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.
Even after stopping the drug, patients who have taken natalizumab (Tysabri) continue to have an increased risk of primary multifocal leukoencephalopathy (PML) for many months. If they were to undergo HSCT during that time, the risk for this rare but serious brain infection carries over and would make the procedure more dangerous.
In their study, Burt pointed out, “we had no opportunistic infections, no PML, nothing, but my worry is, if you’ve had many years of prior treatment with Tysabri, and you’re [positive for John Cunningham virus], then you could get PML and people think it’s our transplant but it’s really all that prior Tysabri.”
Getting Her Life Back
One of Burt’s trial patients, Roxane Beygi, spoke on a panel at the Vatican Adult Stem Cell Conference in 2013. In a video of the event, she describes her life before the study.
Despite being on a DMT prior to the study, Beygi was relapsing regularly and could barely walk. She had trouble writing, brushing her teeth, and even performing simple tasks like drinking from a glass.
“Since I had my transplant, my life changed completely,” said Beygi, speaking more than two years after treatment, “[Before the transplant] I had major fatigue where I couldn’t even get out of bed. … Now I get up at 6 ... and a lot of the time I’m studying and exercising until like 1 a.m.”
Beygi ended her presentation by thanking Dr. Burt for giving her life back. She called him her “hero.”
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.
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