Stem cell transplant shows promising results for reversing disability in patients with MS

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Scientists Continue Stem Cell Research While Courts Debate BanMultiple sclerosis (MS) is an unpredictable, disabling disease of the central nervous system. In its severest form, MS may impair locomotion, vision, bladder function, and even respiratory function, and can be fatal. Many experts estimate that up to 400,000 Americans have MS. Currently, there is no FDA approved treatment that has been found effective in reversing neurological damage from MS. Earlier this month we wrote about encouraging results of a small study looking at stem cell treatment of MS. And now, new research indicates that in patients with relapsing-remitting multiple sclerosis, stem cell transplant may be the first treatment to reverse disability.

The study included 151 patients with relapsing-remitting MS (123 patients) the most common form characterized by intermittent episodes of progressive disease and remission or secondary-progressive MS (28 patients) these patients typically just get worse as time goes on. First, the patients underwent low-dose chemotherapy. Then, they underwent hematopoietic stem cell transplant (HSCT) to reboot their immune systems. Over the next few years, study subjects were periodically given a series of tests to measure their level of disability including cognition, coordination, and walking. Patients also underwent MRI scans and completed questionnaires (MRI scans detect CNS demyelination, a key parameter in objectively measuring MS progress).

Data obtained from 145 patients showed that 50 percent of patients achieved significant improvement in disability after 2 years, and 64 percent of patients showed significant improvement after 4 years. HSCT was associated with improvement in physical function, cognitive function, and quality of life. Also, there was a reduction in the volume of brain lesions associated with MS seen by MRI. The study was led by Dr. Richard K. Burt, Chief of the Division of Medicine - Immunotherapy and Autoimmune Diseases at Northwestern University s Feinberg School of Medicine. The study was published in the Journal of the American Medical Association.

In a recent news release, the authors stated, To our knowledge, this is the first report of significant and sustained improvement in the EDSS score [a measurement system for objectively assessing disability] following any treatment for MS. However, the EDSS score did not improve in patients with secondary-progressive MS or in those with disease duration longer than 10 years.

HSCT is available only in clinical trials and for compassionate use in specific cases, however, Dr. Burt is hopeful that more studies will lead to a rapid FDA approval of HSCT for MS.