The journal JAMA Neurology recently published findings from a cutting-edge phase 2 study, which demonstrated that a course of immunosuppressive therapy, followed by an autologous stem cell transplant, is remarkably effective at achieving permanent remission of multiple sclerosis even years later.
The goal of the study (called the Hematopoietic Cell Transplantation for Relapsing-Remitting Multiple Sclerosis or HALT-MS) was to recreate the patients’ immune systems from the ground up. For this purpose, 24 volunteers with relapsing-remitting form of the disease were given high-dose immunosuppressive therapy (HDIT) which eradicated their immune systems.
Then they received a course of hematopoietic stem cells obtained from their own blood. This, the scientists hoped, would rebuild the subjects’ immune systems with brand new cells, unprejudiced against the myelin sheath which covers nerve cells in the brain and spinal cord.
The results indicate that of the 24 patients who received HDIT, followed by the transplant, the overall rate of event-free survival (defined as survival without death or disease due to neurologic complications, clinical relapse or new lesions) was 78.4 per cent.
Three years after the procedure, over 90 per cent of the patients remained progression-free, while 86.3 per cent experienced no clinically significant relapses. Some of the patients even regained a measure of lost neurologic functionality, leading them to score lower on the Expanded Disability Status Scale (EDESS).
“In the present study, HDIT/HTC [hematopoietic cell transplant] induced remission of MS disease activity up to three years in most participants,” noted study authors. “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.”
Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases, had this to say: “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.”
Fauci is positive that if these findings are confirmed, HDIT/HCT may become a potential therapeutic option, which would be of exceptional importance to those MS sufferers who failed to derive any benefit from conventional immunosuppressive treatment.
Full text of the study can be accessed for free on the JAMA Neurology website.