Experimental Stem Cell Therapy Stops Multiple Sclerosis In Its Tracks

Five-year results suggest the treatment may be ground-breaking

Illustration: Vocativ
Feb 03, 2017 at 10:56 AM ET

The prognosis for people affected by multiple sclerosis (MS), a degenerative autoimmune disorder that decimates the central nervous system, is a bleak one. The disease often begins with a sudden burst of neurological symptoms like muscle spasms, vision problems, and trouble walking, then progresses differently, depending on which form of MS someone has. But eventually, nearly everyone with the disease comes to the point of being unable to move, breathe, or live independently. And sufferers on average live anywhere from five to ten years less than the general public.

Currently, the best medications we have available do little more than slow MS down, or tamp down people’s symptoms. But an experimental therapy continues to provide the first glimmers of something ground-breaking — an actual way to stop one form of the disease in its tracks, and maybe even reverse some of the damage already done.

In this month’s Neurology, researchers detailed the final five-year-old results of a small clinical trial called HALT-MS. Twenty-four volunteers with MS who hadn’t responded to conventional drugs were first given a powerful form of chemotherapy, high-dose immunosuppressive therapy (HDIT), that wiped out their immune system. Then they were given a transplant of their own stem cells taken out earlier, known as autologous hematopoietic cell transplant (HCT). These purified cells, the researchers theorized, would seed a new generation of uncorrupted white blood cells and reset the immune system, freezing MS in its place.

For the most part that’s exactly what the combination HDIT/HCT therapy did. Nearly 70 percent of patients, five years in, have experienced no signs of the disease progressing. They haven’t had a relapse of symptoms, become more disabled, or had new brain lesions show up in imaging exams. Some have actually improved physically in the years since the treatment. And even those not in complete remission appear to be suffering less than before. Importantly, though the treatment isn’t free of side-effects, there haven’t been severe ones. There were three deaths seen during the trial, all of whom experienced worsening MS, but none were attributed to the treatment.

The volunteers all had relapsing-remitting MS, the most common form, in which symptoms come and go with little rhyme or reason.

“The evidence at this time is encouraging, but it isn’t definitive,” study author Dr. Linda Griffith, a researcher at the National Institute of Allergy and Infectious Diseases (NIAID), which sponsored the study, told Vocativ.

As Vocativ has previously reported, this isn’t the first trial to find similar success rates for HDIT/HCT, though it does come with its own dangers. Patients can die from it, and like all kinds of chemotherapy, the deliberate weakening of the immune system often leads to more infections. It also doesn’t seem to be as effective for more advanced types of MS, when the disease has stopped causing active inflammation, said Griffith. And while it could be promising for people in the earliest stages of MS, the research needed to promote it as a first-line treatment isn’t there yet either, she added.

For now, the only trials of HDIT/HCT have been small and isolated. And though the effects of it when successful seem to extend as far out as 13 years later, it’s too early to call it a full-on cure. We still don’t have a clear grasp of why MS happens in the first place, but it’s thought that multiple triggers like infections and unlucky genetics combine to increase people’s risk. So even if resetting someone’s immune system does treat MS completely, it’s plausible that some percentage of patients could fall victim to it again down the road, Griffith explained. We just don’t know enough right now.

But Griffith is hopeful that larger, randomized studies will be underway within the next year or so. And if those prove to be as successful as the HALT-MS trial and others, the therapy could someday soon lead to a light at the end of tunnel for the millions of MS sufferers alive today.