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3 November 2017

Research trial could revolutionise the way patients take their drugs

A team working in Papworth's Research and Development department have begun a trial which could transform the way medicine is delivered to patients with a rare disease.

In a world first, the Pulmonary Vascular Disease Unit (PVDU) has recruited the first three Pulmonary Hypertension (PH) patients to the Novartis study, which is testing a new drug which can be delivered through an inhaler rather than a continuous IV line.

Pulmonary Hypertension raises blood pressure in the lungs which leads to heart failure and early death, with current treatments aiming to relax the vessels in the lung and lower the blood pressures. Patients not only have a poor prognosis but their quality of life is severely affected.

Clinical Research Manager Louise Harlow, who is part of the research team headed by Consultant Physician Dr Joanna Pepke-Zaba, said: “At the moment, comparative drugs are delivered through an IV line, which is in place 24 hours a day. It’s very invasive and there are some nasty side effects, the line can also become infected.

“The patients we are working with are getting to the point in their illness when they need to be thinking about having the line inserted, and they don’t want it because it’s so uncomfortable; some of them have children and work full-time and it would make life very difficult - when they are first put on the drug they can be in hospital for up to two weeks getting the levels right. Others just don’t like the idea of the side effects.

“This new treatment is the same class of drug but it’s delivered through an inhaler, and you only have to take it twice a day - it could be revolutionary for our patients.”

Meanwhile, the team is also involved with the first investigator led clinical trial - involving all UK specialist PH centres - of a drug which targets inflammation and immune problems in PH patients.

The Transform-UK research programme, which is sponsored by Papworth Hospital with joint funding from global healthcare company ROCHE and NIHR, aims to see if treatment with Tocilizumab (RoActmera®) - a drug which binds to proteins in order to decrease inflammation and auto-immunity - reduces the blood pressure in the lungs.

The team, led by Dr Mark Toshner has had an article published in Pulmonary Circulation, entitled ‘Clinical Trial protocol for TRANSFORM-UK: A therapeutic open label study of Tocilizumab in the treatment of pulmonary arterial hypertension’. Read the article here, and visit www.transform-uk.com.

 

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