protein pathway’. While existing treatments are designed to help manage the symptoms of PH, sotatercept is the first to target the root cause of the disease.

Neil Hamilton, Consultant Pharmacist in PH, told us: “Sotatercept is an exciting new treatment which works in a different way to any of the current medicines we can prescribe. We are optimistic about sotatercept because the results from early clinical trials have been very positive.

More evidence of effectiveness and safety is needed, so further studies are underway. These international trials will involve large numbers of patients and if these prove successful, sotatercept could be approved by regulatory bodies next year. As such, it is not currently available to anyone outside clinical trials. Sotatercept may not be suitable for everyone with PH, but expert centres in the UK are recruiting patients to these trials – so if you are eligible, your centre would discuss your participation at your next clinic visit.”

Online reaction to the results of the STELLAR trial:

"This is a big deal in pulmonary hypertension” Dr Mark Toshner (@mark_toshner) “This might be the most exciting advancement in pulmonary arterial hypertension since I started practice” Dr Jason Weatherald (@AlbertaPHdoc)

Blood sample research may help identify the best treatment for CTEPH

A study by scientists at Royal Papworth Hospital could lead to doctors being able to identify which patients with chronic thromboembolic pulmonary hypertension (CTEPH) would be best suited to pulmonary endarterectomy surgery.

The surgery, knows as a PEA, removes scars made by old blood clots in the pulmonary arteries and it is potentially curative. However, some may be left with residual PH, and as a major and complex operation, it is only suitable for approximately 60-70% of patients.

Researchers at Royal Papworth Hospital have studied blood samples from patients before and after this surgery, to identify changes in chemicals contained within the blood.

The study, titled Plasma metabolomics exhibit response to therapy in chronic thromboembolic pulmonary hypertension, has shown differences between healthy individuals and patients with CTEPH, including changes correlating with the severity of the disease.

A small number of the chemicals were also found to be different from those in patients with idiopathic pulmonary arterial hypertension (IPAH), which may lead to a new a way of identifying the type of PH a person has.

Ultimately, those behind the study believe that chemicals that are either produced, used or changed by the heart and lungs may eventually be used to identify which patients are most suitable for PEA surgery to treat CTEPH. The researchers would like to thank all the patients who took part in this important study. .

Have you ever heard the saying ‘food is medicine’? Here at the PHA UK we have long been advocates of a healthy lifestyle alongside medical treatment – and what we eat is a significant part of the puzzle.

A mission for good nutrition!

Nutrition issomething we can all control, even when there so many other things we can’t, and as a charity we will be focusing more on this over the next few months.

This important work will be led by Shaun Clayton (pictured), Director of Membership Support at the PHA UK. As well as ten years of experience supporting the PH community, Shaun is a certified nutritionist – and passionate about the power of food. Here, he explains more…

“It’s so important to look at health holistically, and what we eat is an integral part of ‘feeling good’ – both physically and mentally.

This work will all be focused on helping people live the best lives they can by optimising what they eat, whatever their specific goals may be. Priorities could be weight loss, weight gain, maximising energy, or simply making the best nutritional choices.

My aim is to empower members to make the best choices for them and their nutrition, with tips on how they can eat in a way that fits with their lives as well as their goals.

At the PHA UK we understand that even when you know what you ‘should’ be doing, it isn’t always easy. Challenges can come in the way of time, physical limitations, costs, and of course, willpower and emotional obstructions. That’s why our work will focus on these aspects too.

We have consulted with a focus group of members around priorities in nutrition, and guided by this feedback, we will be producing a number of resources throughout 2023.

The PHA UK will be leading this work whilst collaborating with the network of specialist PH centres, and I’m really looking forward to supporting the PH community in this way.”

Find out more over the page

A mission for good nutrition!

Supporting our ‘mission for good nutrition’ will be Chermaine Kwant, a nutritionist with personal experience of PH. Chermaine was diagnosed with idiopathic pulmonary arterial hypertension in 2011, and she has spent the last ten years learning more about how good nutrition can improve quality of life in illness. She is now a researcher at Amsterdam University Medical Centre and is completing a PhD in the subject of nutrition and pulmonary hypertension.

A cup of tea with Chermaine…

Has food and nutrition always been of interest to you?

“When I was 12 years old, I really gained interest in cooking. I decided I wanted to become a cook. When the brother of my former partner became very sick, he got help from a dietician. I’d never heard of this profession, and it sparked my interest.

I decided I wanted to become a researcher and study a rare disease and help these patients with their nutrition. What I never could have imagined was that I first had to get a rare disease myself, before I would even get the opportunity to become a researcher.”

