299. Can I be of assistance?

Long, long ago in a blog far, far away (well, nearly two years actually), I told you about my final lumbar puncture on the Ambroxol drug trial. And I promised to let you know as soon as the results were out.

I hope you weren’t holding your breath!

About a year ago, we few, we very few – well, about eighteen – trial participants were called for a meeting to share the initial findings. This ‘Stage Two’ trial had gone well, we were told, in that they had ascertained that the drug was tolerable in the doses they wanted to give. Also that lumbar punctures had shown Ambroxol does cross the blood/brain barrier, reaching the relevant parts of the brain. This is what they were hoping to find. So that’s all good.

But does it slow down or reverse or cure Parkinson’s, we all wanted to know? At least, I did, and I’m pretty sure the other seventeen were at least mildly interested. On this, the feedback was cautious. As I understand it, there was some evidence that it helped with some symptoms for some people but this couldn’t be given statistical weight. Too small a trial and no placebos.

We were asked to keep these initial findings to ourselves while the results were finalised and peer reviewed before being published.

Anyway, last week the results of the trial were, finally, made public. There’s a first rate analysis of them in the Science of Parkinson’s blog, with an explanation of how the researchers realised that this was a drug worth exploring in the first place. It puts paid to my working theory that they were trying out everything alphabetically and had already failed with tablets made from aardvarks. It leaves me in awe of the scientists beavering away to try and find something which helps. There’s also a summary of the results on the Cure Parkinson’s Trust website – they partly funded the trial.

So what happens next? Alas, more waiting. Hopefully there will eventually be a Stage Three trial with a much larger group of people for a greater length of time and placebos for comparison.  And hopefully the results will be significant.

In truth, I am finding this frustrating because, as it happens, my lived experience of the trial was that it definitely reduced my tremor. Two years on, said tremor has only recently returned to its pre-trial level. I’m fully aware that this is entirely subjective and not statistically significant. I know that my sort of Parkinson’s is not the same as your sort of Parkinson’s, so what works for me may not work for you. I do know that the placebo effect can be extremely powerful and, particularly with a brain condition, expectations can influence outcome.

I don’t for one minute want people to bypass the proper scientific procedures and start main-lining Ambroxol: current drug regulations are there to protect us from disasters such as Thalidomide. But there is definitely potential in Ambroxol which merits investigating in more depth.

I remember the late, great Tom Isaacs talking about his frustration at how slowly the research wheels grind. He described a meeting – way back – where he’d asked how he could help move things on and one scientist had replied, in all seriousness, “well, you could leave us your brain,” which was not really the answer he was hoping for.

So, putting aside giving you my brain, which I’m using at present, dear scientists and researchers, is there anything we can do to help speed things up? Is it just a question of money? How much do you need? Are there people we can lobby? Would more publicity help? Biscuits? Repeating Tom’s question, how can we help to accelerate the process?

This is not in any way a criticism of the scientists and researchers who are awesome and working their socks off.  I get that you have to do things properly but there is an urgency here. You see, I took my Grade Two piano exam and had to stop during a couple of the pieces to rein in the tremor. It would be really, really good if you could sort something out before I take Grade Three. Probably next December, if you need a deadline.

(Oh, since you ask, I got a merit. Two marks off a distinction. Thanks. Modest face emoji.)

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