Stem Cell Treatment Looks Promising for MS

A recent study describes the first treatment that completely halts all detectable inflammatory activity in the central nervous system (CNS) in patients diagnosed with multiple sclerosis (MS), for a prolonged period and without the use of disease modifying drugs. The study, published in The Lancet online on June 9, 2016, was conducted by Harold Atkins, MD, of the Ottawa Hospital Research Institute in Ottawa, Canada.

This was a phase 2, single-arm trial, involving patients diagnosed with MS with a poor prognosis, across three hospitals in Canada. There were 24 participants between the ages of 18 and 50 years. The participants completed a transplant conditioning program, and then an autologous CD34 hemopoietic stem-cell graft was infused. Following the infusion, the participants were admitted into a stem-cell transplantation ward where they were assessed for treatment-related toxic effects.

The transplantations occurred between October, 2001 and December, 2009. “The primary outcome, multiple sclerosis activity free survival at 3 years after transplantation was 69.9%,” said the authors. They add, “The treatment abolished all clinical and radiological hallmarks of disease-related CNS inflammation, without the use of disease-modifying drugs, during the entire extended follow-up period.”

In addition to halting inflammation, the researchers report, “Many of our patients recovered substantially, indicating that repair mechanisms were still active and might have been suppressed by ongoing inflammations.” Despite the positive outcome of this study, the researchers urge caution, saying that experience in selecting the best candidates and specialized care is necessary for the best outcomes in autologous hemopoietic stem-cell transplantation.

Although this study involved a small cohort without a control group, the researchers conclude, “For a substantial number of patients whose disease was not well-controlled with disease-modifying drugs, this procedure led to neurological improvement and long-lasting remission free of ongoing treatment.”