High-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HDIT/HCT) has had “highly promising” results for patients with relapsing-remitting multiple sclerosis (MS), according to researchers from the HALT-MS trial. In the 5-year study, 69% of 24 participants survived without progression of disability, relapse, or new brain lesions, despite not taking MS medications.
Findings published at the 3-year mark were encouraging. The event-free survival rate was 78%. The extended findings suggest that “onetime treatment with HDIT/HCT may be substantially more effective than long-term treatment with the best available medications” for these patients, said NIAID Director Anthony Fauci, MD.
The treatment “resets” the immune system, the researchers say. First, doctors collect the patient’s blood-forming stem cells, then give the patient chemotherapy to deplete the immune system. Finally, the doctors return the patient’s stem cells to rebuild the immune system.
Adverse events were consistent with those routinely observed after HDIT/HCT. Adverse effects recorded at 4 and 5 years were not related to the transplant and were not considered severe. Three patients died, but their deaths were not related to the study treatments.
Five years later, most trial participants remained in remission and stabilized, and some showed improvements, such as recovering mobility.