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EDSS Disability Scores significantly improved two years after stem cell reimplantation in 150 patients

February 1, 2015

Based on a study published in the Journal of the American Medical Association by Dr. Richard Burt and his team at Northwestern University, utilizing nonmyeloablative hematopoietic stem cell transplantation resulted in substantial improvements in select MS patients' quality of life and neurological disability.

What's hematopoietic stem cell transplantation, or HSCT? It's the process whereby researchers harvest a user's own stem cells from their bone marrow or peripheral blood (stem cells can come from a donor as well), and subsequently infuse these potent stem cells back into the body in an effort to get an MS patients' immune system back onto the right track.

For its study, Northwestern University researchers examined 123 patients with relapse-remitting MS and 28 with secondary progressive MS who had been previously treated with HSCT. At a median of 30 months following treatment, researchers observed that 64% of those tested at the 48-month mark had demonstrated significant Expanded Disability Status Scale (EDSS) scores, as well as 50% of those tested at the 24-month mark. This marked the first true long-term and sustainable improvement in EDSS scores recorded in a clinical study involving MS.

HSCT was also associated with improved cognitive function and quality of life with these patients, as well as a reduction in the volume of brain lesions as demonstrated by the use of an MRI. Furthermore, the relapse-free survival rate after four years was 80%, and the progression-free survival rate (i.e., disease stabilization) was 87%.

There are two notable downsides worth mentioning. First, EDSS scores for those with secondary progressive MS didn't improve. The implication is that HSCT may only work in earlier stages of the disease, although that's merely an educated guess on my part following this study, and would require additional research.

The other concern is HSCT comes with a number of possible complications. HSCT is an aggressive treatment usually reserved for aggressive forms of cancer, so the complications data could be a bit misleading, but its safety profile is nonetheless something worth keeping a close eye on.

Quality of life improvements are already here
It would truly be a great thing if researchers could safely find a way to treat earlier stages of MS in a way that leads to long-term disease stabilization, or perhaps even a cure. Of course, as a realist, I also understand that more time and testing will be needed before this becomes a reality.

The good news is that a handful of drug developers have devoted substantial research and development dollars toward improving MS patients' quality of life. I'm happy to say we're already seeing improvements.

Although Sanofi's Aubagio and Novartis' Gilenya are helping, it's really Biogen Idec's Tecfidera that's making a world of difference.

Tecfidera is a pill MS patients take twice daily that, in clinical studies, lessened relapse rates by 49% and reduced new or expanding lesions as observed via an MRI by 71% to 99%, depending on the study. Best of all, Tecfidera was a substantial improvement in safetyrelative to Aubagio, which comes with a serious black-box warning concerning the potential for severe liver problems, and Gilenya, which, in rare cases, has the potential to cause cardiovascular problems. Strong efficacy and a safer product is the ultimate goal of drug developers, and a reason Tecfidera has been such a runaway success for MS patients.

There's room for more improvement, though. One compelling experimental therapy currently being examined is daclizumab for relapse-remitting MS. Developed by Biogen Idec and AbbVie, daclizumab delivered encouraging results in a late-stage study known as DECIDE, the results of which were announced over the summer. The results showed that daclizumab was superior to Biogen's injectable Avonex based on the study's primary endpoint and led to a statistically significant 45% reduction in annualized relapse rate relative to Avonex.

What's really intriguing about daclizumab is that it's a once-monthly injection, making it a very convenient and quick treatment option for relapse-remitting MS patients. I'd opine that its chances of approval by the Food and Drug Administration are better than 50-50.

Another interesting solution could be to take an existing medication like Tecfidera and, as a play on words, put it on steroids. Alkermes announced the initiation in July of a phase 1 study utilizing a novel monomethyl furmarate (MMF) molecule to treat MS. Tecfidera is a dimethyl fumerate compound that breaks down into MMF once in the body. The idea for Alkermes is to develop a non-inferior MS product in terms of efficacy that potentially has an improved safety profile relative to Tecfidera.

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