Targeting demyelination and virtual hypoxia with high-dose biotin as a treatment for progressive multiple sclerosis

Neuropharmacology. 2016 Nov;110(Pt B):644-653. doi: 10.1016/j.neuropharm.2015.08.028. Epub 2015 Sep 5.


Progressive multiple sclerosis (MS) is a severely disabling neurological condition, and an effective treatment is urgently needed. Recently, high-dose biotin has emerged as a promising therapy for affected individuals. Initial clinical data have shown that daily doses of biotin of up to 300 mg can improve objective measures of MS-related disability. In this article, we review the biology of biotin and explore the properties of this ubiquitous coenzyme that may explain the encouraging responses seen in patients with progressive MS. The gradual worsening of neurological disability in patients with progressive MS is caused by progressive axonal loss or damage. The triggers for axonal loss in MS likely include both inflammatory demyelination of the myelin sheath and primary neurodegeneration caused by a state of virtual hypoxia within the neuron. Accordingly, targeting both these pathological processes could be effective in the treatment of progressive MS. Biotin is an essential co-factor for five carboxylases involved in fatty acid synthesis and energy production. We hypothesize that high-dose biotin is exerting a therapeutic effect in patients with progressive MS through two different and complementary mechanisms: by promoting axonal remyelination by enhancing myelin production and by reducing axonal hypoxia through enhanced energy production. This article is part of the Special Issue entitled 'Oligodendrocytes in Health and Disease'.

Keywords: Biotin; Multiple sclerosis; Promyelinogenic agent; Virtual hypoxia.

Publication types

  • Review

MeSH terms

  • Animals
  • Biotin / therapeutic use*
  • Demyelinating Diseases / etiology*
  • Demyelinating Diseases / therapy*
  • Disease Progression
  • Dose-Response Relationship, Drug
  • Humans
  • Hypoxia / drug therapy*
  • Hypoxia / physiopathology
  • Multiple Sclerosis / complications*
  • Multiple Sclerosis / therapy
  • Vitamin B Complex / therapeutic use*


  • Vitamin B Complex
  • Biotin