Injecting stem cells into the brains of patients with multiple sclerosis (MS) is safe, well-tolerated, and has a long-lasting effect on protecting the brain from further damage, according to a study published in the journal Cell Stem Cell. The team from the University of Cambridge, UK, University of Milan Bicocca, and Hospital Casa Sollievo della Sofferenza, Italy, believe this is a key step in developing new treatments for progressive MS.
In MS, the immune system attacks myelin, which protects the cover around nerve fibres, leading to disruption when messages are sent around the brain and spinal cord. The condition affects over 2 million people around the world, but current treatments can only reduce the severity and frequency of relapses. Eventually, patients progress to a second phase within 25-30 of diagnosis, where symptoms worsen steadily.
In this study, British and Italian researchers completed the first clinical trial using stem cell technology, which involved injecting stem cells into the brains of 15 patients with secondary-stage MS. The stem cells were derived from cells taken from brain tissue from a miscarried foetal donor. “It has taken nearly three decades to translate the discovery of brain stem cells into this experimental therapeutic treatment This study will add to the increasing excitement in this field and pave the way to broader efficacy studies, soon to come,” said Professor Angelo Vescovi from the University of Milano-Bicocca.
The team then followed the patients for 12 months and observed no deaths directly caused by the treatment or serious side-effects. Some patients experienced mild side-effects but only temporarily. In addition, patients didn’t experience worsening symptoms or a decline in cognitive function during the study. Overall, the study showed that stem cells are stable and help patients stop the progression of symptoms.
“We desperately need to develop new treatments for secondary progressive MS, and I am cautiously very excited about our findings, which are a step towards developing a cell therapy for treating MS,” said Professor Stefano Pluchino from the University of Cambridge, who co-led the study. “We recognise that our study has limitations – it was only a small study, and there may have been confounding effects from the immunosuppressant drugs, for example – but the fact that our treatment was safe and that its effects lasted over the 12 months of the trial means that we can proceed to the next stage of clinical trials.”
“These results show that special stem cells injected into the brain were safe and well-tolerated by people with secondary progressive MS. They also suggest this treatment approach might even stabilise disability progression,” concluded Caitlin Astbury, Research Communications Manager at the MS Society. “This was a very small, early-stage study, and we need further clinical trials to find out if this treatment has a beneficial effect on the condition. But this is an encouraging step towards a new way of treating some people with MS.”
Leone, MA, Gelati, M & Profico, DC et al. Intracerebroventricular Transplantation of Foetal Allogeneic Neural Stem Cells in Patients with Secondary Progressive Multiple Sclerosis (hNSC-SPMS): a phase I dose escalation clinical trial. Cell Stem Cell; 7 Dec 2023; DOI: 10.1016/j.stem.2023.11.001