Rapid Progress in Research on Cell-Based Therapies – Including Trial Results from Cleveland Clinic

Cell-based therapies that have the potential to both stop MS in its tracks and restore lost function are getting a lot of attention at this week’s ACTRIMS-ECTRIMS meeting. Stem cells can be found in different tissues and organs and under the right circumstances, can transform into specific types of cells when needed to repair injuries. These stem cells represent a promising area of research for both slowing MS activity and for repairing the brain’s myelin that has been damaged by MS.

But – Which cells? Given how? To whom? How often? – are some of the big questions that need to be answered with ongoing research. And while this approach has much promise, it is still experimental and not yet proven to work. Read more about different approaches to research into stem cells for MS.

One approach is to use these cells to replace the cells that make myelin – the protective cover on nerve wires which is damaged during MS – and nerve cells that have been destroyed.

Finding ample and uncontroversial sources of stem cells would help this effort. One exciting avenue being explored is to take samples of a person’s skin or other adult cells and reprogram them to become stem cells. These cells are called “induced pluripotent stem cells” or iPSC. Dr. Giulia Mallucci of the United Kingdom and Italy described a new process that could speed up research on this approach. The current approach involves reprogramming the skin cells via many steps and months to take them backward to become unspecialized stem cells, then reprogramming them again to force them to become specialized brain stem cells. In the new approach tested by this team, they turned mouse skin cells directly into specialized brain cells in one step.

The team had success with this new technique treating mice that had MS-like disease. (Read the abstract.) Other scientists are also pursuing this exciting area of research, including a group from Stanford University that will begin a Society-sponsored study in October examining the ability of human iPSC cells to repair the nervous system.

Until recently, scientist thought the transplanted stem cells migrated to the nervous system and directly replaced the lost myelin. But Dr. Stefano Pluchino of the U.K. presented data this week showing that instead, the transplanted cells actually provided signals that stimulated natural repair of other cells. As research continues to translate and understand these signals, it will open up the future possibility of creating medicines based on the signals without having to actually transplant cells. 

It was great to hear the much-anticipated first results from a small, Phase 1 clinical trial at the Cleveland Clinic involving people with relapsing MS. This trial tested the ability of people’s own stem cells taken from their bone marrow, called mesenchymal stem cells, to inhibit immune mechanisms and to augment the body’s natural tissue repair processes after they were expanded and re-injected. An early trial like this can’t give a clear read about benefit, but they did report finding no serious safety concerns or any uptick in disease activity. They are still evaluating their results, but Dr. Cohen suggested that so far it’s a green light to the next step – designing a larger, controlled study of this approach. Read the conference abstract.)

Another approach using stem cells is called autologous hematopoietic (blood cell-producing) stem cell transplantation" – or (HSCT). This experimental procedure is being explored in attempts to “reboot” the immune system and hopefully stop MS attacks. (Read an interview with Dr. Richard Burt about his HSCT clinical trial.) Dr. Carmen Alcalá of Valencia, Spain, reported that in their hands, this procedure was more effective against disease activity in people with active relapsing MS, rather than secondary-progressive MS. (Read the conference abstract.)

This is just a small sample of more than 9,000 research presentations being made this week at this incredible meeting. Stay tuned for more updates! 

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Bruce

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.