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While there’s currently no cure for multiple sclerosis (MS), autologous hematopoietic stem cell transplant (AHSCT) — sometimes called an autologous, or self, stem cell transplant — may help slow or stop MS disease activity in some people.
In AHSCT, doctors remove some of a person’s own blood-forming stem cells, give treatment to reduce immune cells, and then return the stem cells to help rebuild the immune system. The goal is to “reset” the immune system so it stops attacking the central nervous system (CNS), which includes the brain, spinal cord, and optic nerves.
Studies have found that AHSCT shows promise for some forms of MS. However, stem cell transplants in general aren’t approved by the U.S. Food and Drug Administration (FDA) for treating MS or other neurological conditions. Without FDA approval, many insurance companies won’t cover the costs, leaving stem cell transplants an expensive treatment that many can’t access.
This article will explore how AHSCT works for MS and what studies are saying about how well it may work. We’ll also cover the risks of AHSCT, costs, and insurance coverage.
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Stem cells are cells that can make copies of themselves and develop into more specialized cells. They help form and repair different parts of the body, including skin, blood, and nerve tissue.
Adult stem cells, also called somatic stem cells, are found in certain tissues and organs. They usually develop into cell types related to the tissue they come from. For instance, skin stem cells usually develop into skin cells but not brain cells.
Hematopoietic stem cells (HSCs) are blood-forming stem cells found in the bone marrow and blood. HSCs can develop into different types of blood cells, including red blood cells and white blood cells. White blood cells are part of the immune system, which mistakenly attacks myelin — the protective coating around nerves — in MS.
A hematopoietic stem cell transplant (HSCT) is a type of stem cell therapy that uses blood-forming stem cells to rebuild the immune system. You may also hear this treatment called a bone marrow transplant.
An autologous HSCT uses a person’s own stem cells. Using your own stem cells may lower the risk of certain complications that can happen with donor stem cells.
MS is an autoimmune disease that develops when immune cells mistakenly attack myelin in the CNS. Myelin is the protective coating around nerve fibers.
When immune cells attack myelin, it can cause inflammation and damage. This damage can disrupt messages between the brain and the rest of the body, leading to MS symptoms.
AHSCT aims to rebuild a new immune system using a person’s own blood-forming stem cells. The idea is that the rebuilt immune system will no longer attack the CNS. In this way, AHSCT is sometimes described as a reset for the immune system. It may help slow or stop MS disease activity in some people, especially those with forms of relapsing MS.
The National Multiple Sclerosis Society (NMSS) notes that every treatment facility has its own AHSCT protocol. However, the process usually follows the same general steps.
In an AHSCT, the blood stem cells come from your own bone marrow. You’ll take medications to release stem cells into your bloodstream. This process is called mobilization. During this step, your MS symptoms may temporarily flare up or get worse.
The extra blood stem cells are then collected from your bloodstream. A special machine separates the stem cells out from the rest of your blood. The stem cells are frozen and stored for later use.
The next step is to suppress your current immune system. You’ll have conditioning treatment, which often includes chemotherapy drugs, to reduce or destroy the immune cells thought to be involved in MS.
Finally, the collected blood stem cells will be infused back into your bloodstream. The stem cells travel to the bone marrow, where they start making new blood and immune cells. The goal is to rebuild, or “reset,” the immune system so it no longer attacks the CNS.
From the time you start chemotherapy until your new immune system has had time to recover, you’re at a high risk of getting sick. You’ll need to stay in the hospital for about three weeks. During this time, it’ll be important to take extra steps to protect yourself from infections.
Disease-modifying therapies (DMTs) are the standard treatment for MS. These medications can help reduce MS relapses, control disease activity, and slow disability progression.
However, not everyone responds to DMTs, and some people’s MS continues to worsen over time. Research suggests that AHSCT may be an option for some people with relapsing-remitting MS (RRMS), especially those with highly active disease.
We’re still learning about how safe and effective AHSCT is for MS. The Multiple Sclerosis International Stem Cell Transplant (MIST) trial compared people with RRMS who received an AHSCT with people who continued taking DMTs.
