Multiple Sclerosis Society of Canada

MD1003

Overview:

  • Also known as: High-dose biotin (vitamin H)
  • Company: MedDay
  • Route of Administration: Oral, 300 mg/day
  • Type: Water-soluble vitamin
  • Emerging treatment for: Progressive MS
  • Status: In Phase III of clinical trials

MD1003 is under study for its potential capacity to promote myelin synthesis, impact disease progression and improve disability in people with primary and secondary progressive MS.

How it Works

MD1003 is a highly concentrated formulation of biotin (vitamin H). D-biotin is an FDA-approved food additive and Health Canada-licensed natural health product which, at concentrations of 100 – 300 mg/day in the MD1003 formulation, is ~10,000 times higher than the recommended daily intake as a food supplement. MD1003 at this dose has been shown to play a role in stimulating myelin production and improving nerve impulse conduction; specifically, it activates several enzymes – including acetylCoA carboxylase – involved in energy production and the synthesis of myelin.

Research and Results

A proof-of-concept, pilot open label study involving 23 participants with primary and secondary progressive MS demonstrated clinical improvement within 2 – 8 months in 91% of participants. Two phase III clinical trials investigating the efficacy of MD1003 in people with MS have been ongoing in France and the UK:

  • MS-SPI is a randomized, double-blind, placebo-controlled phase III clinical trial that was recently completed. Primary outcomes include improvements in disability measures (a decrease in the EDSS score or an improvement of at least 20% in a timed 25-foot walk test) with MD1003 compared to placebo in 154 participants with primary and secondary progressive MS. Following two years of treatment, the primary outcome was met with a significant decrease in EDSS score in the MD1003 group compared to placebo controls. The researchers estimated that participants who were given MD1003 had a 67% decreased risk of disease progression (4% of participants treated with MD1003 exhibited EDSS progression compared to 13% in the placebo group).
  • MS-ON is a randomized, double-blind, placebo-controlled phase III clinical trial investigating visual improvement in participants suffering from chronic visual loss from MS-related optic neuritis. During the two-phase study, 65 participants received MD1003 and 28 received placebo during the initial 24 weeks. For the following 24 weeks, all participants received the drug. Overall, those participants who were treated with MD1003 experienced slightly improved eyesight than those given placebo, but the effect was not statistically significant. Only a subset of participants who had progressive visual loss seemed to experience some benefit from the treatment.

Adverse Effects Reported

In the pilot study of 23 participants, no adverse effects were reported in 20 cases. Two participants were noted to have transient diarrhea. Two participants died from apparently unrelated causes (cardiac failure and pneumopathy, respectively) between 12 and 36 months after treatment onset; in both cases, death could not be attributed to the treatment.

References

Sedel F et al. (2015). High doses of biotin in chronic progressive multiple sclerosis: A pilot study. Mult Scler Relat Disord. 4(2):159-69.