A therapy known as HSCT performed well in a clinical trial, but experts caution the treatment might not be for everybody.

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A stem cell treatment demonstrated in a clinical trial that it is capable of slowing down progression of multiple sclerosis. Getty Images

Stem cells may be an important pathway for the successful treatment of multiple sclerosis, although it can be a long and painful road getting there.

A recent clinical trial bore this out.

In the trial, people with multiple sclerosis (MS) using nonmyeloablative hematopoietic stem cell transplantation (HSCT) performed better than those on disease-modifying treatments (DMTs).

The randomized clinical trial is the first to compare HSCT to DMTs.

Researchers said a significant number of patients on HSCT treatment were able to slow disease progression, compared to those using DMTs.

Of the 103 participants, only three of those on HSCT saw disease progression compared to 34 of those on DMTs.

The research team concluded that HSCT slows progression better than DMT, but they added it is imperative to find the right type of patient to undergo what is a rigorous and sometimes dangerous therapy.

The study was led by Dr. Richard Burt, chief of immunotherapy and autoimmune diseases at the Northwestern Feinberg School of Medicine in Illinois.

“This important study is the first randomized trial of HSCT vs. DMT, and demonstrated that HSCT was superior to conventional DMT in stabilizing persons with relapsing MS with respect to relapses and progression and improving some functions,” said Dr. Barbara Giesser, professor of clinical neurology at the David Geffen School of Medicine at the University of California Los Angeles (UCLA) and clinical director of the UCLA MS program.

HSCT uses chemotherapy to help cells “forget” they have MS.

In doing so, the immune system is stripped down to vulnerable levels. Patients must be hospitalized and treated with extreme care.

“HSCT is not one therapy but a range of therapies from a more mild type of chemo to a stronger type,” Bruce Bebo, PhD, executive vice president of research at the National Multiple Sclerosis Society, told Healthline. “Dr. Burt practices a more mild form of chemotherapy — more mild form of immunosuppression — so the risks are lower.”

Trial participants had relapsing MS with mild to moderate disabilities, ranging from 2 to 6 on the Expanded Disability Status Scale (EDSS).

“This study stands above the others — first controlled randomized autologous stem cell transplant. Burt and his team should be applauded for taking this on. It’s what everyone was asking for,” Bebo said.

“A lot of groups are experimenting with this type of treatment — case studies and evidence is accumulating for its positive benefit and accumulating info for the best patient and approach for success,” he added.

Bebo explained how difficult this study was to perform and how Burt and his team created an unbiased way to compare HSCT to DMTs, excluding ocrelizumab and alemtuzumab, which were not included.

Due to potential visible side effects with the chemo such as hair loss, a “blind reader” reviewed the results of the tests during the trial. This reader was off site and had no access to the participants.

“Although HSCT appears to be efficacious and was well-tolerated in this trial, this is a very aggressive treatment with potential complications of serious infection, infertility, or death, and may not be suitable for everyone,” Giesser told Healthline. “Further work is needed to identify which patients are the optimal candidates for this therapy.”

Stem cell therapies run the gamut from non-FDA approved clinics across the globe to well-trained researchers running clinical trials under strict guidelines for safety and efficacy.

The potential success in HSCT has created high demand for the treatment, resulting in dangerous “stem cell tourism,” where people with MS travel to other countries for unauthorized treatments.

HSCT permanently changes your immune system. The long-term consequences are not known.

“HSCT is not a trivial treatment,” cautions Bebo. “It greatly suppresses bodies’ ability to fight infection for weeks or months. Patients are hospitalized for days or weeks.”

“HSCT has shown potential in various studies, but all have been open label where results could be biased. This study is firm and rigorous,” said Bebo.

Bebo discussed the importance of Burt’s work and expertise.

“He has been working in this area for some 20 years and continues to refine his approach to make it as effective and safe as possible,” he said. “A key component of success is the selection criteria for patients, which Burt has refined.”

“HSCT requires being treated at only a handful of facilities. Don’t mistake this for pop-up clinic. This is different and highly respected. Only few places do this well. In the wrong hands, it can be dangerous,” warned Bebo.

“HSCT is not for all MS,” added Bebo. “There is growing evidence that isn’t going to work for progressive MS but is more appropriate for very aggressive RRMS not responding to other therapies.”

Bebo suggests patients work with their healthcare providers to find centers doing treatments with the lowest risk.

“The results are not definitive as it was still a pretty small trial,” said Bebo. “We are likely to find out more about this treatment as to whose is best to receive it and how does it fit in with other treatments — the big picture.”

Burt is currently recruiting 200 participants for his randomized phase III trial at Northwestern called “Maximizing Outcome of Multiple Sclerosis Transplantation (MOST).”

Editor’s note: Caroline Craven is a patient expert living with MS. Her award-winning blog is GirlwithMS.com, and she can be found on Twitter.