Remyelination therapy goes to trial for multiple sclerosis

With a number of new drugs approved, the last decade has seen tremendous progress in the treatment of multiple sclerosis (MS). All current drugs work by decreasing inflammation through modulation of the immune system, and while they have been very efficient in reducing the rate of relapses, their impact on the chronic disease course is unknown. A current thought in the MS therapeutics community is that drugs that enhance remyelination may be more effective in reducing long-term disability. This hypothesis is based on the observation that disability in MS increases with age as the capacity of oligodendrocytes to remyelinate decreases.¹ Additional support for this hypothesis comes from extensive preclinical studies showing that promoting remyelination either by transplanting myelinating stem cells^2,3 or by pharmacologic enhancement of endogenous myelination processes reduces clinical severity in animal models of MS.^4,5 In this issue of Neurology® Neuroimmunology & Neuroinflammation, Tran et al.⁶ take this possibility to its exciting next step by reporting the results of the phase I clinical trial of the first drug aimed at promoting myelin repair.