This post is a bit different from all my earlier pieces on the complexities, challenges, interpretation of MND research and clinical trials and, in particular, my pet hate: the creative, enthusiastic, and often frankly Olympian level misuse of statistics.
As a fortunate 12‑year survivor since diagnosis, I’ve been privileged to witness more than a decade of research history unfold.
Although there still isn’t an effective treatment for the vast majority of patients, research really has moved forward in this time. Scientific discovery, along with relentless increases in computing power, falling costs, and now the arrival of AI all give genuine reasons to be optimistic.
I hope this short, snappy, post gives you both hope and food for thought, and ideally something more nutritious than the usual social media diet.
This is my first post for many many months, but even in that short time the world has seen what can only be described as an explosion of AI. AI can now happily provide structured information on everything from opening a can of beans, in a detail you really didn’t need to know, to explaining the most complex scientific concepts. And in that same short time ‘generative, general‑purpose” AI appears to be changing writing. Where will it lead us? Who knows.? But it has certainly changed how I approached this very post. And if I’m honest, I’m still not entirely sure where to go next.
To that end, I’m not going to make this a long piece. Think of it as a starter for ten, and yes, an opinion post. AI doesn’t give opinions. So, folks, this is me, OK? Not AI. If AI ever starts writing like this, we’re all in trouble. Perhaps we are already!?
I’m keeping things very short indeed, with a single reference (actually 2 in one item!) for each of 10 chosen highlights of the last decade. You can explore further, or simply take away the very briefest of messages.
Here are my top 10 research advancement highlights from the last decade. They’re not in any particular order, literally just as they came into my head. Are there other advances? Yes, of course. I don’t pretend to cover everything. These are simply the ones I feel just might have impact on the future and show that the research world hasn’t been asleep at the wheel…..
1. The first truly effective treatment, tofersen, for any form of MND (SOD1 MND), has gone from just an idea to an approved drug in the last 10 years.
2. The emergence of virtual biopsy techniques for our disease.
3. Greater understanding of the primary disease pathology: TDP‑43 protein abnormalities.
4. Increased global collaboration.
5. More inclusive and shorter trials, including drug‑screening platforms, enabled by the discovery of the first truly robust biomarker, NfL.
6. The emerging role of the transcriptome.
7. The importance of the immune system in our disease.
8. Epigenetics (see transcriptome!).
9. The first ventures into disease prevention experimentation with the ATLAS trial for SOD1 MND.
10. Gut TDP‑43 changes that might help predict future MND diagnosis.
And that’s my post!
Oh, OK, OK, all right. I’ll say just a little bit more before I terminate! As a completely unrelated point I watched Terminator 2 for the first time in many years. Humm, makes you think now.
The discovery of the first robust biomarker, NfL, could really change the pace of the race for truly effective treatments. I say ‘could’ because researchers still haven’t discovered any treatment candidate, other than tofersen, that has both significantly lowered NfL and produced an accompanying positive clinical change. If that second one appears, then the ‘could’ would most certainly become ‘has changed’.
In my next post, coming very shortly, and in the interests of brutal balance, I’m going to write a more substantive piece on the ‘less attractive’ characteristics of the research world that I have also observed, sadly, in this very same decade of progress. Might AI help research with some of these behaviours? It just might.
Living with Motor Neurone Disease 