HSCT essentially reboots the immune system.
"The goal is to remove the old, the new," Burt said, adding that it was "a one-time treatment." You're done, you do not consume any drugs anymore ". The patient's blood stem cells are first collected and then treated with chemotherapy drugs. Then, the blood stem cells are sent back to the patient to revive the development of a new immune system.
In contrast, disease-modifying treatments work differently. These are a dozen or more drugs designed to be chronic, ongoing treatments that target and modulate the immune system.
the A new comparative study took place in medical centers in the United States, the United Kingdom, Sweden and Brazil, where 110 patients with relapsing-remitting MS participated in the randomized clinical trial, a medical test reference. Patients received either the HSCT protocol or a class of disease-modifying therapies different from that used previously.
HSCT has proven to be the most effective treatment: Of the 55 patients receiving HSCT, only three patients presented the evolution of the disease at one year, showed the study. Yet, 34 of the 55 patients in the modifying treatment group experienced disease progression after one year. Disease progression was measured using the Extended Disability Status Scale, a method of tracking symptoms over time.
In the HSCT group, the proportion of patients with disease progression was (approximately) 2% up to two years, 5% at three years and 10% at four and five years. Meanwhile, the proportion of patients showing no signs of illness – defined by the lack of progression, relapse and the absence of new or enlarged lesions on MRI exams – was (almost) 98% at one year, 93% at two years, 90% at three years and 78% at four and five years.
In contrast, nearly one-quarter of the patients in the treatment arm group experienced disease progression after one year, more than half after two years, and just under three-quarters after five years. And the proportion of patients without signs of illness was about 40% at six months, 21% at one year and 3% at the age of four and five.
What are the side effects of HSCT?
Side effects of HSCT may include infertility, said Burt, who said women can choose to keep their eggs before treatment. And some patients have developed autoimmune thyroid disease, a treatable condition. He noted that disease-modifying treatments also have side effects.
Bruce Bebo, executive vice president of research at the National MS Society, said the study is "too small to be really definitive, but that it adds to a growing body of evidence suggesting that this approach has advantages ".
The disease-modifying treatments tested in the study did not include two of the newest and most effective drugs. "This is a gap in our knowledge," said Bebo, who did not participate in the research. "We did not have [them] when the study began. "
In previous studies on HSCT, patients with progressive MS did not respond to treatment, noted Bebo. Approximately 85% of patients are diagnosed with relapsing-remitting multiple sclerosis, where attacks of symptoms – such as dizziness, pain, and blurred vision – are followed by periods of remission. Still, a "substantial portion" of patients with relapsing multiple sclerosis is moving into a progressive form of multiple sclerosis, where symptoms do not worsen and do not go away and deteriorate over time, Bebo explains.
"In the beginning, the HSCT was also riskier than today, and the protocols have been refined since the early days," said Bebo, who said some patients had died. Today, the risk of death is "really, very low," said Bebo, who added that the chemotherapy drugs used by Burt "have been used to treat blood cancers for many years."
In the end, HSCT could be a beneficial treatment for some MS patients, said Bebo: "Probably people with an aggressive form of the disease that do not respond to other types of treatment."
Bebo advises patients to "pay close attention to an institution offering stem cell therapy for the treatment of MS". Other experimental approaches using stem cells are "less mature" and less studied, he said, and they could endanger the patient's health.
Burt said, "This should be done in a large university medical center." Bebo added that "you have to make sure that this is done by a center with a lot of experience.You really have to do your homework and find a place that has a proven track record."
In the future, Burt will fine-tune HSCT to make it safer.
"The hope is to change the natural history of this disease," Burt said. "These data suggest we are doing it."