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Could stem cell therapy stabilise MS progression?

Positive findings from a first-in-human trial means that an advanced cell therapy for progressive multiple sclerosis will be evaluated in Phase II.

Could stem cell therapy stabilise multiple sclerosis (MS) progression?

Injecting allogenic neural stem cells into the brains of patients living with progressive multiple sclerosis (MS) is safe and well tolerated, a a Phase I trial has shown, with the effects reported to last over 12 months. Findings from the study evaluating the advanced cell therapy treatment for progressive MS have been published in Cell Stem Cell. Recent development in the field suggest that stem cell therapies have neuroprotective capabilities, the researchers acknowledged.

Findings of the multiple sclerosis (MS) study

The stem cells used in the study were derived from cells taken from brain tissue from a single, miscarried foetal donor.

During the 12 month follow up period, none of the 15 patients demonstrated greater disability. None of the patients reported symptoms that suggested a relapse and nor did their cognitive function worsen significantly during the study.

A subgroup of patients was assessed for changes in the volume of brain tissue associated with disease progression. The more stem cells that were injected, the smaller the reduction in this brain volume over time. The researchers theorised that the stem cell transplant dampened inflammation.

As part of the research investigation, the study assessed how the brain’s metabolism changed after patients received the stem cell therapy. Changes in the fluid around the brain and in the blood were measured over time. It found certain signs that are linked to how the brain processes fatty acids. These signs were connected to how well the treatment works and how the disease developed.

Also, it was revealed that the higher the dose of stem cells, the greater the levels of fatty acids, which also persisted over the 12-month period.

The researchers concluded that the treatment facilitated “a substantial stability of the disease, without signs of progression”. Yet high levels of disability seen in patients at the beginning of the trial make this difficult to confirm, they stated. In the trial, some side-effects were observed however, these were either temporary or reversible.

Future potential of the stem cell therapy

[The researchers suggested that the stem cell treatment] approach might even stabilise [multiple sclerosis] disability progression”

“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,” Caitlin Astbury, Research Communications Manager at the MS Society commented.

As such, the findings are a step towards developing a cell therapy for treating MS, noted 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.”

This multiple sclerosis study will “pave the way to broader efficacy studies, soon to come,” Professor Angelo Vescovi from the University of Milano-Bicocca shared.

Recent developments in stem cell therapies