Canadian researchers have succeeded in getting patients with an aggressive form of multiple sclerosis out of wheelchairs and moving again. The protocol is risky, even potentially deadly, but the outcomes have been astonishing and are being closely watched by other neurologists.
Multiple sclerosis is the leading crippler of young adults, with an estimated 2.3 million people around the world affected by the neurological disease. In those with MS, the immune system mistakenly attacks the fatty outer layer of brain neurons, called myelin, a situation that over time that can lead to paralysis.
But what if the faulty immune system could be replaced with a new one? Would it still attack the brain's myelin sheath?
That’s in essence what researchers at the University of Ottawa asked, and answered.
Neurologist Mark Freedman, a senior scientist at the Ottawa Hospital and professor at the university, said investigators identified two dozen patients with an extremely aggressive early form of MS, who had failed at least one year of approved therapy .
“Three, four years into your disease, if you saw the wheelchair imminent into your future and someone offered you an alternative, you might consider it," Freedman said. "That’s the kind of population we went after." Critics, he said, argued that the chance of success was poor because such patients are "very resistant to treatment.”
Stem cells purified, returned
Researchers, describing their work in the journal The Lancet, used strong chemotherapy drugs to destroy the patients’ immune systems. At the same time, investigators collected stem cells from each patient’s bone marrow, purified them, and eventually transplanted the cells back into the participants to repopulate their immune systems.
“Theoretically," Freedman explained to VOA, "the brand-new immune system no longer carries that memory" of attacking the brain's myelin sheath. Whatever caused the attacks in the first place still isn't known, but the new immune system "won’t have that same problem again.”
The results were as breathtaking as they were unexpected.
“Two and three years after the transplant, many of the patients who had significant disability started to recover, recover quite a bit," Freedman said. "So we had patients who were already walking with a cane go back to a life where they were working, they were skating, they were skiing, they’re running, they’re doing everything.”
The results in detail:
— Since the study began in 2001, investigators say, not a single patient has experienced a relapse or flare-up in disease activity.
— No new lesions or scars could be detected in the brains of the participants in any follow-up magnetic resonance imaging (MRI).
— No patient needed any MS-specific drugs to control his or her disease.
— Seventy percent of patients had a complete halt in disease progression.
— Brain shrinkage, which occurs in MS, was reversed.
— Forty percent of participants experienced some reversal of symptoms, including blindness and balance problems.
Not without risk
But the procedure is not without significant risk. One of the 24 patients died from complications.
Paul Wright, chief of neurology at North Shore University Hospital in Long Island, New York, found the results very encouraging but agreed that doctors should move cautiously and take the findings with a grain of salt.
“A lot of the patients had febrile illnesses, where they developed fevers from infections," Wright said. "One patient died. So it’s not without risk and not without extreme concern, even though it still is promising.”
In Wright’s opinion, more studies need to be done. But in Canada, Freedman’s group has now treated an additional 20 patients with aggressive MS and plans to continue.