This comes in all sizes, shapes and flavours. Sometimes it is about how genes contribute to diseases, sometimes it is about how certain drugs work, and sometimes it is a direct question “will this treatment help prevent or cure this disease”?
All of these types of research are important – without the basic understanding of how faults in the genetic code are translated into diseases, we would not be clear about the potential “targets” for new treatments.
In ADPKD there has been a fair amount of research into all of these questions but no overwhelming answers as yet. However, I am optimistic that within a few years we will know a lot more, and the beginnings of a “fight-back” will have started.
It’s important to remember that it can take up to fifteen years from a having a “good idea” to arriving at a “widely prescribed treatment”. This will include time spent on pre clinical research (in the labs) to clinical research trials involving patients, to approval. There are many such trials in progress; some are for liver cyst patients, some for kidney cyst patients, and some for both.
Recent published research about new drugs have focussed on why the cysts in ADPKD start to form, and then to enlarge. Tolvaptan, Octreotide, and Everolimus have all been tried out in the last five years in patients with ADPKD and full medical reports have appeared which have detailed progress and set-backs. It’s fair to say that the beneficial effects of these drugs, which seemed quite promising when used in animals, have been somewhat disappointing when used in real patients.
The impact on the progression of the disease (cyst enlargement) has been modest at best, and sometimes nil. All of these treatments (which, incidentally are “old” ones, previously used for other medical conditions e.g. cancer) have potential and real side-effects.
Two important studies which should be reporting fairly soon are known as “HALT-PKD”. These trials will examine the benefit of reducing blood pressure in ADPKD, and whether using medicines known as ACE inhibitors or angiotensin receptor blockers will also prove beneficial in ADPKD. These types of medicines are already in use and are commonly prescribed for diabetic patients to prevent kidney disease developing or progressing. Prescribing these medicines is something that we could easily do for all patients if the “HALT-PKD” trials give us a clear indication to do so. For more information visit: http://cjasn.asnjournals.org/content/5/1/102.full.
This website lists all of the current research projects in ADPKD:
Currently most of the trials are not taking place within the UK but you may wish to ask your doctors to let you know if they become aware of any new trials happening in the UK, so that if you were interested and eligible, you could be considered for participation in a trail.
One of the goals of the Polycystic Kidney Disease Foundation is to raise the profile and awareness of research trials. You can find out more by visiting their website:http://www.pkdcure.org/research/clinical-trials/awareness
Professor David Goldsmith, Consultant Kidney Doctor