Monday, 25 January 2016

Breakthrough for Pulmonary Hypertension Treatment

Last year there was a significant breakthrough in the genetic research into Pulmonary Arterial Hypertension (PAH) being led by Professor Morrell at the Cambridge Biomedical Research Centre. Professor Morrell is both British Heart Foundation Professor of Cardiopulmonary Medicine and Research Director, National Pulmonary Hypertension Service at Papworth Hospital.

A protein was discovered that targets the effects of and reverses the damage caused by a faulty gene BMPR-11 - this faulty gene is the main identified gene for causing inherited Pulmonary Arterial Hypertension. I have put the links for more detailed information on the research project and on the breakthrough discovered last year below.

The Genetic Research Project

BHF Press Release 2015

This week it was announced that Morphogen-IX, a new Cambridge based biotech, has gained seed funding to develop a new treatment for PAH, following on from the findings last year. This drug will directly target the disease and has the potential to provide the first disease modifying approach to the serious life limiting effects of PAH. The current therapies for PH and PAH help to alleviate symptoms, but as yet there are no medical options to modify the course or outcome of the disease. The company will be solely dedicated to developing a therapy that will tackle the disease rather than just manage its symptoms and they have received grants of 1.5 million to develop the drug. The link below gives more information on Morphogen-IX


This is marvellous news for the Pulmonary Hypertension community - we always live in hope that a breakthrough will happen and that eventually a cure will be found. When a person is diagnosed with PH it is both shocking and life changing. Whatever stage of PH you are in, you know that you face a long journey of hospitals, doctors, tests and treatments. The treatments for PH vary depending on the type of PH and the stage of severity that the PH is in - they vary from oral medications to complex inhaled medications and intravenous medications, which are very daunting to face. There are some surgical alternatives for some patients such as Pulmonary Endarterectomy, Lung Transplantation or Heart and Lung transplantation - high risk surgeries that again can feel terrifying to face. For some there are no surgical alternatives, just therapies that manage the symptoms, although the disease progresses. There is also the knowledge that PH can be a life limiting disease and that you face a future that is uncertain and with some disability.

To know that there is research and development of a drug which may reverse the effects of this devastating disease is wonderful news. These new developments bring so much hope to so many. Hope that they may not have the future they are presently having to face - one of complex drugs, surgeries and one that is potentially life limiting - and hope for a future that could change for the better.

The announcements that this new drug is going to be developed demonstrates great progress has been made with the genetic research. The new drug will then need to be put through the various clinical trials. We wait in hope for more announcements when developments reach this stage. In the meantime there are other drug trials in progress, where it is hoped that symptoms in patients may be helped and controlled better and again, even modified. One is a trial for Tocilizumab, which is currently used for arthritis and it is hoped it may modify PAH too. Again more hope. A link for more details on this is below.

New Clinical Drug Trial

On a personal note, although I don't have PAH any more after undergoing a heart and double lung transplant as an end stage treatment, I'm delighted there are these new developments to help PH sufferers and that there is some hope for the future. I have a keen interest in the genetics research as I underwent genetics testing and took part in this genetics research at the very beginning of the project. I still take part in the research and last year gave more DNA as the project moves on forward and I was given information about the possibility of second generation testing, so it is still of great interest to me. It was an honour to take part in the publicity process about the genetic research and the breakthroughs that are happening over this last week.

My story and involvement with genetic

This week I had the opportunity to take part in an interview with BBC Look East and on a fun note the pictures show my new puppy, Ted making his TV debut in his first ambassador role for PH! Visit my author Facebook page and scroll down to find the whole interview with BBC Look East, Professor Morrell, the research team, Ted and me.

Life is for the Living Facebook page

The 1.5 million funding raised for the new drug will now build on the work of the project funded by donations from the British Heart Foundation. The funding from BHF is vital in developing this research. Their link is below.

British Heart Foundation

We live in hope!

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