![]() Patients in the drug therapy group had worsening scores on the Expanded Disability Status Scale (EDSS) - a method of quantifying disability in patients with MS - while patients in the HSCT group actually saw improvements in their scores. At last follow-up, disease progression occurred in 34 patients in the drug therapy group, but in just three patients in the HSCT group. In the trial, 110 patients were randomized to receive either HSCT or the traditional drug therapy. Previously, HSCT found success in non-randomized patients in trials at Northwestern and elsewhere - including in Sweden and Canada, and at the Fred Hutchinson Cancer Research Center and Duke University - but the current study is the first fully-randomized trial that tested relapse-remitting MS (RRMS) against the current treatment, Burt said. “When the immune system restarts in a non-inflammatory environment, it appears to default back to tolerance and stops the attack, allowing, depending on the stage of disease, for the body to repair itself to some extent,” Burt said. This produces new immune cells that several previous studies suggest may reset the immune system. HSCT, however, relies on a different strategy: MS patients are given five days of immune-specific drugs to shut down their self-destructive immune system, then stem cells are transplanted into the patient’s blood. The current disease-modifying therapies are intense and personalized drug regimens that patients must take indefinitely, even as its effectiveness can eventually fade, according to Burt. It damages the nerve cells in the brain and spinal cord, causing symptoms that range from fatigue to paralysis throughout life. MS is an immune-mediated disorder of the central nervous system often arising in young adulthood. “Now we can work on improving it, refining it and making it safer.” “This opens the door for this therapy,” Burt said. ![]() The success of this trial serves as a proof-of-concept for the treatment, according to Richard Burt, MD, chief of Immunotherapy and Autoimmune Diseases in the Department of Medicine and lead author of the study. The process, called hematopoietic stem cell transplantation (HSCT), temporarily shuts down and reboots patients’ immune systems, allowing the body to rebuild damaged nerve cells. ![]() Richard Burt, MD, chief of Immunotherapy and Autoimmune Diseases in the Department of Medicine, was lead author of the study published in the Journal of the American Medical Association.Ī one-time stem cell transplantation treatment for multiple sclerosis showed improvements over the current therapies, according to a preliminary trial published in the Journal of the American Medical Association (JAMA). ![]()
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