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!

Tuesday, 19 January 2016

The Issue of Family Consent to Organ Donation

Organ Donation and Transplantation made the news once more last week. Sally Johnson, Director of Organ Donation and Transplantation announced that family consent will no longer be required when a potential donor has already made their dying wishes clear by signing up to the the organ donor register.

By law the consent of the 'next of kin' isn't required, but families are asked out of courtesy and respect but unfortunately one in seven families refuse to give consent when they are asked and block the potential organ donation. Their loved one's final dying wishes are ignored and the potential to save and transform the lives of others is lost. Sometimes it is because the family didn't know their loved wishes or that they were on the organ donor register.

The family refusal rate is the biggest single identified obstacle to organ donation in the UK and with the percentage of organ donations decreasing, it is vital that families discuss their views on organ donation and that people let their families know that they are on the organ donor register in case they have an untimely death and are then are in a position to save lives.

Five hundred and forty seven families have blocked organ donation since 2010, meaning potential transplants of approximately one thousand two hundred weren't allowed to go ahead. It also means that the potential donors didn't have their final and last wishes fulfilled - that of giving the gift of life or giving a better quality of life to others.

As I well know, there's no greater gift - the final act of donating organs is truly the greatest gift of all and a wonderful legacy to give to others. Not only does an organ donation save and transform the life of the recipient, it affects the lives of those that surround that person, so that this final legacy ripples out and touches many. It is both tragic and sad that the opportunity to do this could ever be stopped by next of kin when it's what their loved one hoped for - that they could make a difference to someone's life or even save it, and more than one person's life too.

Now nurses will be both speaking on the donor's behalf and there will be a leaflet given to families to explain the process and the organ donation will take place even if the family do not consent as long as  a person is registered on NHSBT's organ donor register. This way the donor's expectations that they will save and transform lives will be respected and carried out. It is a more honest approach on behalf of the donor.

Personally, I think this move cannot come too soon. I've always been surprised to know that a family can turn round and veto their loved one's final wish. Even in the event of a tragedy and trying to understand what they will be going through I still find it shocking. I would always like to think that in these circumstances I would be brave and honourable enough to respect my own family members' wishes. 

It is a known fact that families can take some small comfort in knowing that their loved one has saved and transformed lives. It doesn't replace the grief and loss, I know that, but a miracle does come from tragedy and that is a wonderful legacy to leave and donor families must also take great pride in this. 

This week's news is a small step in the right direction, but there is still a long way to go in the UK, regarding openness in speaking about dying matters. Most of us don't want to think of it, don't want to speak of wills, organ donation or organising our affairs in case of death. It is part of the problem and maybe we need to work on changing our thinking and our attitudes to what is a natural course in everyone's life. We shirk away from it and almost pretend it doesn't happen - it makes us uncomfortable.  Death is a fact of life though and happens to us all, so it should be a sensible and natural thing to have these discussions within our families and then everyone is clear. Being more open about our final wishes could go a long way in increasing the numbers on the organ donor register.

Below are links to the press release, the issues on family consent rates on the ODT website,  an interesting piece in the Guardian by fellow transplantee Sharon and of course the link to sign up just in case you already haven't!

Wednesday, 6 January 2016

CMV Virus

I wanted to explain a little more about cytomegalovirus (CMV virus) after my recent experience with it,  purely to raise awareness and especially for other transplant patients. Obviously I'm not a medic and can only give my own experience and events that happened to me and I know others may have had similar experiences or experiences that are very different as these things can affect us in different ways.

I'd had a busy weekend and then a late night out on the Monday. On the Tuesday I felt extremely tired, so much so I fell asleep on the settee just after breakfast, which was very unusual for me. I don't ever sleep in the day anymore, so at first I thought I was just shattered and really tired from the weekend. I decided to rest and have a lazy day to catch myself up. The following day I was much the same - totally wiped out - so I rested again. As the days went on this didn't improve and as a transplant patient I have to keep a diary and record my temperature daily and I noticed my temperature was raised.

