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Results Published of Phase I Mesenchymal Stem Cell Study

February 12, 2018

SUMMARY
  • Investigators from the Tisch MS Research Center in New York, have published results from a phase I, open label clinical trial involving 20 people with progressive MS, using individuals’ own mesenchymal stem cells (from bone marrow) to derive more specific stem cells called “neural progenitor cells.” These cells were used for three separate intrathecal injections (into the space around the spinal cord).
  • The team reported mild to moderate headache and fever as the most common side effects, and for the primary outcome of the study, which was safety, they reported no serious adverse events.
  • Although the study was not designed to determine potential benefits of this therapy, the team reported clinical observations including some improvements in EDSS scores, strength in some muscle groups, walking speeds and bladder symptoms.
  • Larger, longer-term, controlled studies are needed to determine the safety and effectiveness of using this approach to treat MS. The team is planning a larger phase II trial.
  • The team (Violaine K. Harris, PhD, Saud A. Sadiq, MD, and colleagues at Tisch Multiple Sclerosis Research Center of New York, NY) reports the results in the online journal EBiomedicine (Published online: February 3, 2018).
 
DETAILS
Background: Stem cell therapy is any treatment that uses or targets stem cells, which are the types of cells that can specialize into many different types of cells in our bodies. “Mesenchymal stem cells” are adult stem cells found in several places in the body, including the bone marrow, skin and fat tissue. These cells are being tested in clinical trials in MS. A person’s own mesenchymal stem cells are isolated from the bone marrow or blood stream and multiplied in the lab, and then re-introduced in greater numbers into their body.
 
This Study: This team conducted a clinical trial involving 20 people with progressive MS. The study was “open label,” meaning both the researchers and the participants were aware of the treatment and no placebo group was used. Participants’ own mesenchymal stem cells (from bone marrow) were extracted to derive more specific stem cells called “neural progenitor cells.” The cells were expanded in the lab and then injected into the space around the spinal cord in three separate doses, three months apart.
The primary endpoint for the study was the treatment’s safety and tolerability. Other endpoints, including EDSS (a standard way of measuring physical disability in MS), were also measured.
 
Results: The team reported mild to moderate headache and fever as the most common side effects, and no serious adverse events. Although the study was not designed to determine potential benefits of this therapy, the team reported trends for improvements in EDSS scores, strength in some muscle groups, walking speeds and bladder symptoms compared to before the treatment began. No improvements were observed in upper limb function or cognitive function. In addition, there were no changes in disease activity on MRI scans during the study.
 
The team (Violaine K. Harris, PhD, Saud A. Sadiq, MD, and colleagues at Tisch Multiple Sclerosis Research Center of New York, NY) reports the results in the online journal EBiomedicine (Published online: February 3, 2018).
 
Next Steps:  In their paper, the researchers noted that “Although this open-label single arm study was not designed to establish efficacy, we report our observations.” Larger, longer-term, controlled studies are needed to determine the safety and effectiveness of using this approach to treating MS. The team is planning a larger phase II trial. The National MS Society is in discussions with the team to determine how best to support this important work.
 
Read More:
Read more about Stem Cells in MS

About Multiple Sclerosis

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system that disrupts the flow of information within the brain, and between the brain and body. Symptoms range from numbness and tingling to blindness and paralysis. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are leading to better understanding and moving us closer to a world free of MS. Most people with MS are diagnosed between the ages of 20 and 50, with at least two to three times more women than men being diagnosed with the disease. MS affects more than 2.3 million people worldwide.

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