A new clinical trial, published in JAMA Neurology, has demonstrated a stem cell therapy that ‘resets’ the immune system and prevents disease progression in patients with multiple sclerosis (MS). Autologous hematopoietic stem cell transplantation (AHSCT) prevented worsening of symptoms of severe MS for 5 years in 46% of patients tested, indicating that this could be a promising new treatment avenue, particularly for patients who otherwise do not respond to other therapies. However, the treatment involved aggressive chemotherapy that iself carries a significant risk.
In the study, AHSCT was used in 281 patients with advanced MS who had not responded to other forms of treatment. While symptoms were ‘frozen’ in 46% of patients, and scientists reported that symptoms improved in some patients with relapsing–remitting MS, eight patients died in the 100 days following the treatment. Older patients and those with the most severe advanced forms of MS were found to have the highest risk of death.
“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 5 years, explained lead author Paolo Muraro (Imperial College London, UK). "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.”
AHSCT was found to be particularly beneficial in patients with relapsing–remitting MS, who survived without worsening symptoms for significantly longer than would be expected in untreated patients. In patients with relapsing MS, 73% experienced no worsening of symptoms 5 years after treatment. Younger patients will less advanced MS were more likely to respond to treatment.
The majority of patients in this study had progressive MS. There are currently no treatments for this form of the disease. In patients with progressive MS, one in three experienced no worsening of symptoms 5 years after treatment.
“These findings are very promising – but crucially we didn’t have a placebo group in this study, of patients who didn’t receive the treatment,” added Muraro. “We urgently need more effective treatments for this devastating condition, and so a large randomised controlled trial of this treatment should be the next step.”
Sources: Muraro PA, Pasquini M, Atkins HL et al. Long-term outcomes after autologous hematopoietic stem cell transplantation for multiple sclerosis. JAMA Neurol. doi:10.1001/jamaneurol.2016.5867 (2017) (Epub ahead of print); www3.imperial.ac.uk/newsandeventspggrp/imperialcollege/newssummary/news_20-2-2017-17-40-27