New targets and therapeutics for neuroprotection, remyelination and repair in multiple sclerosis

- Pablo Villosada & Lawrence Steinman


Introduction: Immunotherapies for multiple sclerosis (MS) significantly decrease the risk of new relapses. However, the chronic compartmentalized inflammation and neurodegeneration that define progressive MS are not prevented by these therapies and so significant damage to the brain and spinal cord and resulting disability ensues. Hence, the possibility of combining current immunotherapies with neuroprotective, remyelinating or regenerative therapies should be pursued.

Areas covered: This article sheds light on neuroprotective, remyelination and neurorepair strategies for MS, the numerous mechanisms for therapeutic targeting and the new candidates for combination therapies. We searched PubMed for articles with the terms, ‘neuroprotection’, remyelination’ or ‘regeneration’ and ‘therapies’ or ‘drugs’ and ‘Multiple Sclerosis’.

Expert opinion: An enriched understanding of the neurobiology and molecular changes that are activated by inflammatory CNS damage will provide new opportunities for the identification of neuroprotective, remyelinating and regenerative therapies. Success will depend on the improvement of CNS drug delivery, the identification of new predictive biomarkers, the optimization of clinical trials by assessment of the damage to the visual pathway and the testing of novel therapies in acute optic neuritis.

Article Highlights

  • The prevention of disabilities associated with MS progression is a significant unmet need for patients.
  • Neuroprotection, remyelination and regeneration therapies are being pursued, along with immunomodulatory drugs and combination therapies.
  • Many targets have been proposed for neuroprotection, but their efficacy in clinical trials has not been proven. An enhanced understanding of neurobiology and novel technologies for CNS penetration pave the way for the development of effective therapies to prevent CNS damage.
  • Remyelinating therapies have been improved by our understanding of oligodendrocytes and their precursors.
  • The assessment of the damage to the visual pathway during clinical trials and the testing of novel therapies for acute optic neuritis offers opportunities for the development of new therapeutics.


Date publication: April 29, 2020

Villoslada P, Steinman L. New Targets and Therapeutics for Neuroprotection, Remyelination and Repair in Multiple Sclerosis. Expert Opin Investig Drugs. 2020 Apr 29;1-17.