Five-Year Study Stem Shows Cell Transplants May Induce Long-Term Remission of Multiple Sclerosis

          The five-year results of the HALT-MS clinical trial looking at the effectiveness of high-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HCT — see box) showed that 69% of trial participants did not experience 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 National Institute of Allergy and Infectious Diseases (NIAID) Director Anthony 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.”


         HALT-MS was sponsored by NIAID, part of the National Institutes of Health, and conducted by the NIAID-funded Immune Tolerance Network (ITN). Researchers tested the safety, efficacy (how well the treatment works) and durability (how long the results last) of HDIT/HCT in 24 volunteers aged 26 to 52 years with relapsing-remitting MS (RRMS) who, despite taking clinically available medications, experienced active inflammation, evidenced by frequent severe relapses, and worsened neurological disability.


         Five years after HDIT/HCT, most trial participants had no relapses or progression, and their MS had stabilized. In addition, some participants showed improvements, such as recovery of mobility or other physical capabilities. While three participants died during the study, none of the deaths were considered related to the study treatment.





         “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 Richard Nash, MD, of Colorado Blood Cancer Institute and Presbyterian-St. Luke’s Hospital. Nash served as principal investigator of the HALT-MS study.


         Daniel Rotrosen, MD, director of NIAID’s Division of Allergy, Immunology and Transplantation says he hopes that, “if these findings are confirmed in larger studies, HDIT/HCT may become a potential therapeutic option for patients with active RRMS, particularly those who do not respond to existing therapies.”


Autologous Hematopoietic Cell Transplant


The HDIT/HCT The procedure has three steps:


  • First: Doctors collect a participant’s blood-forming stem cells
  • Second: The participant receives high-dose chemotherapy to deplete the immune system
  • Third: The stem cells collected in step 1 are returned to the participant to rebuild the immune system.


The treatment carries some risks, and many participants experienced the expected side effects of HDIT/HCT, such as infections, pulmonary (lung), or cardiac (heart) complications.


Full HALT-MS results here: