BY FRAN KRITZ
High doses of chemotherapy drugs followed by an infusion of a patient's own stem cells may result in remission for people with remitting-relapsing multiple sclerosis (MS), according to a study published online in Neurology on February 1, 2017.
The new results are from a small clinical trial, called HALT-MS, which was sponsored by the National Institute of Allergy and Infectious Diseases (NIAID) at the National Institutes of Health (NIH). Earlier results were published in the third year of the clinical trial while this new study looked at five years of data. The clinical trial included patients with relapsing-remitting MS, which is the most common form of the disease and often involves through long periods with no or only mild symptoms and occasional flare-ups or relapses. Over years, though, symptoms can worsen and progress.
Twenty four patients, ages 18 to 60 (17 women and 7 men) who had not responded to other MS medications, were included in the trial, which involved high-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HDIT/HC.) The high-dose chemotherapy weakens the immune system while transfusions of the patient's own blood stem cells can "reset" the immune system and potentially knock out MS.
At the end of the trial, 91 percent of patients had no disease progression, 87 percent did not relapse, and 86 percent had no signs of new lesions (scars that can indicate MS) on their brain or spinal cord. "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," says NIAID director Anthony S. Fauci, MD.
Some Adverse Events
Three deaths were reported among the trial participants, but they were unrelated to the transplants. Two were the result of MS progression and one was due to cardiovascular disease. The most common side effect of the treatment was infection.
More Studies Needed
More trials are needed before this can be considered a treatment for relapsing-remitting MS, say experts. "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 in patients with relapsing-remitting MS, who have a poor prognosis," says Richard Nash, MD, a hematologist with the Colorado Blood Cancer Institute and the principal investigator of the HALT-MS study.
The new clinical trial is "an important study" that "contributes to the accumulating knowledge of the possible benefits and risks of…stem cell transplantation in relapsing MS," says Bruce Bebo, PhD, executive vice president of research at the National MS Society. He added that "larger, well-controlled trials are needed to better understand who might benefit from this procedure and how it compares to the benefits of powerful immune-modulating therapies now available."