A clinical trial led by Imperial College London has revealed the potential of a risky new therapy to stop the progression of multiple sclerosis.
Worldwide, there are 2.3 million people with multiple sclerosis (MS), a disease caused by a defective immune system that attacks neurons in the brain and spinal cord. Currently, there are medications that can slow the progression of MS, but none can stop it or cure it.
A study led by Imperial College London and published in JAMA Neurology showcases a new treatment that could be life-saving for some, but deadly for others. The therapy prevented symptoms from worsening in 46% of patients for a span of at least 5 years. However, out of the 281 patients enrolled, eight of them died in the following months, the risk being higher for those older and with more severe forms of MS.
The treatment was autologous hematopoietic stem cell transplantation (AHSCT), a procedure where the patient’s own bone marrow stem cells are harvested and transfused back after eliminating any remaining immune cells with chemotherapy. Previous studies have suggested the transfused cells regrow a “reset” immune system that does not attack neurons anymore.
“In this study, which is the largest long-term follow-up study of this procedure, we’ve shown we can “freeze” a patient’s disease, and stop it from becoming worse, for up to five years,” said Paolo Muraro, lead author of the study. “However, we must take into account that the treatment carries a small risk of death, and this is a disease that is not immediately life-threatening.”
The study found that the treatment was more effective in patients with relapsing MS, a form of the disease characterized by flare-ups followed by periods with improved symptoms. “The treatment is most effective in people with MS who have “active inflammation” in their brain and spinal cord,” commented Sorrel Bickley, Head of Biomedical Research at the MS Society, one of the funding agencies behind the study.
Further studies could help determine in more detail which patients can benefit from this therapy and which are at risk. Muraro added that larger trials including placebo groups are the next step, so we might be hearing from his team again in the coming years.
Meanwhile, biotechs are working on alternatives to better treat patients with MS, such as antibodies developed by Geneuro, now in Phase IIb; shark-inspired antibodies from Ossianix; and personalized multi-omics funded by the EU Horizon 2020.
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