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Stem Cell Therapy for MS

Stem cell replacement therapy generates a lot of talk in the multiple sclerosis community. When people refer to stem cell treatments for MS, they usually mean a procedure called autologous hematopoietic stem cell transplantation (aHSCT). It is the most studied stem cell therapy for MS.In aHSCT, blood stem cells from bone marrow replace cells in the immune system that are mistakenly attacking the nervous system. In this way, the stem cell transplant “reboots” the immune system to slow or halt MS activity.AHSCT is a one-time procedure. It can greatly reduce and potentially end MS disease activity in some people.
A painting showing a stem cell making a copy of itself. An arrow shows that it could also turn into cells such as muscle, skin and nerve cells.

What Happens During AHSCT Treatment

How AHSCT Works

What Happens After AHSCT Treatment

What To Know About Stem Cell Therapy for MS

  • The benefits of aHSCT vary from person to person. Some people who have had aHSCT have gone into long-term remission. Some have even seen their symptoms go away. Others have had MS lesions and relapses return after a period of time.

  • The main risk of aHSCT comes from the chemotherapy treatments. Depleting the immune system makes people vulnerable to infections. These can be life-threatening.Practitioners and researchers debate how intense the chemotherapy for aHSCT should be. Stronger chemotherapy regimens have more health risks, but the benefits appear to last longer.Factors associated with increased risk of life-threatening side effects following aHSCT include:
    • Older age
    • Greater disability
    • Certain health conditions (for example, heart or lung disease)
    Chemotherapy can also cause infertility in women. Your provider may advise you to harvest your eggs if you want to become pregnant in the future.

  • Start by talking to an MS specialist with knowledge of aHSCT. Discuss benefits and risks of aHSCT for your MS, and review alternative options for treatment. Because aHSCT involves suppressing your immune system, there is a risk of serious infection.Studies show that aHSCT can be safe and effective in people who:
    • Have relapsing-remitting MS. Research shows that aHSCT is most effective for active RRMS as it targets ongoing inflammation, acting as an anti-inflammatory. aHSCT cannot regrow damaged nerves or repair damaged myelin.
    • Are younger than age 50. Older people can have weaker immune systems and other health conditions (like heart disease or other age-related diseases) that may make them more susceptible to complications from aHSCT.
    • Were diagnosed with MS less than 10 years ago. People who have had the disease longer may have more disability, which can make the aHSCT procedure more dangerous.
    • Are unable to take high-efficacy disease-modifying therapies(DMTs)*.
    • Have not responded to high-efficacy DMTs. (This means they are still getting new lesions and/or having relapses despite treatment with a high-efficacy DMT).
    *High-efficacy DMTs: alemtuzumab (Lemtrada®), natalizumab (Tysabri®), ocrelizumab (Ocrevus®), ofatumumab (Kesimpta®), rituximab (Rituxan®), or ublituximab-xiiy (Briumvi™). Some MS specialists include cladribine (Mavenclad®) in this group.

Finding Stem Cell Treatment for MS

  • FACT website. Clinical services vary. Also, not all FACT-accredited centers perform aHSCT for MS.Your MS healthcare provider can help you select a treatment center and identify which healthcare providers should be involved in your care during and after aHSCT." c-nmssatomrichtext_nmssatomrichtext-host="">
    It is crucial to get aHSCT therapy at an accredited center. Look for one accredited by the Foundation for Accreditation of Cellular Therapy (FACT). FACT has a strict set of quality and safety standards. The National Multiple Sclerosis Society recommends that aHSCT treatment center transplant teams include: 
    • Neurologists who are MS experts
    • Hematologist-oncologists experienced in performing aHSCT on people with MS
    Search for aHSCT centers on the FACT website. Clinical services vary. Also, not all FACT-accredited centers perform aHSCT for MS.Your MS healthcare provider can help you select a treatment center and identify which healthcare providers should be involved in your care during and after aHSCT.

  • Because the aHSCT protocol can vary, learn everything you can about the center that would perform it. Prepare questions for the center and discuss the answers with your MS specialist before moving forward. The questions below can serve as a starting point.The Center
    • Is the center and its cell production facility accredited by the Foundation for the Accreditation of Cellular Therapy (FACT) in the United States?
    • Does the center have experience working with people with MS?
    • What specialists are included on the treatment team?
    • How many aHSCT procedures have they performed on MS patients?
    • What have the outcomes been?
    The Procedure
    • What kind of procedure do they propose? The most studied type of stem cell therapy for MS is aHSCT.
    • What type of cells will they use for the procedure and why? Stem cells specific to certain tissues cannot easily make cells found in other tissues. It is very unlikely that the same stem cell treatment will work for diseases affecting different tissues and organs within the body.
    • Can they provide studies published in peer-reviewed medical journals that show evidence that the stem cell types and techniques they use have benefits?
    • Will the stem cells come from you or a donor? Life-threatening complications such as graft-versus-host disease or tumors are less likely when the stem cells are “autologous,” meaning they come from your own body.
    • What procedures are used to ensure sanitary conditions and that the cells are free from contaminants and infectious agents?
    After the Procedure
    • Do they provide immediate and long-term follow-up care?
    • What are the benefits of the procedure? 
    • How will those benefits be measured long term?
    • What are the possible short- and long-term side effects and risks? How are they monitored? Will the center provide you with a detailed consent form outlining the procedure, side effects and risks for you to review and sign?
    • How will potential complications be addressed? Will complications be covered by your health insurance?

  • The average total cost of care for inpatient aHSCT in the U.S. is $150,000. This usually includes the care required before and after the procedure. However, the cost of aHSCT varies widely. Prices are based on the patient and their treatment plan.Most insurance companies will probably not cover this procedure. Expect to pay for it out of pocket or enroll in a clinical trial where the costs are covered by the trial.

  • View aHSCT studies in people with MS, such as the BEAT-MS study." c-nmssatomrichtext_nmssatomrichtext-host="">
    Some people may qualify to have aHSCT through a clinical trial. Many people who have undergone aHSCT have been enrolled in a trial. View aHSCT studies in people with MS, such as the BEAT-MS study.

Learn More About Stem Cell Therapy and MS

What’s Ahead for Stem Cell Therapies and MS