New clinical trial results supply evidence that high-dose immunosuppressive treatment followed by transplantation of a person’s very own blood-forming stem cells can cause sustained remission of relapsing-remitting several sclerosis (MS), an autoimmune illness in which the immune system attacks the nervous system.
Five years after receiving the therapy, called high-dose immunosuppressive therapy plus autologous hematopoietic cell transplant (HDIT/HCT), 69 percent of trial individuals had survived without experiencing development of disability, relapse of MICROSOFT symptoms or new brain skin lesions. Notably, participants did not take any kind of MS medications after receiving HDIT/HCT. Other studies have indicated that now available MS drugs have lower success.
The trial, called HALT-MS, had been sponsored by the National Institute associated with Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, and conducted by the NIAID-funded Immune Tolerance Network (ITN). The scientists published three-year results from the study within December 2014, and the final five-year results appear online Feb. one in Neurology, the medical diary of the American Academy of Neurology.
âThese 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,â said NIAID Director Anthony S. Fauci, M. D. âThese encouraging results support the development of a large, randomized trial to directly compare HDIT/HCT to standard of care for this often-debilitating disease.â
MS symptoms vary widely and may even include motor and speech problems, weakness, fatigue and chronic discomfort. The most common form of MS is usually relapsing-remitting MS, which is characterized by intervals of mild or no symptoms interspersed with symptom flare-ups or relapses. Over years, the disease can get worse and shift to a progressive type.
In HALT-MS, researchers examined the safety, efficacy and durability associated with HDIT/HCT in 24 volunteers old 26 to 52 years along with relapsing-remitting MS who, despite using clinically available medications, experienced energetic inflammation, evidenced by frequent serious relapses, and worsened neurological impairment.
The experimental treatment aims in order to suppress active disease and prevent additional disability by removing disease-causing tissues and resetting the immune system. During the process, doctors collect a participantâs blood-forming stem cells, give the participant high-dose chemotherapy to deplete the immune system, plus return the participantâs own originate cells to rebuild the immune system. The treatment carries some risks, and several participants experienced the expected unwanted effects of HDIT/HCT, such as infections. Three participants died during the study; nothing of the deaths were related to the research treatment.
Five years after HDIT/HCT, most trial participants remained within remission, and their MS got stabilized. In addition, some individuals showed improvements, such as recovery associated with mobility or other physical features.
âAlthough 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,â said Richard Nash, Meters. D., of Colorado Blood Cancer Institute and Presbyterian-St. Lukeâs Hospital. Dr. Nash served as primary investigator of the HALT-MS study.
âIf 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,â said Daniel Rotrosen, M. Deb., director of NIAIDâs Division associated with Allergy, Immunology and Transplantation.
This work was sponsored by NIAID, NIH, and conducted by the ITN under award number AI109565 plus NIAID-funded statistical and clinical matching centers under award number AI117870. The ClinicalTrials. gov identifier for your Phase 2 study High-Dose Immunosuppression and Autologous Transplantation for Multiple Sclerosis (HALT-MS) is NCT00288626.
RA Nash et al. High-dose immunosuppressive therapy and autologous HCT for relapsing-remitting MS. Neurology DOI: 10. 1212/WNL. 0000000000003660 ((****************