Cautious Excitement Follows First-in-Human Stem Cell Trial for Progressive MS

Fifteen patients with secondary progressive multiple sclerosis (MS) showed stability in lesion load, brain activity, and clinical and laboratory outcomes, with no serious adverse events, 12 months after receiving an injection of human stem cells into their brains. Researchers reported their findings in the journal Cell Stem Cell.

“The absence of adverse events and the stability of functional and structural outcomes are reassuring and represent a milestone for the safe translation of stem cells into regenerative medicines,” wrote corresponding authors Stefano Pluchino, MD, PhD, of the University of Cambridge in the United Kingdom, and Angelo L. Vescovi, PhD, of the University of Milano-Bicocca in Italy, and study coauthors.

The first-in-human, open-label, phase 1 trial included a total 15 patients with MS and high levels of disability from two hospitals in Italy. Participants received an intracerebroventricular injection of allogeneic human neural stem/progenitor cells in combination with an immunosuppressive regimen.

An analysis of 1-year data showed no serious adverse events or treatment-related deaths among participants. Any reported side effects were temporary or reversible, according to the study.

Furthermore, no participants showed any increase in disability, symptom worsening, or symptoms suggesting a relapse over the 12 months. There was also no evidence of significant worsening of cognitive function.

In a subgroup of patients assessed for changes in the volume of brain tissue associated with disease progression, researchers found larger doses of stem cells were associated with a smaller reduction in brain volume. They also found a time- and dose-dependent increase in cerebrospinal fluid fatty acids.

“I am cautiously very excited about our findings, which are a step towards developing a cell therapy for treating MS,” said Dr Pluchino.

“We recognize 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.”

References

Leone MA, Gelati M, Profico DC, et al. Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive multiple sclerosis. Cell Stem Cell. 2023;30(12):1597-1609.e8. doi:10.1016/j.stem.2023.11.001

Early-stage stem cell therapy trial shows promise for treating progressive MS. News release. University of Cambridge; November 27, 2023. Accessed December 11, 2023.