Breakthrough drug may promote myelin repair

A new drug may be able to help the body replace lost myelin in people with multiple sclerosis (MS), according to recent research.

The new therapy, called PIPE-307, targets an elusive receptor on certain cells in the brain that prompts them to mature into myelin-producing oligodendrocytes. Once the receptor is blocked, the oligodendrocytes spring into action, wrapping themselves around the axons to form a new myelin sheath.

It was crucial to prove that the receptor, known as M1R, was present on the cells that can repair damaged fibres. Scientists originally worked this out by using a toxin found in green mamba snake venom.

The work was undertaken by scientists at UC San Francisco and appears in Proceedings of the National Academy of Sciences, capping a decade of work by UCSF scientists Jonah Chan, Ph.D., and Ari Green, MD

In 2021, PIPE-307 passed a Phase I clinical trial, demonstrating its safety. It is currently being tested in MS patients in Phase II.

If it succeeds, it could transform how MS is treated.