• About 2.8 million people worldwide have multiple sclerosis (MS) as of 2020.
• Two of the four different types of MS are progressive, meaning disability worsens over time.
• An early clinical trial shows that a therapy using a certain type of stem cell may help protect the brain from further damage in progressive cases of MS.

As of 2020, about 2.8 million people globally live with multiple sclerosis (MS) — a disabling neurological condition where the body’s immune system mistakenly attacks the protective covering of nerve fibers.

Of the four different types of MS, two are progressive. This means that a person’s disability worsens over time.

Between 10–15% of all MS diagnoses are primary progressive MS. And previous research shows that secondary progressive MS affects between one to 58 of every 100,000 people with MS.

While there are medications available for treating MS, not all work for those with progressive forms of the disease.

Now, a group of scientists from the University of Cambridge, the University of Milan Bicocca, and Hospital Casa Sollievo della Sofferenza in Italy, show that therapy using a certain type of stem cell may help protect the brain from further damage in progressive cases of MS.

This study was recently published in the journal Cell Stem Cell.

No increased disability after 12 months of stem cell therapy

For this early-stage clinical trial, researchers used neural stem cells taken from the brain tissue of a single, miscarried fetal donor.

The neural stem cells were then injected directly into the brains of 15 people with secondary MS. The study participants were recruited from two hospitals in Italy and the trial was conducted by teams in the United Kingdom, Italy, Switzerland, and the United States. All study participants had high levels of disability at the beginning of the trial.

After following participants for 12 months, researchers found that none of them showed an increase in disability or worsening of symptoms. Additionally, none of the participants reported symptoms suggesting a relapse or significantly worsened cognitive function.

“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,” said Dr. Stefano Pluchino, professor of regenerative neuroimmunology and honorary consultant in neurology in the Department of Clinical Neurosciences at the University of Cambridge, and co-lead author of this study, in a press release.

“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,” Dr. Pluchino added as part of his statement.

Stem cell therapy protects brain tissue volume, fatty acid levels

Researchers also assessed a subgroup of participants to look for changes in brain tissue volume associated with MS progression.

They reported that the larger the dose of injected neural stem cells, the smaller the reduction in brain volume over time. Researchers believe this may be because the stem cells help lessen inflammation.

And the scientists also looked to see if the stem cell therapy helped protect nerve cells from further disease damage.

During the study, researchers measured changes in the fluid around the brain and in the blood over time. They made observations that are linked to how the brain processes fatty acids.

Researchers were able to correlate those observations to how well the stem cell treatment works and how MS develops. They reported the higher the dose of injected neural stem cells, the higher the levels of fatty acids, which continued over the 12-month period.

In the press release, Dr. Angelo L. Vescovi, associate professor of applied biology in the Department of Biotechnology and Biosciences at the University of Milano-Bicocca, and co-lead author of this study, explained why this research constitutes an important step forward:

“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.”

The importance of new treatments for progressive MS

According to Dr. Ben Thrower, medical director of the Andrew C. Carlos MS Institute at the Shepherd Center in Atlanta, GA, and senior medical advisor for the Multiple Sclerosis Foundation, not involved in the current study, new treatments for progressive MS are needed because progressive MS in the absence of relapses and/or new MRI lesions has been a particularly challenging issue.

“Currently we have over 25 FDA [Food and Drug Administration]-approved disease-modifying therapies for relapsing forms of MS,” Dr. Thrower told Medical News Today. “We have one approved option for primary progressive MS and no approved therapies that reverse disability.”

“Since a majority of patients with multiple sclerosis attacks can eventually transition to a more progressive and debilitating form of the disease called secondary progressive MS and the majority of the currently available MS medications show minimal to no effect on this phase of the illness, having a therapy which shows no progression or relapses with no serious side effects is an extremely important development,” Dr. David Duncan, program director for the MS Center at the Hackensack Meridian Neuroscience Institute at Jersey Shore University Medical Center added.

Dr. Duncan was also not involved in this research.

Compelling and cautiously optimistic findings

After reviewing this study, Dr. Krupa Pandey, director of the Center for Multiple Sclerosis and Related Conditions at the Hackensack Meridian Neuroscience Institute at Hackensack University Medical Center, told MNT the direct implantation of stem cells in humans is a vital and groundbreaking step in the right direction to targeting reversal or cessation of progression of disease in MS.

“This study is also compelling because many studies in progressive MS focus on the early stages of the disease, enroll patients younger than 60, and mostly require ambulatory patients,” Dr. Pandey continued.

“This study enrolled older, wheelchair or bed-bound patients who had been afflicted with the disease for more than 15 years. Moreover, there are no agents currently available for advanced secondary progressive MS so a study like this is very exciting,” she told us.

MNT also spoke with Dr. Kalina Sanders, a board-certified neurologist at the Baptist Neurology Group for Baptist Health and a spokesperson for the National Multiple Sclerosis Society, who said she was cautiously optimistic about its results.

“I am cautious because this is a small study using a single donor,” Dr. Sanders detailed. “Additionally, the short duration of the study limits our knowledge of long-term effects for both safety and efficacy. The placement of an intraventricular catheter is an invasive procedure that may limit its widespread use. It is unclear if this is a therapy that can be expanded to a larger number of patients. Lastly, this is a fetal-derived cell line, which may introduce ethical concerns for some patients.”

“Nevertheless, I believe there needs to be an exploration of therapies that go beyond primary immunologic effects,” she added. “Therefore, I remain enthusiastic about the prospects of this therapeutic option.”

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