Early-stage stem cell therapy shows efficient results in trials for treating Progressive Multiple Sclerosis


Source: University of Cambridge Research News | Creative Commons CC


An international team of researchers has demonstrated that injecting a particular kind of stem cell into the brains of people with progressive multiple sclerosis (MS) is safe, well-tolerated, and has a long-lasting impact that seems to shield the brain from further injury.

Leading researchers from the University of Cambridge, University of Milan Bicocca, and Hospital Casa Sollievo della Sofferenza (Italy) conducted the study, which represents a step toward the development of an advanced cell therapy treatment for progressive multiple sclerosis.

Around 2 million people worldwide suffer from multiple sclerosis (MS). Although there are treatments that may reduce the severity and frequency of relapses, two-thirds of MS patients still enter a crippling secondary progressive phase of the disease within 25 to 30 years of diagnosis, during which their disability gradually worsens.

The protective wrapping surrounding nerve fibres, known as myelin, is attacked and damaged by the body’s immune system in multiple sclerosis (MS), disrupting communications that travel through the brain and spinal cord.



Macrophages are important immune cells in this process; normally, they attack and eliminate foreign objects from the body. The brain and spinal cord are filled with microglial cells, a specific kind of macrophage. They target the central nervous system (CNS) in progressive types of multiple sclerosis (MS), resulting in long-term inflammation and nerve cell destruction.

Expectations that stem cell treatments may help mitigate this damage have increased due to recent developments. These entail the transplantation of stem cells, sometimes referred to as the body’s “master cells,” which possess the ability to differentiate into nearly every kind of cell found in the body.

Prior research by the Cambridge team has demonstrated that skin cells that have been reprogrammed to become brain stem cells and then implanted into the central nervous system can help lower inflammation and possibly even assist in healing damage from multiple sclerosis in animals.

Researchers have now finished the first-in-human, early-stage clinical experiment, which involves directly injecting neural stem cells into the brains of fifteen patients with secondary MS who were chosen from two Italian hospitals. The results of the study were published in the Cell Stem Cell. Teams from the University of Colorado (USA), the Hospitals Casa Sollievo della Sofferenza and S. Maria Terni (IT), Ente Ospedaliero Cantonale (Lugano, Switzerland), and the University of Cambridge, Milan Bicocca, performed the trial.

The stem cells originated from brain tissue harvested from a single miscarried fetus donor. In order to address the practical issues surrounding the use of allogeneic foetal tissue, the Italian team had previously demonstrated that it would be possible to produce an almost infinite supply of these stem cells from a single donor. In the future, it may also be possible to derive these cells directly from the patient.

Over a 12-month period, the team monitored the patients and saw no significant adverse events or treatment-related fatalities. Although some negative effects, they were all transient or easily remedied.



At the beginning of the experiment, all of the patients had severe degrees of impairment; for example, the majority used wheelchairs. However, none of the patients had any worsening of their symptoms or rise in disability during the course of the 12-month follow-up period. Throughout the trial, none of the patients experienced any symptoms that would indicate a relapse and their cognitive ability did not deteriorate noticeably. Overall, the researchers conclude that there are no indications of disease progression, notwithstanding the difficulty in verifying this due to the high levels of impairment at the experiment’s inception.

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A subset of individuals was evaluated by the researchers for alterations in brain tissue volume linked to the advancement of the disease. They discovered that the gradual decrease in this brain volume was less than higher the dosage of transplanted stem cells. It is hypothesized that this might be the result of the stem cell transplant reducing inflammation.

Additionally, the group searched for indications that the stem cells were preventing additional harm to nerve cells, or exerting a neuroprotective effect. Their earlier research has shown how altering the body’s energy production process, or metabolism. This latest study examined the post-treatment alterations in the metabolism of the brain. They tracked alterations in the blood and fluid surrounding the brain over time and discovered specific indicators associated with the brain’s fatty acid metabolism. These symptoms were linked to the course of the illness and the efficacy of the therapy. Fatty acid levels increased with increasing stem cell dosage and continued to do so for the full 12-month duration.

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I am cautiously very excited about our findings, which are a step towards developing a cell therapy for treating MS

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.

Professor Stefano Pluchino, co-led the study

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.

Professor Angelo Vescovi from the University of Milano-Bicocca, co-leader

Journal Reference: Leone, M. A., Gelati, M., Profico, D. C., Gobbi, C., Pravatà, E., Copetti, M., Conti, C., Abate, L., Amoruso, L., Apollo, F., Balzano, R. F., Bicchi, I., Carella, M., Ciampini, A., Colosimo, C., Crociani, P., D’Aloisio, G., Di Viesti, P., Ferrari, D., . . . Vescovi, A. L. (2023). Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive multiple sclerosis. Cell Stem Cell. https://doi.org/10.1016/j.stem.2023.11.001


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