The National MS Society reviewed evidence on the use of autologous hematopoietic stem cell transplantation (aHSCT) for the treatment of multiple sclerosis. The Society concluded that aHSCT is a good treatment option for some people. It is most helpful for people who have very aggressive relapsing-remitting MS who have not benefitted from disease modifying therapies (DMTs).
In MS, the immune system mistakenly attacks the brain, spinal cord and optic nerves, damaging them. AHSCT is a type of bone marrow transplantation. It attempts to reset the immune system. The stem cells used during the procedure are “autologous” meaning they are from your own body.
AHSCT is a one-time procedure. It can greatly reduce and potentially end disease activity in some people. There are treatment risks including serious infections. Follow-up and supportive care after the procedure is critical to reduce these risks.
What happens during treatment
The graphic above shows the 6 steps of an aHSCT treatment plan, or protocol. Steps 1-3 involve collecting stem cells to transplant later. This process takes 5 to 15 days.
For the transplant (Steps 4-6), you would spend 3 weeks in the hospital preparing your immune system through chemotherapy, receiving the transplant and rebuilding your immune system.
There is no standard aHSCT protocol for MS. They differ in how stem cells are collected from the body and altered prior to returning them to the body. The immunosuppressive medications used during the procedure vary as well.
Immunosuppressive medications lower the body’s normal immune response and make it harder to fight infections. Stronger immunosuppression may lead to better long-term disease control and greater risk of infection. Scientists are working to find a protocol that:
- Works well
- Uses milder immunosuppressive medications
- Causes less side effects
Review the protocol with your MS specialist before having the procedure.
What happens after treatment
Follow-up appointments are critical after treatment and include:
- Medical, neurological and cognitive evaluations
- MRIs and blood tests
- Support for your mental health and well-being
You should expect frequent follow-up appointments during the first 2 years after aHSCT. MRIs need to be performed within 6 months of aHSCT. They need to be performed at least once a year thereafter.
To reduce risk of infections, like COVID-19, you will need to isolate, wear a mask and socially distance for a period of time after the procedure.
Providers will use what they learn from your response to aHSCT treatment to better understand how aHSCT works in MS.
How to know if aHSCT is right for you
An MS specialist with knowledge of aHSCT can help you understand whether the procedure is right for you. They can discuss benefits and risks and review alternative options for treatment.
Studies show that aHSCT may be a safe and effective treatment in people who:
- Have relapsing-remitting MS
- Are less than 50 years of age
- Have had MS for less than 10 years
- Have new inflammatory lesions on MRI and/or relapses despite treatment with a high-efficacy DMT* or are unable to take a high-efficacy DMT
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)
*High-efficacy DMTs: alemtuzumab (Lemtrada®), natalizumab (Tysabri®), ocrelizumab (Ocrevus®), or ofatumumab (Kesimpta®), rituximab (Rituxan®), or ublituximab-xiiy (Briumvi™). Some MS specialists consider cladribine (Mavenclad®) in this group.
Cost of aHSCT and health insurance coverage
The average total cost of care for inpatient aHSCT is $150,000. The cost of aHSCT varies widely across the United States. There are a variety of factors determining a patient's out-of-pocket spending. For insured patients, out-of-pocket spending for inpatient aHSCT is generally far less.
If your health insurance plan denies coverage for aHSCT as a treatment for MS, you can file an appeal. Connect with an MS Navigator at 1-800-344-4867 or firstname.lastname@example.org for help in working through this process.
Finding an aHSCT treatment center
It is crucial that aHSCT is performed at an accredited treatment location. Look for a center accredited by the Foundation for Accreditation of Cellular Therapy (FACT). Clinical services vary and not all FACT-accredited centers perform aHSCT for MS. Your MS specialist can work with you to select a treatment center. They can help identify which healthcare providers should be involved in your care during and after aHSCT. View all aHSCT centers in the U.S. on the FACT website. From the link, select your state and click “Start a New Search” to limit the search results to locations near you.
The National MS Society is here to help
For help finding an MS specialist or an aHSCT center, or in understanding your health insurance coverage, please contact an MS Navigator at 1-800-344-4867 or email@example.com.