Three-year outcomes from an ongoing clinical trial suggest that high-dose immunosuppressive therapy followed by transplantation of a person's own blood-forming stem cells may induce sustained remission in some people with relapsing-remitting multiple sclerosis (RRMS). The trial is funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, and conducted by the NIAID-funded Immune Tolerance Network.
Three years after the treatment, called high-dose immunosuppressive therapy and autologous hematopoietic cell transplant or HDIT/HCT, nearly 80 percent of trial participants had survived without experiencing an increase in disability, a relapse of MS symptoms, or new brain lesions. Investigators observed few serious early complications or unexpected side effects, although many participants experienced the expected side effects of high-dose immunosuppression, including infections and gastrointestinal problems. The three-year findings were recently published online in JAMA Neurology.
“These promising results support the need for future studies to further evaluate the benefits and risks of HDIT/HCT and directly compare this treatment strategy to current MS therapies,” says NIAID Director Anthony S. Fauci, MD. “If the findings from this study are confirmed, HDIT/HCT may become a potential therapeutic option for people with this often-debilitating disease, particularly those who have not been helped by standard treatments.”
In the study, researchers tested the effectiveness of HDIT/HCT in 25 volunteers with RRMS who had relapsed and experienced worsened neurological disability while taking standard medications. Doctors collected blood-forming stem cells from participants and then gave them high-dose chemotherapy to destroy their immune systems. Stem cells were returned to the participants to rebuild and reset their immune systems.
Researchers plan to follow participants for a total of five years, recording all side effects associated with the treatment. Final results promise to help inform the design of larger trials to further evaluate HDIT/HCT in people with MS.
Read the article at the JAMA Neurology website
