High dose biotin as treatment for progressive multiple sclerosis

Published:September 29, 2017DOI:


      • Daily doses of high dose biotin, 300 mg per day, were well tolerated and safe.
      • Daily high dosed biotin for up to a year did not induce sustained improvement in persons with progressive MS.
      • One third of patients worsened, probably related to their disease.
      • Several patients developed increased causalgic facial pain on high dose biotin, with improvement on biotin cessation.



      Published data suggested high dose biotin improved patients with progressive MS. We wished to determine benefits and side effects of administering daily high dose biotin to patients with progressive multiple sclerosis in a large MS specialty clinic.


      Forty-three patients with progressive multiple scleroses were prescribed pharmaceutical grade biotin as a single daily dose of 300 mg/day. Brain MRIs were performed at baseline and after one year on biotin. Quantitative neurologic exams (EDSS) and blood work monitoring for biotin toxicity were performed at baseline and every three months thereafter.


      High dose biotin was safe, and well tolerated, with no evidence of toxicity on blood work and no new lesions on brain MRIs. None of the patients’ EDSS scores improved. One-third of patients (38–43%) worsened, most often with increased lower extremity weakness, worsened balance, and more falling, with two patients worsening sufficiently to increase their EDSS scores by 0.5. Several worsened patients improved after stopping biotin.


      High dose biotin was safe and well tolerated, but of no demonstrable long-term benefit. More than one-third of patients worsened while on biotin, most likely due to their disease, but in some patients also possibly due to the inability of their injured central nervous systems to respond to the increased metabolic demands induced by biotin.


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        • Anagnostouli M.
        • Livaniou E.
        • Nyalala J.O.
        • et al.
        Cerebrospinal fluid levels of biotin in various neurological disorders.
        Acta Neurol. Scand. 1999; 99: 387-392
        • Barbesino G.
        Misdiagnosis of graves' disease with apparent severe hyperthyroidism in a patient taking biotin megadoses.
        Thyroid. 2016; 26: 860-863
        • Birnbaum G.
        • Iverson J.
        Dalfampridine may activate latent trigeminal neuralgia in patients with multiple sclerosis.
        Neurology. 2014; 83: 1610-1612
        • Elston M.S.
        • Sehgal S.
        • Du Toit S.
        • Yarndley T.
        • Conaglen J.V.
        Factitious graves' disease due to biotin immunoassay interference-a case and review of the literature.
        J. Clin. Endocrinol. Metab. 2016; 101: 3251-3255
        • Heidker R.M.
        • Emerson M.R.
        • LeVine S.M.
        Intersections of pathways involving biotin and iron relative to therapeutic mechanisms for progressive multiple sclerosis.
        Discov. Med. 2016; 22: 381-387
      1. Kappos L., Bar-Or, A., Cree, B., Fox, R., et al. 2017. Efficacy of siponimod in secondary progressive multiple sclerosis: results of the phase 3 study. Annual Meeting of the American Academy of Neurology. Boston, MA, USA: American Academy of Neurolgy.

        • Lublin F.D.
        • Reingold S.C.
        • Cohen J.A.
        • et al.
        Defining the clinical course of multiple sclerosis: the 2013 revisions.
        Neurology. 2014; 83: 278-286
        • Minkovsky A.
        • Lee M.N.
        • Dowlatshahi M.
        • et al.
        High-dose biotin treatment for secondary progressive multiple sclerosis may interfere with thyroid assays.
        AACE Clin. Case Rep. 2016; 2: e370-e373
        • Montalban X.
        • Hauser S.L.
        • Kappos L.
        • et al.
        Ocrelizumab versus placebo in primary progressive multiple sclerosis.
        N. Engl. J. Med. 2016;
        • Patergnani S.
        • Fossati V.
        • Bonora M.
        • et al.
        Mitochondria in multiple sclerosis: molecular mechanisms of pathogenesis.
        Int. Rev. Cell Mol. Biol. 2017; 328: 49-103
        • Polman C.H.
        • Reingold S.C.
        • Banwell B.
        • et al.
        Diagnostic criteria for multiple sclerosis: 2010 revisions to the McDonald criteria.
        Ann. Neurol. 2011; 69: 292-302
        • Sedel F.
        • Bernard D.
        • Mock D.M.
        • Tourbah A.
        Targeting demyelination and virtual hypoxia with high-dose biotin as a treatment for progressive multiple sclerosis.
        Neuropharmacology. 2016; 110: 644-653
        • Tourbah A.
        • Lebrun-Frenay C.
        • Edan G.
        • et al.
        MD1003 (high-dose biotin) for the treatment of progressive multiple sclerosis: A randomised, double-blind, placebo-controlled study.
        Mult. Scler. 2016; 22: 1719-1731