Most of the multiple sclerosis (MS) patients who took part in the cutting-edge stem cell study HALT-MS are still in remission years later. The phase 2 study has demonstrated impressive results by rebuilding the immune system using a patient’s own stem cells.
Studying 24 study volunteers who underwent stem cell transplants between 2006 and 2010, Dr. Richard A. Nash of the Colorado Blood Cancer Institute in Denver and his colleagues recently published their findings in JAMA Neurology.
Researchers found that more than 86 percent of the patients remained relapse free after three years, and nearly 91 percent showed no sign of disease progression.
Building a New Immune System, from the Ground Up
The goal was to reboot the patients’ immune systems. The researchers gauged success based on how long the patients remained relapse-free.
The study involved patients with relapsing-remitting MS whose disease did not respond to at least one FDA-approved disease-modifying drug. Patients also had to score between 3.0 and 5.5 on the Expanded Disability Status Scale (EDSS), a set of tests to measure walking, cognition, dexterity, and quality of life in MS patients. People who fall into this range typically have mild to moderate disability.
Patients were given high-dose immunosuppressive therapy, or HDIT, to erase their native immune system. Then, researchers reintroduced blood-forming stem cells that had been harvested from the patients’ own blood.
The scientists thought that perhaps the new immune system would no longer see the myelin covering of nerve cells in the brain and spinal cord as a threat. If the stem cells could halt the myelin attacks, the MS disease process would be stopped.
“On average patients were hospitalized for three to four weeks,” said Nash in an interview with Healthline. That allowed enough time for the immune system to regenerate so patients could safely return home.
“Patients are immunosuppressed, so they are on prophylactic antimicrobial medications. They are also educated regarding how to reduce the risk of infections after transplant,” explained Nash.
Hear a Patient’s Perspective
“Prior to my admission to the hospital, researchers harvested millions of stem cells from my blood ... and froze them,” Dave Bexfield, founder of ActiveMSers and a participant in the ongoing study, explained in an interview with Healthline. He then spent his first week in the hospital receiving five different types of chemotherapy.
A little more than a week after his stem cells were thawed and reintroduced into his bloodstream, they began to regenerate his immune system. After that, Bexfield was able to leave the hospital within a matter of days.
A stem cell transplant is not without risks. Besides the possibility of serious infections, the toxicity of the chemotherapy drugs used to wipe out the immune system can have unpleasant side effects.
Bexfield admits that the experience was hard, but “fortunately, the chemo never made me very nauseous and there were only a few days where getting out of the bed was all but impossible.”
After 21 days in the hospital, participants were released, but they had to stay close for regular monitoring. “I had to limit my public exposure, eat only well-cooked foods (no salads!),” said Bexfield, “and monitor my body temperature. If there was any sign of a fever, I was instructed to go to the ER immediately. Thankfully, that never happened.”
In all, the procedure took about three months from preliminary testing until the day participants could go home.
The Cost of Volunteering for a Trial
Medical research can offer many benefits to patients, including access to cutting-edge therapy before it is widely available, often at no cost. Although this study was funded by the National Institutes of Health, by the time Bexfield signed up, the funding had been pulled.
Bexfield’s insurance company, Presbyterian Health Plan, declined to cover the hefty cost of $198,000 for the stem cell transplant. He was forced to cash in his savings and turn to family in order to help pay for the treatment.
Only months after his denial, Presbyterian added coverage for stem cell transplants to their plans. Bexfield thought that meant his procedure would be paid for after all. But there was a hitch. Bexfield was told the insurance company would only cover stem cell transplants from donors — a much riskier procedure — not those done using the patient’s own cells.
Bexfield waged an epic battle against the insurance company that lasted several years and consisted of countless emails, YouTube videos, and even help from David Segal, aka “The Haggler” from the New York Times. In the end, Presbyterian reimbursed his $198,000 original payment for the procedure and paid $204,000 in interest.
Are Stem Cell Transplants the Holy Grail of MS Treatment?
Although the HALT-MS study is ongoing, these preliminary results at the three-year mark are astounding. Bexfield, as well as most of the other patients in the study, has remained relapse-free, with no enhancing new lesions visible on an MRI scan.
Moreover, many of the patients have shown remarkable improvement in their EDSS scores. Bexfield went from being housebound at times before the study to resuming many of his favorite activities, including hitting the slopes on his snowboard — if only for yards at a time between falls.
Improvement in the EDSS scores from baseline suggests that nerves may be remyelinating, the holy grail of MS research. Restoring myelin can repair damaged nerves, restoring their function.
Unlike earlier studies of progressive MS patients, which showed no improvement in neurological function, HALT-MS looked at people who had only been diagnosed for 15 years or less and who were still experiencing relapses.
Although it’s too soon to know whether stem cell transplants are the future of MS treatments, Nash is hopeful. “We are doing a study to try to evaluate [remyelination] by MRI when we have our five-year data available,” he said.