I have written a quick description of the talk. No direct quotes. Apologies to those who read this when it was only partially submitted.
I have also fixed some things due to Sharon's comments, below...
This is all paraphrasing and editorial: there are no direct quotations.
The usual suspects have managed to taint only one of the MedDay group (Dr. Tourbah). Either a spy in their midst or a counter-spy maybe. Actually it's the top name on the paper, so maybe he's just being completely transparent, and collecting fees. He was the one speaking and answering questions.
BTW it's quicker to download it if you can, from either place.
They had already done one trial before this one, so they may have ongoing data, because they probably kept those patients on the drug. Same with this study and subsequent ones, which they will report at a future meeting. We will see therefore, if the improvements continue.
The primary purpose was to test effects on the Expanded Disability Scale (EDSS), and timed walking speed measurements.
He said MS might be caused by virtual hypoxia: a mismatch between energy demand and energy production, caused by the inability of mitochondria to consume oxygen. Not clear if this is limited to the brain; I think not, eventually, but maybe the divide is between brain/spine and rest of nervous system. That would mean it follows the CSF (Cerebro-Spinal Fluid) from brain to spine (it does, all the way to mice, in other experiments).
They weren't taking DMDs, introduced withing 3 years, though they could if introduced less recently, or Fampridine (improves walking speed in relapsing-remitting patients), if introduced within 3 months. The fact that these were not RR patients may mean it works for them too, indicating they are treating the same disease (relapsing and progressive diseases share the same cause).
154 patients, 1/3 placebo, had 2 adverse events in each group, with 1 suicide, not related.
13 treated patients improved by trial standards, and none in placebo group.
Twice as many patients improved in EDSS as in walking. This is because EDSS is defined more by ability to walk at all, than by speed of walking.
Number of improved patients was statistically significant. Looking at the EDSS graph, it looked like progression was halted. This means something to me because I have always said, halt the progression and I'll do the rest.
This is borne out by slight improvement overall in average EDSS.
Improvements in EDSS at 3 months continued until 9 months, with a deviation at 3 from placebo, of 67%, illustrating the end of placebo effect. Continuation of the improvement lasted for the full 9 months.
When combined with the results of the other study, the results on EDSS are similar.
For timed walking, on average the treated patients were 6 seconds faster than the placebo group. This might not seem like much, and it doesn't get me out of my walker, but I can probably do that myself once I stop backsliding. The trend looks like I will stop backsliding.
Twice as many placebo people as the treated ones, had adverse events, but biotin appears to change the results of antibody tests, so the tests themselves will have to be reconsidered for people on Biotin.
There were questions both from audience and phone calls.
The question session contained:
Will you get marketing approval?
We have to do two more studies, and then European approval, then (possibly) one more, for the US, depending on whether they like what we have done by the end of the year.
Note re Sharon's update: Are they waiting till 2017 to give the other DMD vendors a chance to make some more money before they go to market?
Any other improvements?
We are testing for them. Some look like they do improve.
Cognition?
Yes we have seen this, seems significant. Some others look good too.
Do you plan to look at relapsing-remitting? If not,why not?
Not specifically, but we do have some R-R patients in our Optic Neuritis study who have permanent ON problems, so if they improve, we will know it does not matter whether they have remissions of other symptoms or not. So we will test both. Biotin does not seem to care. It is a tough requirement, but we are hopeful.
Does it rebuild myelin, or stop progression?
Both. We think it modifies the primary disease. We aren't sure if it does both, repair myelin, and decrease virtual hypoxia, but we think it will.
Should people start buying biotin?
Don't know what people will do, but reasons they shouldn't are: a) it might be toxic in those doses, especially for rabbits and pregnant women, and b) because it also seems to dramatically change the results of medical tests including thyroid and pregnancy tests. Plus medical grade biotin at these doses is more of a guarantee of purity, and even whether it is biotin at all.
Can you combine it with other DMDs?
Yes, many subjects did, with no problems.
Is your research platform able to look at CSF?
No; we couldn't ask them to have spinal taps, but research will continue.
Can you predict who will benefit?
No.