Can you tell us about your personal journey with PH and how that led to researching nutrition in a professional capacity?

“In the summer of 2011, I was feeling very sick during my vacation, and I thought I had pneumonia. When I returned, I kept feeling really tired and was short of breath.

On Christmas Eve of that same year, I had a mild stroke and whilst in hospital the doctors noticed that there was something really wrong with my heart. After a few tests they told me that I suffered from idiopathic PAH and had approximately one year left to live.

However, I responded very well to treatment, and couldn’t believe I was that sick. But after the first year, things worsened. Although I travelled, worked, and lived my life to the fullest, I needed more and more medication and I started trying different diets to make myself feel better.

They did make me feel better, but the PAH still progressed, and a lung transplant was inevitable. This took place five years after my diagnosis.

“It’s not about ‘curing’ the disease. But every opportunity to make yourself feel better is a win.”

A year after the surgery, I visited the hospital in Amsterdam where I was treated for my PH and spoke to my former doctor. He asked about my thoughts on nutrition and PH and while I spoke, he started to draw a timeline on a whiteboard. ‘And there is your thesis!’ he said.

He explained he was asking me to start a PhD. It felt like a fairytale after being hospitalised for so long, and the long recovery after my transplant.”

Can you give us an overview of your research in the area of nutrition, and what the findings have been so far?

“I study how nutrition and lifestyle interventions in pulmonary arterial hypertension affect quality of life.

First, my research involved studying what patients actually eat and if there were any deficiencies of vitamins and minerals in their blood. The most important finding of this part of the study was that sugar plays a bigger role when it comes to fluid retention than salt – which is different to what is commonly advised in terms of cardiovascular disease.

In the second part of my study, we tested an e-learning tool and two types of diets.

The main focus of the e-learning was to give insight on nutrition in relation to quality of life. The patients were coached in setting their own goals in making nutritional changes. This part of the intervention was very effective and the patients that participated best, and made most changes during the e-learning, had the best improvement in their quality of life.

We are very excited about these outcomes, and we want to study the effect of this e-learning in a worldwide cohort. There is a lot of interest in nutrition, so hopefully this will help us the raise money for what I believe is a very important study.”

Why is nutrition so important when it comes to managing chronic health conditions like PH?

“I think nutrition is the base of every form of life. Every cell in your body can function, whether this is good or maybe not so good, because of the way you feed it. You can compare yourself with a car. When you need petrol for fuel, and you use diesel instead, you know that you won’t be able to drive as far (or maybe at all).

I also think nutrition is a relatively easy way to empower yourself. You eat and drink every day, so why not take control over your disease and try to make yourself feel better?

It’s not about ‘curing’ the disease. But every opportunity to make yourself feel better is a win.

I’m not saying this as a researcher or as a nutritionist, but as a patient. Because I know from that point of view that nutrition can make a difference. It can make the difference between feeling completely tired, feeling okay, or maybe even feeling really good. And that is a huge difference when it comes to PH.”

How do you feel about being able to help educate people with PH in the UK about the importance of nutrition?

“I’m really excited about this next step and can’t wait to meet the PH teams and some patients. Hopefully I can help and inspire them to make different choices when it comes to nutrition, that will improve their quality of life.”. Take 5

In future issues of Emphasis, we will bring you detailed guidance on specific aspects of nutrition and PH. If you’re keen to make changes now, these are Shaun’s top five tips for eating well with PH:

11 With PH you can have good days and bad ones and often cooking can be the last thing on our minds during those bad days. Something like batch cooking on the good days can really help. It sounds simple but knowing that you have a hearty stew sat in the freezer ready to go may just make the day that bit easier and gives you one less thing to think about.

2We often hear about how we should cook using ‘organic’ produce or how ‘fresh’ vegetables are best. Whilst there is a small amount of truth in this, the evidence shows that frozen vegetables are just as good. They still contain all the vitamins and minerals to aid a healthy diet but at a fraction of the cost of their organic counterparts. 3 3 Certain medications can cause nasty side effects which makes it hard to gain weight. Simple changes like adding more olive oil to your cooking can add extra calories without having to give it too much thought. These extra calories could help you gain weight healthily quite quickly. 4 ‘What if’ statements are something I’ll talk about more in depth in the future. But for now, it’s always handy to ‘plan’ for things. So, for example “what if I’m going to be an inpatient for a few days, is there anything I can pack to take with me that match my nutritional goals?”. 5 Finally, don’t underestimate the power of small changes. Don’t put undue pressure on yourself to get your diet perfect. Good in this case is great! And making a few small changes can make a huge difference.