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The approach, called hematopoietic stem cell transplantation (HSCT), has long been part of certain cancer treatments. A round of chemotherapy knocks out the immune system and an infusion of stem cells—either from a patient’s own blood or, in some cases, that of a donor—rebuilds it. The procedure is already in use for MS and other autoimmune diseases at several clinical centers around the world, but it has serious risks and is far from routine. Now, new results from a randomized clinical trial suggest it can be more effective than some currently approved MS drugs.

“A side-by-side comparison of this magnitude had never been done,” says Paolo Muraro, a neurologist at Imperial College London who has also studied HSCT for MS. “It illustrates really the power of this treatment—the level of efficacy—in a way that’s very eloquent.”

Nearly 30 years ago, when hematologist Richard Burt saw how HSCT worked in patients with leukemia and lymphoma, he was struck by a curious effect: After those patients rebuilt their immune systems, their childhood vaccines no longer protected them, recalls Burt, now at Northwestern University’s Feinberg School of Medicine in Evanston, Illinois. Without a new vaccination, the new immune cells wouldn’t recognize viruses such as measles and mumps and launch a prompt counterattack. That suggested that in the case of an autoimmune disease, reseeding the immune system might help the body “forget” that its own cells were the enemy.

Burt and others have since used HSCT for a variety of autoimmune diseases, including rheumatoid arthritis and lupus. In the past few years, several teams have reported encouraging results in MS. But only one study—which evaluated just 17 patients—directly compared HSCT to other available drug treatments.

In the new trial, Burt and his colleagues recruited 110 people with the most common form of MS, known as relapsing-remitting. In that form of the disease, patients can go long periods without symptoms—which include muscle weakness and vision problems—before inflammation flares up. Trial participants had at least two such relapses in the previous year, despite being on one of several approved MS drugs.

Half the participants continued with drug treatment but switched from a drug that wasn’t working for them to a drug of a different class. The other half underwent HSCT. First, the researchers collected their blood to reinfuse later. Then, they gave patients a combination of drugs to kill most of their immune cells. In this trial, the patients would have regenerated their own immune systems with stem cells in bone marrow that were spared annihilation, Burt notes. But they received the reinfusion of their own stem cell-rich blood to help speed recovery by several days.

A year later, the researchers evaluated how far the disease had progressed in each of the patients. According to a zero-to-10 scale of disability that includes measures of strength, coordination, and speech, roughly 25% of those in the drug treatment group showed at least a one-point worsening in their score, compared with just 2% of those in the transplant group, the researchers report online today in The Journal of the American Medical Association (JAMA).

MRI scans also revealed less extensive brain lesions in the transplant group and improvements in a patient survey about quality of life. (Survey scores worsened slightly in the drug-treated group.) Five years after treatment, about 15% of people in the transplant group had had a relapse, versus about 85% of the control group.

The two groups diverged “in a massive way,” Muraro says. But he adds that two of the most effective MS drugs weren’t included in the control group. One, ocrelizumab, had not been approved when the study was enrolling participants. Another, alemtuzumab, was excluded because it also depletes immune cells…

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A new Intense Stem Cell Therapy known as HSCT has shown promising results during a clinical trial. However, experts give words of caution, saying that it might not work for everyone.

Stem cells might prove to be an important breakthrough for the treatment of multiple sclerosis, but the road to there might be a long and painful one.

A new clinical trial tried to explain this. During the trial, participants with multiple sclerosis (MS)
that were using nonmyeloablative hematopoietic stem cell transplantation (HSCT) showed better results than the participants on disease-modifying treatments (DMTs).

This randomized clinical trial is the first one of its kind, comparing HSCT to DMTs.

Researchers involved in this trial stated that a significant portion of participants on HSCT treatment was successful in slowing down the progression of the disease, in comparison with the participants that were using DMTs.

Out of 103 participants, only three (on HSCT) showed disease progression when compared to the 34 that were on DMTs. Because of this, the research team made a conclusion that HSCT is better at slowing down progression than DMTs, but pointed out that it is important to come up with the right type of patients that will be subjected to what has been shown to be a rigorous and somewhat dangerous therapy.

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

Dr. Barbara Geisser, 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 had this to say on the matter: “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.”

A rigorous treatment

HSCT works by using chemotherapy in order to aid cells to “forget” they have MS. By doing this, the immune system is brought down to some quite vulnerable levels. The patient has to be hospitalized and treated with the greatest care.

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

The participants of the trial had relapsing MS, characterized with mild to moderate disabilities, with a range of 2 to 6 on the Expanded Disability Status Scale (EDSS).

Bebo said: “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,” adding that “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 further continued to explain the difficulty of this study in performance, and elaborated on how Burt and his team were able to create an unbiased way to make a comparison between HSCT and DMTs, by way of excluding ocrelizumab and alemtuzumab.

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(CNN)-An experimental stem cell therapy proved effective and safe in patients with a relapsing form of multiple sclerosis , an autoimmune disease that affects the central nervous system, new research finds.

A stem cell transplant using a lower-dose regimen of chemotherapy plus immune system suppressors is more effective at preventing disease progression compared to currently used disease-modifying therapies, according to the new study published Tuesday in the journal JAMA. (The stem cell treatment is known as “autologous hematopoietic stem cell transplantation” or HSCT.)

Among the HSCT group, the proportion of patients with disease progression was (roughly) 2% up to two years, 5% at three years, and 10% at 4 and 5 years. Meanwhile, the proportion of patients with no evidence of disease — defined as no progression, no relapses, and no new or enlarging lesions on MRI scans — was (nearly) 98% at one year, 93% at two years, 90% at three years, and 78% at four and five years.

By contrast, almost one quarter of the patients in the disease-modifying therapy group showed disease progression at one year, more than half at two years, and just under three-quarters at five years. And the proportion of patients with no evidence of disease was (about) 40% at six months, 21% at one year and 3% by years four and five.

What are the side effects of HSCT?

Side effects of HSCT can include infertility, said Burt, who noted that women can choose to preserve their eggs before treatment. And some patients developed autoimmune thyroid disease, a treatable condition. He noted that the disease-modifying treatments also have side effects.

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