Stem Cell Transplants Result in Sustained Multiple Sclerosis Remission

Most patients treated with high-dose immunosuppressive therapy and who had their own hematopoietic stem cells transplanted experienced sustained remission after 3 years.

Most patients treated with high-dose immunosuppressive therapy and who had their own hematopoietic stem cells transplanted experienced sustained remission after 3 years.

Among a limited number of multiple sclerosis (MS) patients treated with high-dose immunosuppressive therapy (HDIT) before being transplanted with their own hematopoietic stem cells, most had improvements in neurological function and sustained remission of active relapsing-remitting MS (RRMS) after 3 years.

Autologous hematopoietic cell transplant (HCT) has previously been evaluated for the treatment of MS to potentially remove disease-causing immune cells while resetting the immune system, according to a study published online by JAMA Neurology in December 2014.

The study found that in 24 MS patients who received HDIT/HCT, after 3 years the overall rate of event-free survival was 78.4%, defined as survival without death or loss of neurologic function, clinical relapse, or newly observed lesions.

Rates of progression-free survival were 90.9% and clinical relapse-free survival were 86.3% after 3 years. Adverse events were found to be consistent with the anticipated toxic effect of HDIT/HCT, while there were no acute treatment-related neurologic adverse events reported. Additionally, there were improvements found in neurologic disability, quality-of-life, and functional scores.

"In the present study, HDIT/HCT induced remission of MS disease activity up to three years in most participants,” the authors wrote. “It may therefore represent a potential therapeutic option for patients with MS in whom conventional immunotherapy fails, as well as for other severe immune-mediated diseases of the central nervous system. Most early toxic effects were hematologic and gastrointestinal and were expected and reversible. Longer follow-up is needed to determine the durability of the response.”

An accompanying editorial noted that the study leaves little doubt that high-dose immunotherapy can lead to substantial suppression of inflammatory disease activity in MS patients with active disease in the short term.

“There is some evidence for long-term suppression of MS. Lessons have been learned about how treatment-related morbidity and mortality may be reduced,” M. Mateo Paz Soldán, MD, PhD, and Brian G. Weinshenker, MD, wrote in the editorial. “However, deaths have occurred, even in small studies, and aggressive regimens have resulted in lymphomas associated with Epstein-Barr virus."

The editorial concluded that in light of clinical or radiologic evidence of relapse that trumps immunologic evidence of immune reset, and the study raises concern that these end points have not been adequately achieved.

“The jury is still out regarding the appropriateness and indication of HCT for MS,” they added.