Among hypotheses that tend to explain the cause(s) of progressive MS, it has been proposed that progressive degeneration could be linked to a phenomenon of “virtual hypoxia” caused by a mismatch between increased energy demand by the demyelinated axon and decreased energy production because of mitochondrial injury. No drug has been found to have any impact on progressive multiple sclerosis (MS).

Biotin is a vitamin acting as a coenzyme for carboxylases involved in key steps of energy metabolism and fatty acids synthesis. Among others, biotin activates acetyl CoA carboxylase, a potentially key regulating enzyme in myelin synthesis.  The aim of a pilot study was to assess the clinical efficacy and safety of high doses of biotin in patients suffering from progressive MS. Twenty-three consecutive patients with primary and secondary progressive MS who originated from three different French MS reference centers were treated with high doses of biotin (100–600 mg/day; median 300 mg/day in 3 divided doses) from 2 to 36 months (mean=9.2 months). Judgement criteria varied according to clinical presentations and included quantitative and qualitative measures.

In four patients with prominent visual impairment related to optic nerve injury, visual acuity improved significantly. Visual evoked potentials in two patients exhibited progressive reappearance of P100 waves, with normalization of latencies in one case. Proton magnetic resonance spectroscopy (H-MRS) in one case showed a progressive normalization of the choline/creatine ratio. One patient with left homonymous hemianopia continued to improve from 2 to 16 months following onset of treatment. Sixteen patients out of 18 (89%) with prominent spinal cord involvement were considered improved as confirmed by blinded review of videotaped clinical examination in 9 cases. In all cases, improvement began 2 to 8 months after starting treatment.

• Biotin activates enzymes involved in energy production and myelin synthesis.
• 91.3% of SPMS or PPMS patients improved clinically with high doses of biotin.
• Improved conditions included chronic optic neuropathy, homonymous hemianopia or myelopathy.
• In all cases improvement was delayed from 2 to 8 months following treatment׳s onset.
• These preliminary data suggest that high doses of biotin might have an impact on disability and progression in progressive MS.
• Two multi-centric double-blind placebo-controlled trials are currently underway.

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ReferencesMultiple Sclerosis and Related Disorders. March 2015; 4(2):159–169