Immunosuppressive Therapy, Stem Cell Transplant Induces Remission in Relapsing-Remitting Multiple Sclerosis

One round of investigational combination treatment may be more effective than long-term treatment for multiple sclerosis.

Treatment with high-dose immunosuppressive therapy may induce sustained remission in patients with relapsing-remitting multiple sclerosis (RRMS), according to a study published in Neurology.

Five years after patients in the HALT-MS trial received high-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HDIT/HCT), the results showed that 69% had survived without experiencing progression of disability, relapse of MS symptoms, or new brain lesions.

“These extended findings suggest that one-time treatment with HDIT/HCT may be substantially more effective than long-term treatment with the best available medications for people with a certain type of MS,” said Anthony S. Fauci, MD, director of the National Institute of Allergy and Infectious Diseases (NIAID). “These encouraging results support the development of a large, randomized trial to directly compare HDIT/HCT to standard of care for this often-debilitating disease.”

The investigators examined the safety, efficacy, and durability of HDIT/HCT in 24 volunteers aged 26 to 52 years with RRMS. Although the participants had previously been on medication, they were still experiencing active inflammation made evident through frequent severe relapses and worsening neurological disability, according to the study.

For the study, blood-forming stem cells were collected from the participants, who were then administered a high-dose of chemotherapy to deplete the immune system. The patients’ own stem cells were then transplanted back into their bodies to rebuild the immune system.

Adverse events, such as infections, were both minimal and expected. Three participants died during the study, however, none of the deaths were due to the treatment.

After a follow-up of 5 years post treatment, a majority of the participants remained in remission and their disease stabilized, according to the authors. Some of the participants saw improvements in their physical capabilities, such as recovery of mobility.

“Although further evaluation of the benefits and risk of HDIT/HCT is needed, these 5-year results suggest the promise of this treatment for inducing long-term, sustained remission of poor prognosis relapsing-remitting MS,” said principal investigator Richard Nash, MD.

The Multiple Sclerosis Foundation estimates more than 400,000 individuals the United States have MS, followed by approximately 2.5 million people worldwide. RRMS is the most common form of MS, and is characterized by periods of mild or no symptoms, and symptom flare-ups or relapses.

“If these findings are confirmed in larger studies, HDIT/HCT may become a potential therapeutic option for patients with active relapsing-remitting MS, particularly those who do not respond to existing therapies,” said Daniel Rotrosen, MD, director of NIAID’s Division of Allergy, Immunology and Transplantation.