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


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!

New Life, New Opportunities, New Year!

The last year was the most amazing year when I started a new life with many new experience and opportunities. It started with Sarah and Oli's engagement and a flurry of searching for wedding venues in January. January also saw me eventually coming off all the inhaler medications I had been on since my transplant - three of them three times a day - and coming off heart failure drugs at long last now my new heart had become stable after my transplant. Late January was the last time I had to have a bronchoscopy after having to have them nearly every week for months. Without all that came new freedom and we visited London to meet friends. We were pinching ourselves and I couldn't believe how much better I felt, how much I could suddenly manage and how far I could walk.

The year continued well and I'd thought I couldn't get much better, but I just kept on getting better and better. Rob and I did quite a few national and local media interviews over the course of the year to help raise the profile of transplant, organ donation and pulmonary hypertension including ones for Hertfordshire Life, Heart Matters (BHF magazine) and The Weekly News. We featured several times throughout the year in The Hertfordshire Mercury, The local Comet newspaper and on local radio Bob FM Home Counties. We also worked with NHSBT during National transplant Week and for their Christmas campaign and were featured on BBC Three Counties Radio, where we were given the opportunity to visit the radio studios in Luton. Speaking about organ donation and pulmonary hypertension is our way of trying to give something back for all the good fortune we have been given.

We seemed to get one wonderful opportunity after another and they just kept on coming. I enrolled on a writing course at the beginning of the year, so I could start writing my own articles for publication and one of my articles on my transplant was published in Cumbria magazine. I won a competition run by Woman and Home Magazine for writing a small article on 'Seizing the Day' and had a full style, makeover, photoshoot and interview published in the September issue of their magazine, plus a thousand pounds prize money for Papworth Hospital Charity. In October I was invited to speak at one of their reader events about my transplant and making the most of life. They were all interesting and new experiences to be part of and at the same time raise more awareness of organ donation and pulmonary hypertension to new audiences.

In July we were asked to take part in a launch of the government's 100,000 Genomes Project and were featured on all the main TV channels as well as getting to visit the Sanger Institute in Cambridge to see how the genetic testing machines work. We also had the wonderful and exciting opportunity to visit Downing St when the project was officially launched and this led to more local press interviews. At Downing St, I was pinching myself because I was able to walk at a fast pace up those famous stairs. It never ceases to amaze me what having my transplant has given me.

I had many small personal goals and dreams to work towards as I recovered including going for proper long walks once more. I set myself a challenge to walk around Tarn Hows in the Lake District in summer, but managed it in March. I wanted to get back in my kayak once more and managed this in June when I kayaked across Coniston Water once again, much to my delight. I travelled abroad once more, after nearly five years and went to Paris by Eurostar. After falling ill I had been deemed too unfit to fly and was unable to anyway being on the transplant list. I made my first flight abroad in October, just a short one to Amsterdam and this gave me confidence to make another dream come true and make a long haul flight to New York in December. It was wonderful to visit some new places.

To a lot of people these are just normal and everyday occurrences, but for me they are massive steps as they are all things I thought I would never be able to do ever again. Achieving these things have been a bonus and the icing on the cake, but the thing that still really amazes me is the fact that I can walk around normally again without using a wheelchair or measuring how far I have walked so I can make sure I can manage the walk back again. It is still special to bath and shower normally without worrying about medical equipment getting wet and it is amazing to run up steps. I run up stairs every day now just because I can! These are the things that healthy people take for granted, having had these taken away from me over the years and then suddenly getting them back is something that still astounds me even a year on.

Standing at the top of the Empire State Building at night time watching the lights of New York twinkling and fireworks going off over the Hudson River was a spectacular moment and more than a dream come true at the end of this year, but it still dazes me and fills me with awe when I walk around Tescos pushing a trolley and filling it with shopping. Extraordinary things and just ordinary things are equally amazing when you have a new chance of life. I had lost my independence and to have that back is a miracle. 

