Talkin’ about my Regeneration….

“Cure sometimes, treat often and comfort always.” 
Hippocrates
Hippocrates, an interesting fella….

Hippocrates was an interesting fella, who said, or is certainly thought to have said, a lot of interesting and thought provoking stuff. He’s considered the father of medicine, a rather grandiose accolade, indeed he’s thought to have written, or at very least inspired the hippocratic oath, which is still the primary oath of medical ethics to this day. A bit of a legend.

The quote that starts this blog popped up on my Bloomberg terminal one day. Those of you who work in financial services and who use Bloomberg, will be familiar with how they have a daily quote upon logging in, something cerebral to broaden your horizons prior to commencing the daily grind. This particular quote struck a chord with me.

What chord did it strike Martin!? I hear you excitedly ask, patience dear reader, patience. Let’s dissect the quote:

“Cure sometimes” — You have young onset Parkinson’s don’t you Martin, so you want a cure? Yes I also have ginger hair but, although it’s receding a bit, like my Parkinson’s it’s likely to be a part of me for a long time yet. A ‘cure’ would be nice, but so would a win on the Euromillions and there’s long odds on either happening anytime soon.

“Treat often” — But Parkinson’s has lots of effective treatments doesn’t it Martin, just take your pills, feel better and carry on, carrying on! Hmmm not sure I agree with you on this one. Yes it’s true that compared to many other neurological conditions, there are a range of treatment options, some of which can be quite effective. But even the most effective treatment levodopa can have to severe side effects as the condition progresses, such as dyskinesia, which is arguably worse than the disease itself.

“Comfort always” — I think you are probably needing comforted after reading the above Martin, have a hug! Aww thanks, there’s is hope for the future though, in fact now is as prolific a time for Parkinson’s research as there has perhaps ever been. You only have to read the excellent Science of Parkinson’s Disease blog, written by the excellent Simon Stott, to realise just how much great work is going on.

Let’s be brutally honest for a second. As a patient community we all wonder, how relevant is this going to be to us living with the condition today?

Those of you who have been ‘with’ Parkinson’s for longer than my 3 years ‘with’ our shared bedfellow, have been hearing breakthroughs in 5 to 10 years for, well, a heck of a lot longer than 5 to 10 years….. 6 months is a long stretch with Parkinson’s let alone another cycle of 5 to 10 years. Yet there is optimism that disease modification, the stopping or slowing of the condition, may be possible. There are now a number of novel compounds targeting alpha synuclein, the protein of which the misfolding of which is theorised to be the cause of disease progression. An example is PRX002, which aims to clear away the toxic protein, which is in early stage clinical trials.

The success of these clinical trials from an altruistic, whole of human race kind of view would be brilliant. Stopping disease progression would be a major milestone in the history of neuroscience. However, while this would be great for society at large — which is facing a time bomb of the burden of neurological illness increasing with an ageing population, coupled with the inadequacy of current treatments — it’s very much a stable door, bolting horse scenario for those of us already diagnosed.

The reason being is that at even at a wildly optimistic best case scenario timescale, treatments that are disease modifying are probably at least…..groan…..5 to 10 years away from reaching patients. That is not necessarily the time it will take to discover a disease modifying therapy, as we’ve discussed in previous blogs, some may already exist. The recent exenatide phase 2 trial, provided some evidence that disease modification may be possible. But taking a new treatment as an example, it has to pass through….

  • Phase 1 clinical trials to prove it’s safe
  • Phase 2 clinical trials to prove efficacy and safety in a small sample group
  • Phase 3 clinical trials to prove efficacy and safety in a large sample group

If it passes through all of the above, and phase 2/3 are where most drugs go to die, it then has to pass both regulatory approval and be commercially viable to the pharmaceutical company.

Lastly, it has to also be financially viable to national health bodies and there are numerous examples where financial reasons (drugs tend to be VERY expensive when they first come to market) have delayed drugs being made available to patients. For example the recent news story concerning the late restaurant critic AA Gill. There’s also a possibility that such treatments will only be prescribed to patients with X years disease duration, for example previously leukemia patients were only eligible for one stem cell transplant in the UK and if they relapsed were denied treatment. Furthermore much of the current thinking in Parkinson’s research is that disease modification is more likely the earlier the course of the disease, hence much of the focus on early detection.

So there’s a lot of obstacles to overcome before disease modifying drugs become available, I would question how relevant that will be for those of us living with the condition today. Parkinson’s is fundamentally a degenerative condition, so with each year spent waiting for drugs that stop/slow progression, the underlying patient gets worse. I have slow progression, yet I shudder to think where I’ll be in two years time let alone ten.

So to link back to Hippocrates and comfort always. Those of us with Parkinson’s today are likely to need some regeneration to give us the best hope of a better quality of life as stopping/slowing is not likely to come in a timescale relevant to us. The comfort is that there is hope of this, particularly with gene therapy and stem cell transplantation. For the Parkinson’s community today, the prospect of regeneration and/or better symptomatic treatment is probably far more relevant than stopping disease progression, even though that would be a huge achievement for medicine and humanity in general.

Martin


Martin is a Volunteer Research Blogger at Parkinson’s UK, he writes perspective pieces about Parkinson’s research. Read other blog posts by Martin