Great lecture by Dr. Burt at the The Future of Genomics in Healthcare 2015 Thought Leaders Consortiu

Stem Cell Transplants Offer First-Ever MS Treatment That Reverse Disability

Only 2 years after getting a stem cell transplant, half of volunteers showed improvement in their disability scores — a first for any MS therapy. Dr. Richard K. Burt performed the first hematopoietic stem cell transplant (HSCT) for a multiple sclerosis (MS) patient in the United States at Chicago’s Northwestern Memorial Hospital. Now Burt, Chief of the Division of Medicine-Immunotherapy and Autoimmune Diseases at Northwestern University’s Feinberg School of Medicine, is making headlines again.

Burt and his colleagues published the results of their newest HSCT study earlier this week in the Journal of the American Medical Association. Their results show that HSCT could be the first MS therapy to reverse disability. Though the study group was small, the results have experts hopeful.

For this trial, 151 patients underwent a stem cell transplant. First, their immune systems were tamped down using low-dose chemotherapy. Then, doctors used HSCT therapy, involving an infusion of the patients’ own stem cells, previously harvested from their blood, to reboot their immune systems. After a short stay in the hospital, the volunteers went about their normal lives, needing no “maintenance” drugs.

Over the next several years, the volunteers were periodically given a series of tests to measure their disability. One test, known as the Expanded Disability Status Scale, or EDSS, measures cognition, coordination, and walking, among other things. Participants underwent MRI scans and completed questionnaires to measure their overall quality of life.

The researchers found that at two years post-transplant half of the patients showed a marked improvement in disability. Of the patients who were followed for four years, more than 80 percent remained relapse-free.

Since 1993, the FDA has approved 12 disease-modifying therapies (DMTs) to treat relapsing-remitting MS (RRMS). All are designed to suppress the immune system to one degree or another. These drugs cost about $5,000 per month and they must be taken indefinitely, since relapses will occur if the drugs are stopped. While patients now have many options to stave off disease progression, no DMT has been proven to reverse disability.

HSCT costs about $125,000 per patient. “Although we haven’t done a cost analysis, given how expensive Tysabri is, and Fingolimod, [since HSCT is a one-time treatment] it should start paying for itself around 18 months,” Burt told Healthline. Although HSCT is currently only available in clinical trials and for “compassionate use” in certain cases, Burt is hopeful that more studies will lead the FDA to approve stem cell transplantation for MS.

In fact, his team is currently conducting a larger study comparing HSCT to FDA-approved DMTs at three centers worldwide. The trial is currently enrolling, and interested patients can learn more at http://www.stemcell-immunotherapy.com/research_clinical.html.

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