I'm not quite sure how 2015 is ever going to measure up to last year, but I have lots of plans and dreams already, so I'm hoping for it to be even better if that's possible. It is a big year for our family as we have Rose's 21st, Sarah and Oliver's wedding and our 25th wedding anniversary. These are all massive milestones for me to aim for, moments I'm looking forward to that I didn't dare ever hope to have. 

I'm also well underway with writing my book and one of my New Year resolutions is to get my first draft completed during the first part of this year. I'm also continuing with my writing course and hope to have more articles published during this New Year.

I'm not going for all the slimming and exercise resolutions, but after all the festivities I just plan to get back to eating sensibly and healthily and making sure I keep up my walking, yoga and exercise bike. I have got some new yoga DVDs for Christmas and a yoga mat, so I'm planning to step up the yoga a little. 

Of course, it goes without saying that our family will continue to raise awareness of both organ donation and pulmonary hypertension and in this respect the New Year has started well with an article in the Japanese Times, a Japanese national newspaper, about the UK's Genomics programme. The article features an interview I did with Kyodo news on genetic testing. 

http://www.japantimes.co.jp/news/2015/01/01/world/science-health-world/major-genomics-program-underway/#.VKVD9ktMI9e

I know that time has gone on now since my transplant and it is hard to gauge where I would be without it. I cannot thank my donor enough for the wonderful experiences and opportunities that I have had since being given my second chance at life. Without my donor none of them would have been possible including all the new hopes and dreams I have for this new year.

I wish everyone a very happy and healthy New Year and hope that you have many plans and dreams that you can make come true. Here's hoping it is going to be a good one with more new opportunities and experiences.

Genetics may be the Heart of the Matter

It's May already and although the first of May has started off wet and cool it can often be a glorious month, so I am really looking forward to it. The forecast for the first bank holiday of the month and this weekend is looking good. I'm now seven months post transplant and still doing incredibly well.

I had a bit of a blip with my lung function levels during the Easter period, which went a bit haywire and dropped a lot more than they should, but as everything else showed up totally fine I just had to keep a careful watch on things to make sure they didn't continue declining. It was put down to my reduction in steroids, which the team had reduced on my last visit. I ended up missing the PH conference because of it, but decided it safest to err on the side of caution, as I wasn't sure if I was coming down with something or not and my immune system is already very low. I didn't want to risk the chance of infection as the conference is very busy with lots of people, but I was disappointed to miss my friends. My lung function readings are now much better again and back to where they were, so I think I did the right thing.

This month there is a feature on the genetics of Pulmonary Arterial Hypertension in the British Heart Foundation magazine, 'Heart Matters' and Professor Morrell, who is heading the research, explains all about it. I was also interviewed for this feature, having had Idiopathic Pulmonary Arterial Hypertension before my transplant and having been tested to see if I carry the known genes that can be an indicator that it may be in the family. The research work is a continuing process, but there is hope that there may be a breakthrough, which will enable the development of future drugs to help all PH sufferers. The British Heart Foundation have given generous funding to enable this research. Below are the links to the articles.

http://www.bhf.org.uk/heart-matters-online/may-june-2014/research/pah/kath-graham.aspx

http://www.bhf.org.uk/heart-matters-online/may-june-2014/research/pah.aspx

A cure or breakthrough cannot come too soon. Some of my friends are really suffering with this disease. Several are waiting for transplants and are having to face long waits because of the shortage of organ donors and then they will have to go through the process of the transplant and the ongoing care and drugs that are required afterwards. If a cure could be found or better drugs to control the condition then patients wouldn't have to face these options. 

Some of my friends are coping with other diseases, such as Lupus, which has also caused their PH. Others have PH through having bloods clots on their lungs and although they have had their PH improved through having the Pulmonary Endarterectomy operation to remove the clots, some of the clots are too distal for removal, so they still have to cope with PH. Many PH patients are not eligible to have a transplant, so the biggest hope is that a cure can be found or a breakthrough that can help the treatment of PH more efficiently. 

We all live in hope for this day and for a breakthrough to come soon.