This short blog is on the excellent Autosomal Dominant Polycystic Kidney Disease (ADPKD) event held at Salford Royal Hospital on Saturday 16th November, 2013. The event was organised by Tess Harris of the PKD Charity and hosted by Dr Grahame Wood, consultant Nephrologist at Salford Royal.
If I had to sum up the event in one word that word would be ‘Progress’.
Dr Kath Hillman gave the clearest explanation I’ve heard of the genetics behind ADPKD and in the process addressed some of the false preconceptions that I and many others have acquired over the years. Put simply, if one parent has ADPKD then any child of that parent has a 50% chance of inheriting the disease. When my mother was diagnosed with the disease I believed that ADPKD affected the first born child and only females. I’ve no idea where or when that belief arose but clearly it’s incorrect.
In August of this year the US Food and Drug Administration (FDA) vetoed the approval of a drug called Tolvaptan which for a number of years has given ADPKD sufferers hope that a treatment would prevent or slow down the development of the cysts that eventually destroy the kidney. The FDA decision was based on the discovery of liver problems in some of the patients involved in the trial coupled with a lack of data associated with 23% of patients in the trial. The good news is that the drug company may amend the trial and try again and the European Regulator is also interested in pursuing further trials.
Professor Pat Wilson, chair of the PKD Charity Research Advisory Board give a detailed explanation of the cell biology that causes ADPKD and the many strands of research that are currently taking place. The majority of those strands are focused on slowing down or stopping cyst development and cover solutions from drugs to diet.
It struck me during Professor Wilson’s talk how lucky we are that we have people with the passion and dedication to devote themselves to research potential treatments and cures for our condition.
It was in the area of transplantation of ADPKD patients in a talk by Mr David Van Dellen, Consultant Transplant Surgeon at Manchester Royal Infirmary, that I sensed real progress. I’m a transplant patient just past my tenth anniversary and the progress and change over that ten year period amazed me. Not only are surgeons now transplanting from donors who in the past would have been incompatible with the patient including those with differing blood types but they now have techniques to flush toxins and antibodies from donor kidneys. This news, coupled with the work that Dr Grahame Wood and his colleagues are performing looking for potential live donors may mean an increase in the number of ADPKD sufferers that don’t have to dialyse prior to transplantation. Real progress that has a personal resonance as my son is also a sufferer of ADPKD.
I’ll end by saying you should try to get to events like this as they provide such valuable information and advice. I began this blog by using the word Progress to sum up the days events. I’ll close by choosing two more words. Optimism and hope.