Stem Cell Therapy May Delay Disability Longer in Multiple Sclerosis

Multiple Sclerosis (MS) is a chronic inflammatory, autoimmune, and neurodegenerative disease of the central nervous system (CNS). MS affects approximately 2.5 million people worldwide. High prevalence of MS is seen in northern parts of Europe and North America.

Multiple sclerosis (MS) is a potentially disabling disease of the brain and spinal cord (central nervous system).

In MS, the immune system attacks the protective sheath (myelin) that covers nerve fibers and causes communication problems between your brain and the rest of your body. Eventually, the disease can cause permanent damage or deterioration of the nerves.

It is characterized by demyelination and neuronal loss that is induced by attack of autoreactive T cells to the myelin sheath and endogenous remyelination failure, leading to functional neurological disability. Its very complex pathogenesis is not completely understood. 

While most people with MS are first diagnosed with relapsing-remitting MS, marked by symptom flare-ups followed by periods of remission, many people with relapsing-remitting MS eventually transition to secondary progressive MS, which does not have wide swings in symptoms but instead a slow, steady worsening of the disease.

Hematopoietic stem cell transplants may delay disability longer than some other MS medications

Matilde Inglese, MD, PhD, of the University of Genoa in Italy and a member of the American Academy of Neurology and her colleagues did a retrospective study included 79 people with active secondary progressive MS who received stem cell transplants and 1,975 people from the Italian MS registry who were treated with MS drugs. All received treatment after being diagnosed with active secondary progressive MS. 

The two groups were matched for age, sex and level of disability. Drugs included beta-interferons, azathioprine, glatiramer acetate, mitoxantrone, fingolimod, natalizumab, methotrexate, teriflunomide, cyclophosphamide, dimethyl fumarate and alemtuzumab.

Participants’ level of disability was measured on the Expanded Disability Status Scale, a common method to quantify disability with scores ranging from 0, no symptoms, to 10 points, death due to MS. Participants were assessed at various time points over 10 years.

At the beginning of the study, participants had a median score of 6.5 for both those who received transplants and those receiving the medications. Scores of 6.0 are defined as needing to use a cane or brace intermittently or on one side to walk about 100 meters with or without resting. Scores of 6.5 are defined as needing to use a cane or brace constantly on both sides to walk about 20 meters without resting.

Five years into the study, researchers found 62% of the people who had stem cell transplants experienced no worsening of their MS disability compared to 46% of those who took medications.

Also, at five years, researchers found people who received stem cell transplants were more likely to see sustained improvements over time, with 19% experiencing less disability than at the start of the study, compared to just 4% of people taking medications.

Over 10 years, the disability score for people who had stem cell transplants decreased by an average of 0.01 points per year, signifying less disability, while the average score for people taking medications increased by 0.16 points per year, an increase in disability.

While these results are encouraging, they are not applicable to patients with secondary progressive MS who do not have signs of inflammatory disease activity; more research is needed in larger groups of people to confirm these findings.


Giacomo Boffa et al. Hematopoietic Stem Cell Transplantation in People With Active Secondary Progressive Multiple Sclerosis. Neurology, 2022 DOI: 10.1212/WNL.0000000000206750