Long-Term Remission Seen with Experimental Stem Cell Treatment for Multiple Sclerosis

A majority of patients with multiple sclerosis treated with chemotherapy and a stem cell transplant remained in remission for at least 5 years.

Researchers in a new study found that a new stem cell transplant therapy may prompt sustained remission in patients with multiple sclerosis (MS).

Findings from the study, published by Neurology, suggest that treatment with high-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HDIT/HCT) can induce prolonged remission in patients with relapsing-remitting multiple sclerosis.

Five years after initial HDIT/HCT treatment, 69% of participants had not experienced disease progression, as measured by disability, symptoms, and brain lesions. No study participants were not treated with any MS treatments after undergoing HDIT/HCT.

Currently, other MS treatments have lower success rates, according to the study.

“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 National Institute of Allergy and Infectious Disease Director Anthony S. Fauci, MD. “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.”

Patients with MS can experience a wide range of symptoms, including weakness, fatigue, chronic pain, and speech difficulties. Relapsing-remitting MS is the most common form, and is characterized by periods of remission followed by periods of relapse.

Included in the HALT-MS trial were 24 patients with relapsing-remitting MS who experience frequent relapses due to active inflammation, and worsened neurological disability despite receiving treatment with approved drugs, according to the study.

The HDIT/HCT treatment suppressed disease activity and prevents disability through removal of immune cells, and create a new immune system.

For this procedure, physicians obtain blood-forming stem cells from the patient, administer high-dose chemotherapy, and return the patient’s stem cells to rebuild the immune system, according to the study.

However, this approach carries risks, and many patients experienced side effects, such as infections.

After 5 years, a majority of patients remained in remission, and some even showed improvements, such as regained mobility or other physical capabilities, according to the study.

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

If similar results are seen in future studies, patients with this form of MS may have an effective treatment option that can slow or delay disease progression.

“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 National Institute of Allergy and Infectious Disease’s Division of Allergy, Immunology and Transplantation.