A high-risk, experimental stem cell treatment has proven that it can halt and even reverse the symptoms of aggressive multiple sclerosis.
Multiple sclerosis is a disease of the central nervous system that damages the material that surrounds your nerve cells, which are cells responsible for transmitting signals around the body. Cutting off those signals can lead to symptoms like muscle weakness, trouble with coordination, and problems with memory. Researchers aren’t entirely sure what causes MS, though some genetic markers have recently been identified, suggesting it’s at least partially hereditary. What is well established is that it has something to do with the immune system attacking these nerve cells. So researchers at the Ottawa Hospital Research Institute had an idea. They wanted to see what happens if you suppress the immune system, then add back stem cells that have been cleaned up so they no longer had the MS cells that cause the damage to nerve cells. If they could do that, would it act as a re-set button on the immune system?
The phase 2 clinical trial reported Thursday in the journal Lancet enrolled 24 people with multiple sclerosis whose disease was progressing quickly. All of the patients were given the stem cell/chemotherapy treatment. Stem cells are found all over the body and are unspecified cells that can regenerate. The ones used in the trial came from the person’s own bone marrow, which were withdrawn from the patient’s body, then added back in after they’d been cleaned and the body’s immune system had been wiped out by chemotherapy. Because messing with the immune system is rather risky, one patient died in the process.
Of those treated, 16 were able to go on without disease progression, and some were even able to regain some abilities that they had lost as the disease progressed. The Guardian reports that six of the 24 went back to work or college, five got married or engaged, and two had children. The study monitored the patients’ disease after treatment for up to 13 years after they got treatment.
Because the trial didn’t compare itself to anything else (patients taking medications), the next step, Dr. Paolo Muraro of the Imperial College London told the Genetic Expert News Service, will be to show if this treatment works better to other existing treatments. But, he noted, “Although no direct comparisons are available with standard therapy, none of the drugs that are currently approved, or are in late phase clinical trials, have been reported to achieve a similarly profound control of the disease.”