MD1003 biotin trial

Tourbah A, Lebrun-Frenay C, Edan G, Clanet M, Papeix C, Vukusic S, De Sèze J, Debouverie M, Gout O, Clavelou P, Defer G, Laplaud DA, Moreau T, Labauge P, Brochet B, Sedel F, Pelletier J; MS-SPI study group. MD1003 (high-dose biotin) for the treatment of progressive multiple sclerosis: A randomised, double-blind, placebo-controlled study. Mult Scler. 2016 . pii: 1352458516667568. [Epub ahead of print]

BACKGROUND:Treatment with MD1003 (high-dose biotin) showed promising results in progressive multiple sclerosis (MS) in a pilot open-label study.
OBJECTIVE:To confirm the efficacy and safety of MD1003 in progressive MS in a double-blind, placebo-controlled study.
METHODS:Patients (n = 154) with a baseline Expanded Disability Status Scale (EDSS) score of 4.5-7 and evidence of disease worsening within the previous 2 years were randomised to 12-month MD1003 (100 mg biotin) or placebo thrice daily, followed by 12-month MD1003 for all patients. The primary endpoint was the proportion of patients with disability reversal at month 9, confirmed at month 12, defined as an EDSS decrease of ⩾1 point (⩾0.5 for EDSS 6-7) or a ⩾20% decrease in timed 25-foot walk time compared with the best baseline among screening or randomisation visits.
RESULTS:A total of 13 (12.6%) MD1003-treated patients achieved the primary endpoint versus none of the placebo-treated patients (p = 0.005). MD1003 treatment also reduced EDSS progression and improved clinical impression of change compared with placebo. Efficacy was maintained over follow-up, and the safety profile of MD1003 was similar to that of placebo.
CONCLUSION:MD1003 achieves sustained reversal of MS-related disability in a subset of patients with progressive MS and is well tolerated

The promise of High dose biotin has prompted some people to smaffle loads of low dose pills but what is the promise.? 

This is the report of the trial in progressive MS and it would appear that 87.4%  did not meet the desired result. 

With this level of efficacy it would be hard to see how this would get approved, but hey copaxone and beta interferons got approved when it was thought to not work for most of the users, so what do I know.

If I am thinking a drug was stopping progression, I would be aiming for the MS not to get worse, i.e. halt the disease process.

So to ask for something to make things better is a harder ask and suggests to me either MD1003 is (a) a symptom control agent or (b) someting that is a remyelination/repair agent.

The route to the development of MD1003 was not from a hypothesis science-based route ,but by a serendipodous observation in a study on MD1003 where someone with suspected basal ganglia disease happened to have MS.

The authors claim that biotin is promoting axonal remyelination by enhancing myelin production and by reducing axonal hypoxia  (lack of oxygen) through enhanced energy production.

More studies are planned,

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  • The following is relevant to this: A paper from 1999 suggests there is a significant reduction in cerebrospinal fluid biotin in epileptics and patients with multiple sclerosis compared to controls.
    (Acta Neurol Scand. 1999 Jun;99(6):387-92. Cerebrospinal fluid levels of biotin in various neurological disorders). I have mentioned this paper before.

    Reduction of biotin levels in patients with multiple sclerosis could be attributed to intestinal malabsorption caused by the underlying disease or a biotin-binding immunoglobulin which may be involved in multiple sclerosis pathogenesis.

  • “This is the first time that a drug has reversed the progression of the disease in a statistically significant proportion of patients,” said Dr. Tourbah. “Almost no progression was observed in patients treated with MD1003 for 24 months, and this has never been observed before."

    So if the comments from the lead investigator are right – it halts (pretty much) PPMS in its tracks.

    Of course, much more needs to be done… but this is something, surely?

    • Well you can't fault Dr Tourbah's positivity. Whether its warranted is another matter entirely as MD observes.

  • It seems to me that from the two published trials to date that only the inactive progressive MSers achieved a positive result. Like over 3k Facebook Biotin group members I am one that after 7 months of taking it, it has done diddly squat to my MS however one blissful side effect is that rate of hair growth has been phenomenal now long lustrous locks!

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