Stem cell transplant approach for treating multiple sclerosis shows promise

Newsweek reported:



The study built on work by Dr. Richard Burt, a stem cell specialist at Northwestern University’s Feinberg School of Medicine, from 2015, the investigation focused on immune cells as a treatment for the illness. The new, highly anticipated data (which have not yet been published or reviewed by experts in the field) were presented on Sunday at the European Society for Bone and Marrow Transplantation in Portugal.



link:
http://www.newsweek.com/stem-cell-transplant-multiple-sclerosis-may-revolutionize-care-one-million-851332

From the BBC:


[The approach] involves wiping out a patient's immune system using cancer drugs and then rebooting it with a stem cell transplant.

(...)
The patients received either haematopoietic stem cell transplantation (HSCT) or drug treatment.

After one year, only one relapse occurred among the stem cell group compared with 39 in the drug group.

After an average follow-up of three years, the transplants had failed in three out of 52 patients (6%), compared with 30 of 50 (60%) in the control group.

Those in the transplant group experienced a reduction in disability, whereas symptoms worsened in the drug group.

Prof Richard Burt, lead investigator, Northwestern University Chicago, told me: "The data is stunningly in favour of transplant against the best available drugs - the neurological community has been sceptical about this treatment, but these results will change that."




link: http://www.bbc.com/news/health-43435868


Some current MS drugs deplete white cells (either B or B/T) and allow natural re-population.

Separately, there was some criticism at Futurism:


The results reported in the BBC piece are just the preliminary findings. And that leaves a number of questions still unanswered — are these results permanent? What are the risks? Who isn’t suited to have their immune system wiped out through aggressive chemo?

The U.S. Food and Drug Administration (FDA) has also flagged some serious issues in the study’s protocol.If that sounds boring and bureaucratic, think of it this way: for a few months, the lead investigator somehow forgot to report a number of nasty side effects of the treatment, including chest infection and the worsening of conditions as diverse as vertigo, narcolepsy, stuttering, and hyperglycemia, among others.

One thing we know for sure? It’s real expensive. The BBC noted it cost patients £30,000 ($42,000) to receive the experimental treatment, but biomedical scientist and science writer Paul Knoepfler, who has been following the trial since last year, says it ran some patients between $100,000 and $200,000.

Clinical trials are expensive, so it’s not totally uncommon for some of the cost to be passed on to patients that want to enroll, as long as they are clear about what they are getting.

But it’s patients’ expectations where things get a little murky. In his article, Knoepfler notes how the trial’s homepage, now password-protected but still visible through Wayback Machine, seems to overpromise: the homepage indirectly references the word “cure” in several places, showcasing headlines that mention it (though in an interview Burt said he wouldn’t use that word). Overall the page makes some awfully big claims for an experimental treatment, Knoepfler notes.



link: https://futurism.com/stem-cell-treatment-ms-multiple-sclerosis/

Background on stem cell approach from wikipedia:




Hematopoietic stem cell transplantation (HSCT) is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood.[1][2] It may be autologous (the patient's own stem cells are used), allogeneic (the stem cells come from a donor) or syngeneic (from an identical twin)