Researchers are investigating an aggressive approach to treating multiple sclerosis (MS) that has shown promise in long-term prevention of relapses, but also comes with a risky safety profile.
The 24-patient Candian study found that 're-setting' the immune system with high-sensitivity chemotherapy, followed by autologous haematopoietic stem cell transplantation (AHSCT), can halt relapses for people with highly active, relapsing forms of the condition.
The findings, published in The Lancet, show that none of the trial participants suffered a relapse during the follow-up period, which ranged from four to 13 years. Also, 70 percent did not experience any worsening of disability after the treatment, and 40 percent actually experienced an improvement during follow-up.
However, the procedure is linked with significant safety risks: one trial participant died from liver failure after undergoing AHSCT, while another needed intensive care following the aggressive chemotherapy. A quarter developed infections and all bar one had a toxic response to treatment.
Nevertheless, the prospect of potentially being able to offer patients a curative therapy marks a massive leap forward in field, although larger clinical trials with control groups will be needed in other to confirm the benefit of this approach.
Also, "since this is an aggressive treatment, the potential benefits should be weighed against the risks of serious complications associated with AHSCT, and this treatment should only be offered in specialist centres experienced both in MS treatment and stem cell therapy, or as part of a clinical trial," noted trial researcher Dr Freedman.
"Future research will be directed at reducing the risks of this treatment as well as understanding which patients would best benefit from the treatment."