MS Stem Cell Therapies Show Promise, But More Work Is Needed, Researcher Tells ACTRIMS 2016

Dr. Andrew Goodman of the University of Rochester discussed the latest research and perspectives on stem cell strategies for people with multiple sclerosis (MS), saying in a presentation at the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum 2016 that such therapies, while promising, are not yet ready for widespread clinical use.

New therapies for MS patients that might both restore the immune system and protect the nervous system are greatly needed. Dr. Goodman, a professor of Neurology, chief of the Neuroimmunology Unit, and director of the Multiple Sclerosis Center at the university, reviewed current stem cell therapy approaches, including the use of “hematopoietic stem cell reconstitution following immunoablation, mesenchymal stem cells, and oligodendrocyte precursor cells” in a presentation titled “Stem Cell Therapy for MS.”

His presentation at the forum, which ran Feb. 18–20 in New Orleans, Louisiana, offered:

• an overview of the biology of stem cells
• an understanding of the distinct goals of the various cell-based therapeutic strategies under study as potential MS therapies
• an appreciation for the potential benefits, risks, and methodological uncertainties associated with stem cell transplantation

In MS, the immune system attacks the body’s own myelin, the fatty covering that insulates nerves and facilitates nervous system communication. Stem cell therapies may be directed at replacing the faulty immune system, at increasing levels of oligodendrocytes (the cells that create myelin), or even at increasing neurons that could be damaged in MS.

Numerous issues still need to be answered regarding the safety and effectiveness of stem cell therapy, Dr. Goodman said in the presentation, such as understanding whether immune system restoration via stem cells should be combined with transplantation of nervous system cells, specifically oligodendrocytes and neurons. Goodman also considered whether autologous (self-derived) stem cells should be used, or if they may have intrinsic deficiencies, since they come from a person with a neurodegenerative disease.

According to Dr. Goodman’s PowerPoint presentation at ACTRIMS, which was made available, he concluded that, overall, “Several types of cell-based therapeutic strategies are under investigation, with different risks, benefits, and goals. Some of these strategies show promise but significant methodological questions need to be answered. Stem cell transplantation is not yet appropriate for general use to treat MS.”

One issue Dr. Goodman highlighted was the need for extensive genetic screening of cells to assure that possible cancer-causing mutations are not introduced.