NIHR: New treatment approach for Pulmonary Arterial Hypertension being tested

A new NIHR clinical research study is examining whether tocilizumab, a biological drug already available for treatment of rheumatoid arthritis, could be used to treat the rare but debilitating condition pulmonary arterial hypertension (PAH). Although some treatments are available for PAH, they are only effective at relieving symptoms of the disease. Tocilizumab has the potential to become the first disease modifying drug for PAH.

The TRANSFORM-UK study is a partnership between the NIHR Rare Diseases Translational Research Collaboration and the global pharmaceutical company Roche. The NIHR established the Rare Diseases Translational Research Collaboration in 2013, supported by a £20M investment, to bring together the country's leading experts in the clinical research of rare diseases.

PAH is a progressive disease caused by narrowing or tightening of the pulmonary arteries, which connect the heart to the lungs. This results in the right side of the heart becoming enlarged due to the increased strain of pumping blood through the lungs. Strain on the heart and decrease in blood circulation through the lungs lead to the common symptoms of PAH including breathlessness, fatigue, weakness and angina. Fewer than 40% of patients currently live beyond five years after diagnosis and there is no cure.

Life Sciences Minister George Freeman MP said: ‘We are determined to give NHS patients the fastest access to the most innovative, effective medicines, which is why we invest over £1 billion each year in the National Institute for Health Research.

Through the NIHR’s £20 million Rare Diseases research collaboration we are bringing together the country’s leading health researchers in hospitals, Universities and companies like Roche to help them get access to world-leading research infrastructure in the NHS and accelerate the access for NHS patients to new treatments.’

The NIHR is contributing project funding and providing expert researchers for the study, and Roche are providing the drug tocilizumab, as well as supporting a proportion of the trial costs. The NIHR Office for Clinical Research Infrastructure (NOCRI) worked closely with Roche and the NIHR Rare Diseases Translational Research Collaboration to set up the partnership.

Dr Madhi Farhan, Roche’s Head of the Office of I2O Innovation commented: 'Working with NOCRI and the NIHR has been invaluable to ensure rapid connections and fruitful partnership with the UK's leading experts in translational research of PAH. Being able to look at the in-depth science of how one of our current treatments could be applied to a real unmet medical need is what attracted us to carry out this work in the UK. We hope this research will soon lead to benefit for patients with this debilitating disease.'

Mark Samuels from the NIHR said: ‘The NIHR has invested significantly in the research of rare and neglected diseases, and its Rare Diseases Translational Research Collaboration is instrumental in translating cutting edge science into immediate benefits for patients. This innovative and exciting trial of an available biological drug is further evidence for the success of the NIHR in collaborating with industry on the development of groundbreaking therapies. I am delighted to see the important role NOCRI played in building and supporting this collaboration with Roche.’

The study is led by Dr Mark Toshner from the Papworth Hospital NHS Foundation Trust and is hosted by Cambridge University Hospitals NHS Trust.

ENDS

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