CMSC INforMS: Stem Cell Transplants – an Exciting MS Therapy Development
Monday, June 6, 2016
Posted by: Elizabeth Porco
Stem cell treatment of MS, which came under the spotlight at last week’s 16th Annual Meeting of the Consortium of Multiple Sclerosis Centers (CMSC) when Dr. Mark Freedman discussed the procedure, is an exciting area within the development of therapies.
Dr. Freedman, professor of medicine (neurology) at the University of Ottawa in Canada, talked about collecting suitable stem cells from bone marrow and developing them in vitro (in a laboratory) to produce suitable stem cells to be transplanted.
Not being either a doctor or a scientist, but having MS, I am looking at this from a patient’s perspective. Therefore, this column will only include essential scientific terms.
At this point, it is enough to say, Dr. Freedman said that certain stem cells have several distinctive properties that might make them useful for the treatment of MS – including the protection and repair of myelin.
Treatment of a mouse model of MS — the experimental autoimmune encephalomyelitis (EAE) mouse — with human stem cells from the bone marrow, has been promising. It has been shown to enhance the formation of the cells producing myelin in the central nervous system, and to reduce an abnormal increase in the number of astrocytes (connecting tissue cells that link nerve cells to blood vessels) due to the destruction of nearby neurons, Freedman said.
Freedman also discussed his own “MEsenchymal Stem cell therapy for CAnadian MS patients” (MESCAMS) study, a Phase 2a clinical trial, part of an international stem cell research effort involving nine countries.
The goal of MESCAMS is to determine if treatment with a single intravenous infusion of autologous stem cells (cells originating from the same person receiving treatment) is safe in MS patients with either relapsing or progressive forms of the illness. It will also attempt to see if it can reduce brain inflammation, brain lesions, brain atrophy, relapse rate, and slow disability progression. The potential of MSC to stimulate protection or myelin repair will also be investigated.
Results of all national trials will be pooled and, according to Dr Freedman, should provide enough statistical power to draw conclusions regarding the safety and efficacy of autologous stem cell transplants (AHSCT) as a therapy for MS.
Stem cell therapy already in use for MS
This international trial is well overdue with AHSCT therapy, which started as a cancer treatment including high doses of chemotherapy, is available — at a cost — to MS patients at various centers throughout the world, most notably in Moscow, Mexico, and Israel.
Anecdotal reports from patients who have been through this procedure are extremely positive. It’s a hard journey through the month-long treatment in the clinic, followed by an extended period of recovery at home and further medical checks, but it is a journey of hope and confidence in the future.
Two recent comments on Facebook stick in my mind. They were both from patients now recovering at home. One said she had that morning put on her panties and shorts (she lives in the Bahamas) while standing up, something that she had not been able to do for years. On another day, the same woman, said that she had been upstairs when the phone rang and that it was only as she answered it that she realized she had run down the stairs without holding the banister whereas before the treatment she struggled with the stairs.
A different person reported that she had just finished a meal and used her right hand to eat. Nothing unusual for a right-handed person, right? No, except MS had made using that hand impossible before she underwent that treatment!
Back at CMSC, Dr. Freedman showed results from a previous study in which neurological recovery was achieved after destruction of patents’ immune systems followed by and autologous stem cell transplantation. That is exactly the process involved in current AHSCT therapies with chemotherapy being used to destroy the immune system before ‘rebooting’ it with the transplanted cells. In the trial, Freedman added, the treatment also halted the damage induced by acute inflammation, further widespread damage associated with progressive disability and atrophy, and allowed or promoted disability recovery. This therapeutic strategy also eliminated the need for further treatment with disease modifying therapies (DMTs).
“Not a single patient required DMTs after therapies. They are taking nothing,” Freedman said.
Speaking to Multiple Sclerosis News Today after his presentation, Dr Freedman talked about possible future for these therapies.
He said: “The sky is the limit. We are able to make any cell we want. The next major hurdle is to be able to show that those cells, once put in, (…) are going to renew functionality, and maybe counteract wherever the disease’s process is that we are talking about, be it a genetic disorder, be it an acquired inflammatory disease like MS.”
I have to say that trial results are eagerly awaited by the patient population in the hope and expectation that stem cell treatment will soon be readily available as an accepted MS therapy worldwide.
By Ian Franks
Multiple Sclerosis News Today
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