Conceptual image of a multiple sclerosis neuron.
Credit: Stocktrek Images/Getty Images

A stem cell-based treatment for progressive multiple sclerosis (MS) achieved good results in a Phase I trial led by researchers at the University of Cambridge and the University of Milan Bicocca.

Almost one million people in the U.S. have MS, with around 200 new cases diagnosed every week. It is a chronic neuroinflammatory condition where the body’s immune system attacks its own myelin, the material that protects nerve cells from damage.

MS can be broadly split into relapsing-remitting MS, where those with the condition have spells of disease activity with neurological symptoms interspersed with no disease activity, and progressive MS where the symptoms continue to worsen over time. Many people who start with relapsing-remitting disease eventually transition to secondary progressive disease.

“Although progress toward the development of disease-modifying therapies has made a substantial impact on the severity and frequency of MS relapses, two-thirds of patients with MS still transition into a debilitating secondary progressive phase of disease within 25–30 years of diagnosis,” write co-lead investigator Stefano Pluchino, a professor at the University of Cambridge, and colleagues in the journal Cell Stem Cell.

“Most disease-modifying therapies have indeed shown only a marginal effect on disease progression and MS-associated rates of global and regional brain volume loss.”

The field is still in its infancy, but preclinical research shows neural stem cells can help reduce immune responses and help protect neurological function in animal models of MS.

“In vivo studies in rodent and non-human primate models of MS (or MS-like disease) have shown that both syngeneic and xenogeneic (human) somatic brain-specific neural stem/progenitor cells are safe and promote clinicopathological amelioration via multiple mechanisms of action,” explain the authors.

This study is the first in-human trial of a neural stem cell treatment for MS. The trial included 15 people with secondary progressive MS and high levels of disability recruited from two Italian hospitals who were injected with four different doses of donor human neural stem/progenitor cells into the brain. The patients were followed up for 12 months and monitored for adverse effects and disease activity.

No relapses or significant adverse effects were observed during the study or reductions in cognitive function.

This study was carried out mostly to assess safety, but some of the patients were also monitored for changes in brain tissue volume linked to their disease. The researchers found that participants who received higher doses of stem cells had a smaller reduction in brain volume over time.

It is hard to make definite conclusions, as the study was not powered to assess efficacy, but this may be due to a beneficial effect of the therapy on MS-related inflammation.

“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,” said Pluchino in a press statement.

“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,” added co-lead author Angelo Vescovi, a professor at the University of Milano-Bicocca, in the same statement.

The team now plans to develop the treatment further and aims to progress the therapy into later stage, larger clinical trials.

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