Neural Stem Cell Transplantation for Progressive MS

Research Summary: Neural stem cell transplantation in patients with progressive multiple sclerosis: an open-label, phase 1 study

The treatment of progressive multiple sclerosis remains an unmet need.  Today, we are going to summarise a recent publication that has identified a new potential therapy for this purpose.

What?

The article described the results of a Phase I clinical trial called STEMS, which investigated the therapeutic potential of injecting neural stem cells into the spinal cords of people living with progressive MS.

Who?

This research was conducted by a team at the IRCCS San Raffaele Scientific Institute in Milan, Italy and involved other collaborating institutes throughout Italy.

Where?

The article was published in Nature Medicine.  The full-text is currently freely available and can be viewed here.

When?

The study was published online in January, 2023.

BACKGROUND #1:  Currently, there are very few treatments that are approved for progressive multiple sclerosis.  Those that are approved generally provide small benefits, though this is significant in an environment where other options don’t exist.

BACKGROUND #2:  Neural stem cells have the ability to become many different cell types in the nervous system.  This gives them the potential to be important in repairing the structure and function of damaged nerves.

BACKGROUND #3:  Neural stem cells have shown positive results in animal models (both mouse and primate) of multiple sclerosis.

FINDING #1:  The trial involved 12 people living with progressive multiple sclerosis (5 with secondary progressive and 7 with primary progressive).  The participants were split into 4 groups that received different doses of the neural stem cells.

FINDING #2:  The primary finding of this initial trial was that the procedure was safe and well-tolerated by the participants.  At 3 years post stem cell transplant, all participants had survived and reported no serious adverse events that could be linked to the treatment.

FINDING #3: While safety was the primary aspect to be tested in this preliminary trial, some interesting findings were also made around the therapeutic potential.  A significant reduction in brain volume loss was observed, which was associated with the dose of cells transplanted (i.e. participants that received the highest dose had the lowest amount of brain volume loss).

There is always a lot of excitement whenever there is a new study that suggests the development of a novel treatment for people living with progressive MS.  This study has suggested that the injection of neural stem cells could have potential, but there are still some important points that need to be considered.

THOUGHT #1: The results from this trial are based on a very small number of participants and only limited amounts of data were able to be collected after the initial assessment.  This is not unexpected, as this trial was largely aimed at determining whether the procedure would be safe to explore further.  Having seen that it is, it is worth following up with larger numbers of participants to get a better idea of how effective it can be as a treatment for progressive MS.

THOUGHT #2:  It will be important to understand whether or not any of the observed benefits are from the cells themselves, or some factor that they release.  This will help guide the development of even more targeted therapies in the future, which may not only provide greater benefits, but also have less side-effects.

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