I phoned the transplant team like we are advised and as I was going into clinic that week for blood tests, the transplant nurse said she would arrange for one of the doctors to check me over. Friday came and I was feeling really unwell - extremely tired and spiking temperatures on and off all day long. The doctor checked me over, but my lungs sounded clear and a chest X-ray was clear so he wasn't too worried and thought it looked like it was probably a virus. I was advised to stay in, keep warm and drink plenty of fluids and then go to my GP should it continue and update the team should I need to do so.

The symptoms carried on and the tiredness felt more extreme and so I went to see the GP.  He increased my Azithromycin, a strong antibiotic which I take three times every week anyway to protect my lungs. I was advised to take it every day for the next ten days and see how things went. He thought it better to get these strong antibiotics underway as my temperature indicated infection. I advised my transplant team and they were happy with this, it was their next plan of action and they advised me to phone them after a week if things weren't any better.

A few more days passed and I felt no different and at times when my temperature was high, I felt dreadful, I was weary and I had very little appetite. I had a few stomach upsets, but nothing that made me think it could be something I'd eaten causing it all. It almost felt like I was being poisoned or something. I even started to Google carbon monoxide poisoning although we have alarms to detect it as I just couldn't place how I felt. I knew it wasn't the usual cold or flu though and I made my mind up that I'd phone the transplant team first thing in the morning although a week hadn't quite passed. I'd recently had one of my immunesuppressant drugs reduced so I'd started worrying about rejection although I'd only had my blood levels checked about ten days beforehand.

When I woke in the morning though I could barely move, my temperature had hit near forty degrees and I felt extremely unwell. Rob phoned the transplant team and the nurse said I probably need intravenous antibiotics and to phone the GP so I could be taken into hospital. The GP was brilliant and came out more or less straight away after speaking to Rob and when she arrived I had deteriorated even more, the high temperature actually making me sick. She did a quick check and then phoned straight for an ambulance to blue light me to hospital and within minutes we could hear the sirens screeching down our road. I was so relieved the doctor had come out to me, she was very concerned it may be sepsis with my temperature.

So my main symptoms during the lead up to this had been extreme fatigue - an unusual tiredness; high temperatures - spiking over 39 and 40 at times; irregular and sparse stomach upsets and loss of appetite. Other symptoms of CMV can be joint and muscle pain, swollen glands and a sore throat, but I didn't experience these.

CMV can go virtually unnoticed in many people, but for those with weakened immune systems it can cause more serious symptoms: visual problems, pneumonia, retinitis and hepatitis. In a person with a weak immune system the virus can spread rapidly throughout the body damaging organs particularly the digestive system, lungs and eyes. It is a common virus belonging to the herpes family of viruses such as chickenpox and glandular fever and is primarily spread through bodily fluids, most people contracting it in early childhood, but then the virus will lie dormant in the body's cells forever.

After transplantation if the recipient or donor or both are carriers of the virus then patients are treated with an oral medication called Valganciclovir to prevent re-activation of the virus due to their weakened immune system. This treatment usually continues for at least six months. The virus is at most risk from re-activation in these first months post transplant. I had this course of medication for the first eight months after my transplant as CMV had been detected following my transplant.

At my local hospital I was admitted and treated with intravenous antibiotics for several days as we still didn't know what was wrong with me, but my symptoms did not improve and the doctors were quite flummoxed with me. I saw quite a few consultants over the five days I was there and they worked with my transplant team, but were most concerned that my temperature still kept running high and that something was still amiss. They were also quite bothered that they didn't have the expertise to deal with a heart and lung transplant patient. It was agreed between the teams to transfer me by ambulance to Papworth so they could investigate matters further.

I was very relieved once I arrived at Papworth and my consultant was by this stage thinking it could be CMV virus, although it's unusual for it to re-activate this far down the line. He decided we should start the treatment straight away before we had the blood test result back and while they did more investigations to get to the bottom of things. I started on an intravenous drug called Ganciclovir, but was warned that it may be damaging to my kidneys so treatment may have to be slowed depending on my kidney function (I already have some renal failure). The results of the CMV blood test take several days to come through and when they did it was confirmed that I had CMV and that the levels of it were extremely high. There is a complex measuring system for CMV, and I don't pretend to understand it, but I was told in simple terms that the level should be in the low hundreds and advised that my levels were currently at circa seventeen million, so things had become quite serious and my liver function tests had also revealed inflammation caused by the virus.