The study authors found that AHSCT was more effective than DMTs at slowing disease progression in this group of people with RRMS. During the study, three people who received AHSCT had disease progression, compared with 34 people who received DMTs. AHSCT also helped some people improve their MS-related disability scores.
The MIST trial suggests that AHSCT may help slow MS progression more than some DMTs for certain people with RRMS. However, AHSCT isn’t considered a standard treatment or a cure for MS.
The MIST trial showed that AHSCT may be effective for some people with RRMS. However, the study compared stem cell therapy with older DMTs, not newer highly effective DMTs (HE DMTs).
HE DMTs are stronger MS treatments that can be more effective at reducing relapses and slowing disease activity than some older DMTs. Examples include:
Newer clinical trials are now comparing AHSCT with HE DMTs to find out how their safety and effectiveness compare. The StarMS trial is an ongoing study in the United Kingdom comparing the two treatments in people with highly active RRMS.
The BEAT-MS study is comparing AHSCT with best available therapy (BAT) for people with treatment-resistant relapsing MS, meaning their MS is still active despite taking MS medication. People with RRMS or active secondary progressive MS (SPMS) may qualify for the BEAT-MS study.
AHSCT for MS is still being studied, and researchers are continuing to learn more about its short- and long-term risks and benefits.
As more studies are completed, doctors will have a clearer picture of who may benefit most from AHSCT and who may face higher risks.
One of the major complications of an AHSCT is infection. The chemotherapy drugs used during conditioning suppress your immune system, making it harder for your body to fight off bacteria and viruses. You’ll be closely monitored in the hospital and afterward for any signs of infection.
Chemotherapy drugs can also affect healthy cells in other parts of the body, not just immune cells. Side effects may include nausea, vomiting, and a higher risk of infertility, which means having trouble getting pregnant or getting someone else pregnant in the future.
You’ll likely receive medication to help manage nausea and vomiting during your hospital stay. If you may want to have children in the future, your doctor may recommend fertility preservation, such as freezing sperm, eggs, or embryos, before chemotherapy.
AHSCT may not be right for everyone. Your doctor will consider your overall health before recommending it. People who are older or who have certain health conditions, such as heart or lung disease, may have a higher risk of complications. Your doctor will help you weigh the risks and benefits of AHSCT.
At this time, AHSCT is not FDA approved specifically for treating MS. The FDA says regenerative medicine therapies, including many stem cell-based therapies, have not been approved to treat any neurological disorder, including MS.
The FDA also warns people to be careful about unapproved stem cell treatments sold outside of regulated clinical trials. These treatments may be marketed with misleading claims, and they haven’t been proven safe or effective for MS. In some cases, they may cause serious side effects.
Many U.S. insurance companies only cover treatments that are FDA-approved specifically to treat MS. Experimental treatments — which AHSCT is considered for MS — usually aren’t covered.
Unless you’re part of an official clinical trial, you’ll likely have to pay out of pocket for an AHSCT. The NMSS notes that the average cost of an AHSCT is around $150,000.
Some people choose to travel outside the U.S. to get an AHSCT for their MS. Before seeking healthcare in another country, it’s important to research the treatment center, the medical team, and the country’s safety rules for stem cell treatments.
Stem cell therapy clinics are available in many countries. Some may advertise lower costs, but unapproved or poorly regulated treatments can come with serious health risks. Treatments at these clinics may not be safe, especially if they aren’t regulated or monitored by health authorities in that country.
Before considering stem cell treatment, be sure to talk to your neurologist. They can help you weigh the possible benefits and risks of AHSCT, discuss whether a clinical trial may be an option, and review other MS treatments that may be available to you.
On MyMSTeam, people share their experiences with multiple sclerosis, get advice, and find support from others who understand.
Have you received or considered a stem cell transplant to treat your MS? Let others know in the comments below.
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i had hsct 7 years ago off study at northwestern in chicago. i was 32/33 and in a nursing home and it was a final option. my edss went from 6.5 to 3.5. got rid of my wheelchair, walker, quad cane… read more
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