MS Experimental Treatment: First Phase Trial of Brain-Injected Stem Cells Shows Promise for Progressive Multiple Sclerosis

A stem cell-based therapy for the treatment of multiple sclerosis (MS) has shown promising results in a new clinical trial. The results of the trial were published in the journal Cell Stem Cell under the title “Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive multiple sclerosis”.

A well-tolerated treatment for multiple sclerosis

The study included 15 patients with secondary multiple sclerosis – the progressive phase of the disease – aged 38-57 years, all with a high level of disability.

MS Study Design

Credit: Cell Stem Cell

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The neural stem cells were injected directly into the patients’ brains and over the following 12 months, they were closely monitored for any side effects or changes in symptoms. They also had to take medication to suppress their immune system for half of the follow-up period.

The following year showed that the treatment was safe and well-tolerated: there were no deaths or serious side effects, and when side effects did occur, they were temporary or reversible. The most important finding of this trial is that none of the patients showed an increase in disability or worsening of symptoms.

Impact on brain metabolism

The team also monitored changes in the brain’s metabolism, the way it produces energy, over the 12 months, as previous research had shown that altering this metabolism could reprogram the immune cells that attack the central nervous system in MS. They found evidence that the stem cell therapy had affected metabolism and could therefore have an anti-inflammatory effect. The higher the dose of stem cells, the higher the levels of fatty acids, which are fundamental molecules in brain metabolism.
Whether this result is solely due to the neural stem cells needs to be confirmed by further tests, but the researchers believe this is the case.

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“I am cautious but very excited about our results, which represent a step towards the development of a cell therapy for the treatment of multiple sclerosis,” said Stefano Pluchino, co-leader of the study, in a press release. “However, we recognize that our study has its limitations. It was a small study and it is possible that the immunosuppressive drugs had interfering effects,” he concluded.

What are the current treatments for multiple sclerosis?

Multiple sclerosis is an autoimmune disease that affects the central nervous system. A dysfunction in the immune system leads to lesions that cause motor, sensory, cognitive, visual, and sphincter (usually urinary and bowel) disorders. Over time, these disorders can progress to irreversible disability.
Although current treatments can reduce relapses and improve patients’ quality of life, they are generally not effective enough to prevent the development of disability in the medium term.

Read Also: Multiple Sclerosis Breakthrough: Targeting Microglia in the Brain Could Help Reverse the Disease


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., Fogli, D., Fontana, A., Frondizi, D., Grespi, V., Kuhle, J., Laborante, A., Lombardi, I., Muzi, G., Paci, F., Placentino, G., Popolizio, T., Ricciolini, C., Sabatini, S., Silveri, G., Spera, C., Stephenson, D., Stipa, G., Tinella, E., Zarrelli, M., Zecca, C., Ventura, Y., D’Alessandro, A., Peruzzotti-Jametti, L., Pluchino, S., & 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.



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