Stem-cell therapy hope for progressive multiple sclerosis

Injecting stem cell into the brains of patients with progressive multiple sclerosis is safe and seems to protect the brain from further damage, a new study has found.

Researchers from the University of Cambridge, Milan Bicocca and the Hospitals Casa Sollievo della Sofferenza and S. Maria Terni, Italy, Ospedaliero Cantonale, in Lugano, Switzerland, and the University of Colorado, USA, hope their findings mean an advanced cell therapy treatment for progressive MS is a step closer.

In MS, the body’s immune system attacks and damages myelin, with the key immune cells being macrophages.

Microglial cells, a type of macrophage, are found throughout the brain and spinal cord but in progressive forms of MS, they attack the central nervous system (CNS), causing chronic inflammation and damage to nerve cells.

Recent studies have suggested stem cell therapies might help ameliorate this damage and the Cambridge team has shown that in mice, skin cells re-programmed into brain stem cells and transplanted into the central nervous system can help to reduce inflammation and may help repair the damage caused by MS.

In this latest study, published in Cell Stem Cell, the team finished the first-in-human, early stage clinical trial with patients recruited from two Italian hospitals.

Each of the 15 patients with secondary MS had neural stem cells injected directly into their brains. The stem cells were derived from cells taken from brain tissue from a single, miscarried foetal donor.

The patients were followed for 12 months and there were no treatment-related deaths or serious adverse events and any side-effects were either temporary or reversible.

At the beginning of the trial, all participants had high levels of disability, with most needing a wheelchair, and there was no increase in disability or a worsening of symptoms during the follow up period. None reported symptoms that suggested a relapse and their cognitive function did not worsen significantly during the study.

The researchers say these results point to a substantial stability of the disease.

When they assessed a subgroup of patients for changes in the volume of brain tissue associated with disease progression, they found that the larger the dose of injected stem cells, the smaller the reduction in this brain volume over time. This could be because the stem cell transplant dampened inflammation.

The team also looked for signs that the stem cells were having a neuroprotective effect, measuring changes in the fluid around the brain and in the blood over time. They found some signs that are linked to how the brain processes fatty acids, which were connected to how well the treatment works and how the disease develops. They found the higher the dose of stem cells, the greater the levels of fatty acids, which continued over the follow-up period.

Co-study leader Professor Stefano Pluchino from the University of Cambridge, said: “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.

“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.”

Co-leader Professor Angelo Vescovi from the University of Milano-Bicocca added: “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.”

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 27 November 2023; doi: 10.1016/j.stem.2023.11.001


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