One of the most prominent clinical trials of stem cell transplants for MS will enroll new participants in early 2018.
By Quinn Phillips
A few years ago, Everyday Health reported on a clinical trial called HALT-MS. This ongoing study is one of several in recent years to investigate stem cell transplants for multiple sclerosis (MS), in which a person’s own stem cells are harvested before a course of chemotherapy is given to almost completely destroy the immune system, after which the stem cells are infused back into the body.
HALT-MS is one of the most prominent ongoing trials involving stem cell transplants for MS, and its researchers have their sights set on making transplantation a mainstream treatment for MS. But first, they need to directly compare transplantation with the best drug-based treatments currently available — which is exactly what phase 3 of the trial will do.
Whether you’re potentially interested in participating in future clinical trials, or just eager to learn about where MS treatment may be headed, here’s an overview of what researchers hope to learn in the study’s next phase and what it may mean for participants.
The Promise of Stem Cells
The idea behind stem cell transplantation is to replace a malfunctioning immune system — in the case of MS, one that attacks the myelin sheaths of nerves — with one that properly recognizes these nerves as the body’s own tissue and doesn’t attack them. It’s sometimes referred to as “rebooting” the immune system, and the hope in every case is that the “new” immune system never begins attacking nerve cells the way the old one did.
Stem cell transplants have been shown to do this with mixed effectiveness. In a study published in April 2017 in the journal JAMA Neurology, researchers found that within a group of 281 people with MS — most of whom had progressive forms of MS — who had the treatment between 1995 and 2006, 46 percent experienced no progression of the disease in the five years after their transplant. Younger people and those with relapsing-remitting MS were more likely to have their disease halted by the procedure.
Stem cell transplants also come with some risks, including low blood cell counts, infections, gastrointestinal problems, blood clots, pain, and even death.
But Michael Racke, MD, a neurologist at the Ohio State University Wexner Medical Center in Columbus and a researcher in the HALT-MS trial, says the effectiveness and safety of stem cell transplants has continued to improve since 2006, when the first participants were screened for enrollment in phase 2 of HALT-MS.
Out of three major trials that have published results in recent years — including HALT-MS — all showed about a 70 percent rate of success in stalling disease activity five years after the procedure. Disease activity was measured either by relapses, progression of disability, or the appearance of new lesions on MRI.
“As in all of medicine, we just keep getting better at the things we do,” Dr. Racke says, noting that the mortality rate of stem cell transplants has dropped from about 3.5 percent before 2006 to about 0.3 percent since then.
Stem Cell Transplantation: ‘A Hard Road’
One participant in phase 2 of HALT-MS was Dave Bexfield, a New Mexico resident who was diagnosed with MS 11 years ago at age 37. He’s the founder and editor of ActiveMSers, a support site for people with MS with a strong focus on fitness that also covers the scientific literature on stem cell transplants.
“I was getting desperate,” says Bexfield of his physical condition when he enrolled in the trial in 2010. Over the previous six months, he had gone from occasionally using a cane to using a walker all the time, and his hand dexterity and cognition were getting worse quickly. He had to train with his sister, a physical therapist, to be able to walk the 100 meters unaided to qualify for the trial, which he says created “major, major stress” at the time.
The entire transplant process, Bexfield says, was “a hard road,” because of the constant tests and especially the chemotherapy drugs involved during his three-week hospital stay. After the first week, during which chemotherapy was given, he was still feeling “pretty decent.” Then, he says, “the chemotherapy really started to kick in. I was in bed for about four straight days,” and once passed out from vomiting.
High Risk of Infection
But Bexfield improved gradually during the rest of his stay, and managed to avoid getting a serious infection — something that researchers are on the lookout for when participants’ immune systems are compromised. Even after being discharged, Bexfield says, “Your immune system is so low, you need to be really careful about every food you eat, who you see. I was basically in isolation.”
The dramatic improvements he experienced following the procedure, Bexfield says, more than made up for the risks and unpleasantness he endured. “Within the first couple of months, I went from barely being able to walk to being able to walk 500 meters.” His brain fog cleared up, and his strength improved “on every level.”
These improvements lasted for about four years, after which, Bexfield says, “I noticed my legs weren’t working quite as well.” He’s now back to using a walker all the time, but in all areas except walking, he still feels much better than he did before the transplant. “I would do this treatment 100 times over. It was that valuable,” he says.
The Next Phase: BEAT-MS
When HALT-MS begins its phase 3 trial (called BEAT-MS) in early 2018, says Racke, it will enroll about 120 participants, ages 18 to 50, with relapsing-remitting MS. To qualify for the trial, he says, you must have failed to achieve successful outcomes on two different disease-modifying therapies, as demonstrated by a relapse on one and MRI activity on the other.
About half of the participants will be randomly assigned to receive stem cell transplants, while the other half will receive the best possible medication therapy, as determined on an individual basis. Participants in both groups will be tracked for five years.
Racke emphasizes that in addition to looking at disease-related outcomes, phase 3 will also compare the costs of treatments in both groups. He notes that many drug treatments for MS cost as much as $70,000 to $90,000 a year, while the cost of a stem cell transplant at Ohio State during phase 2 was between $40,000 and $100,000 — with beneficial effects that lasted for years.
“What’s even more important to point out,” says Racke, is that the stem cell transplants worked “significantly better in patients who were actually much worse. So this will hopefully be something that, down the road, could be a significant option for people who have really bad MS.”