Stem cell therapy for multiple sclerosis

Multiple sclerosis (MS) occurs when your immune system mistakenly attacks the fatty tissue (myelin) that covers the nerve fibers in your brain and spinal cord. When myelin is damaged, nerve signals are interrupted, causing a variety of symptoms like numbness and weakness.

Current MS-modifying drugs help reduce relapses or “flare-ups” of symptoms and slow the natural progression of the disease.

Stem cell therapies have the potential to hold more promise in putting patients into long-term remission and / or repairing damage caused by the disease.

This article will discuss the risks, benefits, and research behind two types of stem cell treatments used or explored in MS care. These treatments include autologous hematopoietic stem cell transplant and mesenchymal stem cell transplant.


What is stem cell therapy for MS?

A stem cell is a single cell that can divide and replicate or transform into a specialized cell type like a nerve or a blood cell. Stem cell therapy is any treatment that uses stem cells to relieve or treat a disease.

Autologous hematopoietic stem cell therapy (aHSCT)

Autologous hematopoietic stem cell therapy (aHSCT) is a type of bone marrow transplant that attempts to ‘reset’ a person’s immune system using hematopoietic (hematopoietic) stem cells taken from their own body.

What cells can hematopoietic stem cells form?

Hematopoietic stem cells give rise to red blood cells, white blood cells and platelets.

The main goal of aHSCT is to suppress a patient’s overactive immune system and create a new, healthy one. This treatment works best for patients with relapsing-remitting MS whose disease continues to be very active and aggressive despite the use of various disease-modifying drugs.

Since aHSCTs are still considered experimental for patients with MS, doctors use slightly different protocols when performing them.

That said, the basic steps of aHSCT include:

  • Preperation: You are given medicines to help your body make more hematopoietic stem cells. You may also be given medicines to help the stem cells pass from your bone marrow to your bloodstream.
  • Collection: The hematopoietic stem cells are taken from your bloodstream and frozen in the laboratory.
  • Deletion: You receive chemotherapy in the hospital over a period of several days to suppress your immune system.
  • Transplantation: Frozen hematopoietic stem cells are thawed and put back into your bloodstream.
  • Rebuild: The transplanted stem cells pass from your bloodstream to your bone marrow and your immune system begins to rebuild.

Mesenchymal stem cell transplant

Mesenchymal stem cells are found in many tissues in your body, including bone marrow, skin, and fatty tissue.

These cells are believed to have regenerative properties, providing support for the tissue’s natural repair process if that specific tissue is damaged. They can also help reduce inflammation and protect nerves from damage.

Mesenchymal stem cell transplantation first involves isolating stem cells from a patient’s fat, skin, or bone marrow. The cells are then multiplied in the laboratory or treated with special factors and reintroduced into the body using various methods (for example, by injecting them into the bloodstream or through the spinal canal).

Once back in your body, the cells can calm your immune system and help repair damaged myelin in your brain and spinal cord.

Myelin repair in progressive MS

Promoting myelin repair in MS may be a particularly useful treatment option for patients with progressive MS. These patients experience little or no “relapses,” but their symptoms slowly worsen over time and they become increasingly disabled.

Risks and Benefits

Research studies on autologous hematopoietic stem cell transplants in MS have always found the procedure beneficial. The security of the procedure has also improved over the years.

A meta-analysis evaluated over 700 transplant patients from 15 different trials. After collating all the data, investigators found that 83% of patients had no signs of disease activity two years after the transplant.

In a 2021 study, 71% of participants with relapsing-remitting MS and 57% of participants with progressive MS continued to experience no worsening of their disability 10 years after their transplant.

The risk of undergoing aHSCT is that when the immune system is suppressed, patients are vulnerable to life-threatening bacterial, viral and fungal infections.

In addition, having a transplant is expensive and time consuming. Patients usually stay in the hospital for three weeks and they may experience unpleasant side effects from the chemotherapy.

Research on mesenchymal stem cell transplant is still in its very early stages. Most of the studies have been done in animals, although there are ongoing and completed clinical trials in humans. Preliminary results seem to suggest that this therapy is safe and possibly beneficial.

In a phase 2 clinical trial, 16 patients with primary progressive or secondary progressive MS underwent three injections of mesenchymal stem cells into their spinal canal every two months. The stem cells had been treated with factors designed to help nerve cells grow and survive.

The results revealed that some of the patients in the study exhibited improvements in mobility, finger dexterity, and cognitive and visual dysfunctions. Headache and back pain from lumbar punctures were the most frequently reported side effects. No deaths or treatment-related adverse events due to worsening MS occurred during the study.

However, larger and longer-term studies and studies with control groups are needed to adequately determine whether mesenchymal stem cell transplants are safe and effective.

Future steps and development

For autologous hematopoietic stem cell transplants, the next big step is to compare a transplant to taking a disease-modifying drug, in terms of inducing long-term remission of MS and improving quality of life.

A clinical trial called BEAT-MS is already looking to do this by comparing aHSCT to some of the most effective disease-modifying therapies used to treat recurrent MS.

The study follows 156 patients over six years. The MS medicines being compared include Tysabri (natalizumab), Lemtrada (alemtuzumab), Ocrevus (ocrelizumab), and Rituxan (rituximab).

For mesenchymal stem cell transplants, it’s really about doing more human studies to find out what issues like the easiest, safest, and most efficient cells to use (if any). The best way to deliver mesenchymal stem cells to patients also requires further investigation.


Stem cell treatments involve the use of self-replicating cells, or cells that can transform into a specialized type of cell, to treat a disease. Autologous hematopoietic stem cell and mesenchymal stem cell transplants are experimental stem cell treatments used or explored in the care of MS.

Research is much more advanced for autologous hematopoietic stem cell transplants and indicates that the procedure is effective and safe for patients with highly active relapsing-remitting MS.

A word from Verywell

Research into stem cell therapy in MS offers hope, particularly for those who do not respond to current disease-modifying drugs or for those with progressive MS (which most disease-modifying drugs do not respond to). cannot be used to treat).

That said, stem cell therapies for the treatment of MS are not yet approved by the Food and Drug Administration. If you are planning to undergo stem cell therapy, it is important to do so only at an accredited center with significant expertise or as part of a clinical trial.

Frequently Asked Questions

  • How Much Does Stem Cell Treatment Cost for MS?

    In the United States, the average cost per stem cell treatment is $ 7,000 to $ 10,000.

  • Are stem cells safe?

    Safety depends on the specific stem cell therapy being performed. With a hematopoietic stem cell transplant, there is a risk of life-threatening infections when your immune system is weakened.

  • What is the success rate of stem cell therapy?

    For autologous hematopoietic stem cell transplants, research has shown the procedure to be effective in inducing long-term remission in patients with MS. In a study, 71% of study participants with relapsing-remitting MS and 57% of participants with progressive MS continued to experience no worsening disability 10 years after their transplant.

About Antoine L. Cassell

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