Repairing MS-caused damage with stem cells

Results of a investigator-initiated Phase I stem cell therapy trial  have shown promise as a treatment for progressive multiple sclerosis. The injection neural donor stem cell into the brains of patients living with progressive multiple sclerosis (MS) was demonstrated to be safe, well tolerated and to show a long-lasting effect that appears to protect the brain from further damage.

In progressive forms of MS, a particular type of macrophage attack the central nervous system (CNS), causing chronic inflammation and damage to nerve cells. Previous work from the British research team has shown in mice that skin cells re-programmed into induced pluripotent stem cells,  differentiated into brain stem cells, and then transplanted into the central nervous system, can help reduce inflammation and may be able to help repair damage caused by MS.

Now, the researchers headed by Angelo  Vescov injected (a intracerebroventricularly) allogeneic foetal neural stem cells directly into the brains of 15 patients with secondary MS recruited from two hospitals in Italy. The trial was conducted by teams at the University of Cambridge, Milan Bicocca and the Hospitals Casa Sollievo della Sofferenza and S. Maria Terni  (IT) and Ente Ospedaliero Cantonale (Lugano, Switzerland) and the University of Colorado (USA).

They observed no treatment-related deaths or serious adverse events. While some side-effects were observed, all were either temporary or reversible. No patient showed any increase in disability or a worsening of symptoms. None of the patients reported symptoms that suggested a relapse and nor did their cognitive function worsen significantly during the study. Overall, say the researchers, this points to a substantial stability of the disease, without signs of progression, though the high levels of disability at the start of the trial make this difficult to confirm.

In a subgroup, the researchers found that the larger the dose of injected stem cells was, the smaller the reduction in this brain volume over time. They speculate that this may be because the stem cell transplant dampened inflammation. Looking for signs that the stem cells were having a neuroprotective effect they found a dose-dependent increase in fatty acids, which persisted over 12-month follow up, and increased levels of acyl-carnitines cerebrospinal fluid (CSF)..

Professor Stefano Pluchino from the University of Cambridge, who co-led the study, said: “I am cautiously very excited about our findings, which are a step towards developing a cell therapy for treating MS.nThis 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.”