A further approach was staging a ‘burning debate’ for which I stepped into the ring with Professor Patricia Coyle from Stonybrook, NY to discuss the motion, that pwMS in wheelchairs should indeed be included in DMT trials. Whilst this may appear like a home run for readers of this blog, the debate was more controversial. I still won, though not by a margin of 95:5…
Here are my slides:
And here’s a graphic from Maria Patestas’ & Leslie Gartner’s recent edition of their textbook of neuroanatomy highlighting the uneven distribution of cortico-spinal (= motor) tracts along the spinal cord. In my view this is an important factor to explain the heterogenous distribution of disability affecting the lower and upper limbs in people with chronic deteriorating (‘progressive’) MS.
Where there are more fibres, more function can be preserved. Industry and investigators need to step up, and stop giving up, trying to modify MS even when lower limb function is essentially lost – #ThinkHand !
CoI: I have been the local PI at BartsMS of the INFORMS and ORATORIO studies, and am keen to get a trial for people with EDSS >6 underway.
I still want to know the arguments against. I'm aware of the arguments for because I do read this blog.
Still dealing with my burning desire to understand the arguments against. I'm starting to think I will live in wonder forever….
I'll ask Patricia Coyle to provide her slides.
This makes so much sense, I cannot imagine how anyone could argue against it. For that reason, I'd be interested to see Coyle's slides.
It wasn't good listening
It simply makes sense. I understand that broader targets and the concern of that skewing trials. On the other side of the coin such things are constantly done with a variety of products. Trials across a more broadband demographic.
In reality this should be done across PPMS patients be they ambulatory or not. The data that comes forth as a comparator may well be highly valuable.
Quite frankly I do not understand other than for reasons of complete disclosure why tiered trials are not the standard. In many many areas of testing be that computer processors, software or even new cereals for breakfast it is. Sure, they try utilize data to target market demographics. So?
Tiered trials allow for data gathering and extraction completely aside from the main waypoints or endpoints. In medical trials this clearly seems to be a "We dont want to" .vs. "Yes we can." Is the disclosure the crux? Aka: Peoples upper limb function degraded on experimental med xxx-yyy yet it showed impacts against say EDSS?
Why cant such integrated trials have separate disclosures then?
One might think the concept of trials is to also learn all that can be learned?
I just saw a video not two days ago about medicinal cannabis that spoke to the tossing and mediocre extraction of data due to the highly focused endpoints not to mention the costing.