MS Repair: Rapidly moving out of the lab and into people

When I was in the lab, my research focused largely on using mouse models of MS to test concepts that couldn’t be explored in people yet. Studies presented at this week’s AAN meeting are making it clear how far research has advanced since that time. Case in point: I never would have thought just a few years ago that I would be writing about myelin repair in people, not mice.

So it was pretty exciting to see a presentation Wednesday evening showing the first results of a clinical trial of the myelin repair strategy called anti-LINGO, which is being developed by Biogen. This study involved giving this IV infusion or a placebo every 4 weeks for 20 weeks to 82 people who had experienced their first episode of optic neuritis. Optic neuritis involves inflammation of the nerve that connects to the eye, and it’s often one of the first signs of MS.

The study measured the speed at which electrical signals traveled along the optic nerve before and after 24 weeks of treatment. The investigators found that those who had been given Anti-LINGO therapy had faster nerve signals after treatment, compared to those who were given the placebo. This speeding up of signals is thought to be a consequence of myelin repair. They didn’t find differences between the groups in terms of vision, and the therapy seemed to be well tolerated except for some infusion reactions. (Abstract S56.008)

This is exciting stuff! But it is still early. Benefits will need to be demonstrated in larger and longer clinical trials before the FDA will consider this treatment for approval.

I’m now really looking forward to seeing the results from a parallel study of anti-LINGO in people with relapsing MS. This study will add important new information on the potential of anti-LINGO for restoring myelin and function for people with MS.

We’ve talked before about the potential of various kinds of stem cells to stop disease activity and possibly repair myelin. There have been a few updates at AAN about trials of adult mesenchymal stem cells, which are present in many tissues of the body. These trials isolate a person’s own mesenchymal cells from the bone marrow or blood stream, which are then multiplied in the lab, and then re-introduced in greater numbers into the body.

Previously, a small phase I clinical trial at Cleveland Clinic tested the ability of mesenchymal stem cells to both inhibit immune mechanisms and to augment intrinsic tissue repair processes in people with relapsing forms of MS. This study was led by Dr. Jeffrey A. Cohen and supported by the Congressionally Directed Medical Research Programs. This trial was designed to evaluate safety and not designed to determine benefits, and preliminary results were presented last year suggesting that this approach was safe.

The team has done some further probing to try to get at the question of whether these cells stimulated myelin repair and protected nerve fibers. We think degenerating nerve fibers are driving disability progression, so if this works, it could potentially slow down or stop progression. They looked at specific brain lesions before and after the infusions of stem cells using advance imaging called DTI. They reported that compared to before treatment, measures of axon loss stabilized, hinting that the cells may have stimulated repair and/or protected nerve tissue. A follow up clinical trial, which is now in planning stages, should shed more light on potential benefits of this exciting approach. (Abstract P1.152)

Researchers from the Tisch MS Research Center of New York presented some early findings from their trial of mesenchymal stem cells. These investigators are purifying and expanding neural progenitor cells from the bone marrow and then injecting them into the spinal fluid. In an interim update from this ongoing study, they reported no safety issues after treating 7 participants with one dose and 2 participants with three doses. (Abstract P7.200)

I’m proud that the National MS Society made strategic investments to encourage research in the area of nervous system repair and protection, and it’s really gratifying to see promising early results with potential implications for people with MS. We’ll be keeping a close eye on these and other studies that are the translation of many long years of basic lab work.  

Anyone can get a preview of the summaries, or abstracts, of presentations to be given at the Academy’s Annual Meeting at this link. Registration is necessary, but is free.

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Bruce Bebo, PhD

Bruce Bebo, PhD, is Executive Vice President, Research at the National MS Society, and was previously a research immunologist focusing on the influence of sex hormones on MS. He is a driven and passionate Society volunteer, successful fundraiser and advocate, fueled in part by the fact that his mother had MS.