One wheelchair-ridden patient was given back her mobility.
In a 24-person clinical trial, a risky stem cell treatment for multiple sclerosis (MS) was found to stop the progression of the disease dead in its tracks, and some patients were even restored with the ability to move.
The patients, aged 18 to 50, were selected for the procedure based on whether their condition was already having or going to have a severe effect later in life.
Now, going 13 years strong after the initial treatment, 23 of the 24 patients are showing no new signs of MS as well as no relapses, and some recovered their mobility. Tragically, one of the patients died during the risky procedure.
Despite the small sample size, medical experts see promise in the pioneering technique.
“The clinical results are truly impressive, in some cases close to being curative,” Stephen Minger, a stem cell biologist who wasn’t involved in the study told The Guardian, “though we need longer-term follow-up to know for certain whether the patients continue to do well or if there is a chance of relapse. And of course this trial will need replication by other groups too.”
In the treatment, called immunoablation, the patient’s immune system is completely destroyed, thus giving the stem cells the opportunity to rebuild the immune system without any of the MS triggers.
Writing in The Lancet, the researchers explain that the technique employed “autologous haemopoietic stem cells,” which means the cells were derived from the patients’ own bodies, typically from bone marrow or peripheral blood.
However, the high risk of the treatment is that the patients become vulnerable to life-threatening infections during the timeframe in which their immune systems are wiped out. Unfortunately, the patient who passed away during the trial suffered from a bacterial infection that caused blood poisoning as a result of his wiped immune system, as well as severe liver damage.
“Stem cell therapy is still very experimental, and is not suitable for everyone,” Amy Bowen of the Multiple Sclerosis Trust told The Guardian.
Nevertheless, the 23 other patients had generally positive experiences with the treatment, and one of the participants, Jennifer Molson, went from being wheelchair-ridden in a rehab center to being able to work, ski, get her driver’s license, and dance at her wedding.
"Everyone is hesitating to use the 'c word,' but these patients are cured," Michael Rudnicki, director of the Regenerative Medicine Program and the Sprott Centre for Stem Cell Research, who was not involved with the research, told Vox.
Although the study lacked a control group, the researchers followed up on the patients for anywhere between four and 13 years after the treatment, which provides a lengthy picture of how the treatment will affect patients in the subsequent years. The scientists report that MS progression was halted in 70 percent of the patients in the study.
“We describe the first treatment for multiple sclerosis to fully halt all detectable CNS inflammatory activity for a long period in the absence of disease-modifying drugs,” the researchers write in The Lancet.
“For a substantial number of patients whose disease was not well-controlled with disease-modifying drugs, this procedure led to neurological improvement and long-lasting remission free of ongoing treatment,” they concluded.
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