The National MS Society reviewed evidence on the use of autologous hematopoietic stem cell transplantation (aHSCT) for the treatment of multiple sclerosis and concluded that aHSCT is a good treatment option for some people, specifically for people who have very aggressive relapsing-remitting MS who have not benefitted from disease modifying therapies (DMTs).
AHSCT is a type of bone marrow transplantation that attempts to reset the immune system, which is responsible for damaging the brain and spinal cord in MS. The stem cells used during the procedure are ‘autologous’ meaning they are derived from your own body.
AHSCT is a one-time procedure that can significantly reduce and potentially eliminate 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.
How to know if aHSCT is right for you
An MS specialist with knowledge of aHSCT can help you understand whether aHSCT is right for you, discuss benefits and risks and review alternative options for treatment.
Studies show 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 breakthrough disease activity (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 including older age, greater disability and certain health conditions (for example, heart or lung disease) are associated with increased risk of life-threatening side effects following aHSCT.
*High-efficacy DMTs: natalizumab (Tysabri®), alemtuzumab (Lemtrada®), ocrelizumab (Ocrevus®), rituximab (Rituxan®), or ofatumumab (Kesimpta®). Some MS specialists consider cladribine (Mavenclad®) in this group.
What happens during treatment
The initial stem cell preparation and harvest takes 5 to 15 days. Then there is a 3-week hospital stay to prepare your immune system for the transplant, perform the stem cell transplant and to allow time for you to recover.
There is no agreed upon aHSCT protocol for MS. Protocols differ in how stem cells are harvested and manipulated and what immunosuppressive medications are used during the procedure – more intense immunosuppression may result in better long-term disease control, but may be associated with a greater risk of infection or other complications. Research is underway to determine whether less intensive immunosuppressive protocols produce effective results and fewer side effects.
We recommend having the protocol reviewed by your MS specialist before moving forward with 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 wellbeing
You should expect frequent follow up appointments during the first 2 years after aHSCT. MRIs need to be performed within 6 months of aHSCT and at least annually thereafter. You will need to isolate, wear a mask and socially distance for a period of time following the procedure to reduce the risk of infections including COVID-19.
The outcomes of your treatment and information learned from your follow up appointments will further inform how aHSCT works in MS.
Cost of aHSCT and health insurance coverage
The average total cost of care for inpatient aHSCT is $150,000. However, widespread variations in cost exist across the United States with 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 initially 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 contactusnmss@nmss.org for a supportive partner in working through this process.
Finding an aHSCT treatment center
It is crucial that aHSCT is performed at a treatment location that is 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 and identify which healthcare providers should be involved in your care during and after aHSCT.
View all aHSCT centers in the United States 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.
Current aHSCT clinical trial
There is currently one clinical trial on aHSCT in MS in the United States. The BEAT-MS clinical trial is randomly assigning eligible participants to undergo aHSCT or receive certain DMTs to compare the costs, safety and effectiveness of the two treatments. For study details and to locate the site nearest you, visit the study website.
The National MS Society is here to help
For help finding an MS specialist, an aHSCT center or understanding your health insurance coverage, please contact an MS Navigator at 1-800-344-4867 or contactusnmss@nmss.org.