We then had to wait a week before another test could be done to see how the virus was doing and if the levels were lowering or not. During this period the team had expected to see some improvement in my symptoms, but I wasn't improving much at all. The team began to worry the treatment wasn't working or that the virus was a strain that's become resistant to the anti- viral drug they were using or that I may even have another problem as well as CMV. I therefore had more tests to try and rule out if I had another problem or not and samples were sent off to check if my virus was one of the resistant strains to the anti-viral drugs. It all felt very worrying and scary. I couldn't help but wonder about rejection again and worrying about what would happen if the drugs weren't working.

I was warned that I would be in hospital for quite a few weeks even if the drug was working and that they had an alternative drug lined up to try called Foscarnet, which they would have to use. My team weren't keen on this option as it is a very toxic drug on the kidneys and therefore it was going to be quite damaging on them. I knew I would have the test results back on Christmas Eve, so as we headed towards it, I was becoming quite worried. I'd been told I'd be able to go home on Christmas day for a few hours if they found the drug had lowered the virus levels, but at the same time, as it was the christmas period the team were organising the alternative drug in readiness. My consultant, however, did keep saying it may be because the virus levels are so high that it's taking so long to see improvement in my symptoms, so I hoped against hope that this would be the case and tried hard not to worry.

Christmas eve morning came around and I found out that my test results showed that the virus levels were lowering, still much too high but now a few million compared to the seventeen million of a week ago. Luckily my team were happy that things were now going in the right direction and it was decided that everything was just taking time as the virus levels were so high and that they would continue on the treatment I had been started on. I was so relieved and pleased this was the case and it was the good news that I'd hoped for. I was allowed to go home for a few hours on Christmas day and have lunch with my family and this all made my Christmas so special.

As the week went on my symptoms began to improve and my temperature lowered and I began to feel better than I had. The biggest problem I had though by this point was with my veins. By then I was on my third week in hospital and on IVs and having regular blood tests. My veins are only small and thin and the drug is very toxic, so every time I had a cannula put in, it would only last for half a day before it needed changing again, because not only was it sore at the entry point, but the drug caused bruising, swelling and blistering up my arms as my veins couldn't cope. It was decided to change to using long lines, as they are supposed to last much longer, but I was only managing a day or two using these, as my arms were so pianful.  It was a struggle for the doctors and nurses to find any veins for the next line each time and they were actually having to undertake ultrasound scans on my arms to try and locate any decent veins. I had been booked to have a Pic line put in my arm at Addenbrookes to ease things, but as it was Christmas there wasn't any availability to have this done until after the New Year.

Luckily on New Year's eve, my test results came through showing that my CMV levels had lowered again and although they were still in the thousands, they were low enough for me to switch to the oral medication Valganciclovir. It was a huge relief as it meant no more IVs or pain in my arms and also that I could go home and start my New Year waking up at home in my own bed!

Now I need to go to clinic each week to ensure the virus levels are still decreasing and check my blood levels as my immunesuppressant drugs had to be altered quite drastically so I could fight the virus and to stop it going out of control. It's going to take some time to get this all back in balance once more and probably at least six months on the oral medication for the CMV.  I also had to be taken off some of my more routine medication, which helps to protect my heart because of the damage to my liver. Hopefully these will be reinstated soon as things become more settled.

So that is my experience of CMV virus, not an easy one, but it could have been much worse and I'm getting myself back on track now. I wanted to share it and just say if you're a transplant patient and are experiencing symptoms that you're not quite sure about then it's worth asking for a CMV test at the outset just to rule it out if anything. Everything is easier on hindsight and after the experience, but obviously if I come down with any more unexplained viruses then it's the first thing I will be asking!

I'm grateful to all the staff who were involved in my care and who played a part in helping me through all this from my GP, the kind and caring paramedics who ambulanced me to both hospitals, the staff at the Lister Hospital and the transplant team, physiotherapists and all the staff on Baron ward at Papworth. Thanks to all my family and friends too for all the lovely encouraging messages, cards, pressies and popping in to see me. It really does make a massive difference when things are feeling difficult. 

I have taken some information on CMV in this blog from the NHS website below.


I would also be interested in hearing about others' experiences of CMV and especially when it may have activated, as it appears to be more common in the first year rather than a few years down the line.