MD1003 (high-dose biotin) for the treatment of progressive multiple sclerosis: A randomised, double-blind, placebo-controlled study

Abstract

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.

Complementary and alternative treatments of multiple sclerosis: a review of the evidence from 2001 to 2016

“…Tourbah et al , a double-blind RCT analysing 154 participants,21 found that high-dose biotin (MD1003) reduced disability progression and improved the clinical impression of change in patients with progressive MS compared with placebo. It also resulted in a sustained reversal of disability in 12.6% of treated patients, which was significantly more than the control (0 patient).…”

“…One paper21 was included based on personal knowledge and was assessed by the same criteria. A single author (SBC) reviewed all articles.…”

Is there a Link between Vitamin B and Multiple Sclerosis?

“…Furthermore, in a randomized double blind placebo controlled clinical study in 154 patients with progressive MS, high dose of MD1003 (100 mg) resulted in reduced Expanded Disability Status Scale (EDSS) progression and improved clinical impression of change compared to placebo group. MD1003 was shown to be safe and achieved sustained reversal of MS related disability in 13 patients with progressive MS [52]. These studies suggest that biotin plays a role in MS; however, further research is required to determine the role of biotin deficiency in MS and the mechanism of action of high dose biotin supplementation in MS patients.…”

Population Pharmacokinetics of Exogenous Biotin and the Relationship Between Biotin Serum Levels and In Vitro Immunoassay Interference

“…It should be noted that the doses examined were approximately 100-to 500-times higher than the recommended daily intake of 30 μg/day [10]. Higher doses were also simulated; currently, very-high biotin doses are only being used in the clinical trial setting, as a potential treatment for multiple sclerosis [21,22]. A study in healthy volunteers (n = 8) found that, following a single 100 or 300 mg dose of biotin, median t max occurred at 1.25 and 1.5 h, respectively.…”