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Immunosuppressive therapy, stem cell transplant can lead to long-term remission of MS

Five years after trial participants with multiple sclerosis received high-dose immunosuppressive therapy followed by autologous hematopoietic cell transplant, researchers found that nearly 70% survived without experiencing progression of disability, relapse of symptoms or new brain lesions, according to recent clinical results.

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

Led by Richard Nash, MD, from the Colorado Blood Cancer Institute and Presbyterian-St. Luke’s Hospital, a group of researchers from the NIAID-funded Immune Tolerance Network published the results of the trial, HALT-MS, after 3 years in December 2014 and again after 5 years on Feb. 1, 2017. With this experimental treatment, they sought to control active multiple sclerosis disease and prevent further disability by eliminating disease-causing cells and resetting the immune system.

In their study, investigators assessed the safety, efficacy and durability of high-dose immunosuppressive therapy/HCT in 24 participants aged 26 to 52 years with relapsing-remitting MS, who experienced frequent severe relapses and worsened neurological disability despite taking available MS medications. They collected a participant’s blood-forming stem cells during the procedure, then gave the participants high-dose chemotherapy to deplete their immune system before returning their own stem cells to rebuild the immune system.

Five years later, most participants remained in remission, and had stable MS. Of the remaining 21 participants, 69% survived without suffering disability progression, relapse or new lesions in the brain without the aid of MS medications after receiving the stem cell treatment. The researchers found that some even showed neurological improvements, such as recovery of mobility. Currently available MS drugs have shown lower success rates than high-dose immunosuppressive therapy/HCT. As expected, infection was a common adverse effect of the treatment.

“Although further evaluation of the benefits and risks of [high-dose immunosuppressive therapy]/HCT is needed, these 5-year results suggest the promise of this treatment for inducing long-term, sustained remissions of poor-prognosis relapsing-remitting MS,” Nash said in the release. – by Savannah Demko

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