Multiple sclerosis (MS) is a chronic autoimmune disease that affects the central nervous system. It occurs when the immune system attacks the protective covering of nerve fibers (myelin), causing inflammation and disrupting nerve signaling. Common symptoms include fatigue, muscle weakness, vision problems and coordination difficulties. The exact cause of MS is unknown, but genetics and environmental factors are believed to play a role.
What are the treatments for multiple sclerosis?
MS treatment focuses on managing symptoms, slowing disease progression, and reducing relapses. Traditional options include disease-modifying therapies (DMTs) such as interferon-beta, glatiramer acetate, and monoclonal antibodies such as natalizumab and ocrelizumab. Symptom-specific therapies, physical therapy, and lifestyle changes also play important roles in treatment. In recent years, hematopoietic stem cell transplantation (HSCT) has emerged as a promising approach.
What does hematopoietic stem cell mean?
Stem cells have the ability to develop into many different types of cells. Hematopoietic stem cells, in particular, produce blood cells, including immune cells.
HSCT removes the harmful immune cells that cause the disease and replaces them with healthy stem cells to rebuild the immune system. This is a promising treatment option, especially for diseases such as MS, where the immune system does not function properly.
What is autologous hematopoietic stem cell transplantation (aHSCT) in MS?
Hematopoietic stem cell transplantation (HSCT) aims to “reset” the immune system by replacing faulty immune cells with healthy ones. While still considered experimental, ongoing research highlights the potential to significantly improve outcomes for some people with MS, especially those with aggressive or relapsing forms of the disease.
There are multiple types of HSCT that destroy the immune system in different amounts to “reset.” Myeloablative HSCT completely destroys the immune system. Another, called “non-myeloablative,” partially destroys the system.
When talking about stem cell treatments for MS, what is usually meant is a procedure called autologous hematopoietic stem cell transplantation (aHSCT). It is the most studied stem cell treatment for MS. In aHSCT, blood stem cells taken from the bone marrow replace cells in the immune system that attack the nervous system. In this way, the stem cell transplant “reboots” the immune system to slow down MS activity.
Who is a suitable candidate for HSCT?
The suitability of a patient is determined by detailed tests by specialists. However, if you have very active relapsing MS and are still experiencing relapses despite taking DMTs, you may benefit from this procedure. This is because HSCT targets the immune system, which causes inflammation.
How is HSCT treatment applied in MS?
HSCT is a type of bone marrow transplant designed to reset the immune system and stop the inflammation that contributes to relapsing MS. There is no standard HSCT protocol for MS. Protocols vary from center to center. The immunosuppressive drugs used in chemotherapy during the procedure also vary. However, the steps of HSCT are generally as follows:
Steps 1-3 involve stimulating the production and release of stem cells into the blood. The process of collecting and freezing the stem cells takes 5 to 15 days. Treatment usually includes chemotherapy to help prevent relapses. The transplant (steps 4-6) usually requires a 3-week stay in the hospital.
What happens after MS stem cell therapy?
You will have frequent check-ups for the next 2 years after HSCT, and the following assessments and procedures will usually be performed:
- Physical, neurological and cognitive assessments
- MRI and blood tests
- Psychological support
You will need to have an MRI within 6 months after HSCT. After that, you will be asked to have an MRI at least once a year.
Why should you be careful about stem cell treatment for MS?
The results of stem cell therapy vary from person to person. You may have heard many different stories. You should visit a specialized and trusted health center to decide if the procedure is right for you and what risks and benefits you can expect.
Some centers do not have the experience to perform HSCT on MS patients. Some exaggerate their patient experiences to attract patients. Make sure centers can back up their claims with peer-reviewed research.
What are the latest research on stem cell treatment of MS?
The Role of Glia in MS
A study was published in Cell Stem in August that will contribute to the development of stem cell therapy. So far, treatments have generally focused on suppressing the immune system. However, a study conducted by a team of scientists from the New York Stem Cell Foundation (NYSCF) Research Institute and Case Western Reserve University revealed that support cells called “glia” located in the brain also play an important role in the progression of MS.
This study was conducted on cells taken from MS patients and these cells were regenerated in the laboratory with stem cell technology. The researchers realized that these glial cells contribute to the disease on their own, independent of the immune system.
In particular, it was seen that the cells that produce the “myelin” sheath that protects the nerves were deficient. These findings show that new methods can be developed in the treatment of MS that target not only the immune system but also glial cells. This could help stop the progression of the disease and provide MS patients with a better life.
Stem Cell Therapy Success in Relapsing MS
A randomized controlled trial looked at 110 people with ‘very active’ relapsing MS. Half were treated with non-myeloablative HSCT and the other half with other DMTs. The study found:
- 99% of people treated with HSCT were relapse-free at 1 year. Only 1 person had a relapse. There were 39 relapses in people treated with medication.
- 94% of people treated with HSCT saw their disability not get worse over 3 years. This was the case for 40% of those treated with medication.
- EDSS scores (a measure of disability) improved in people treated with HSCT. Average EDSS scores worsened in people on medication.
What is the Expanded Disability Status Scale (EDSS)?
The Expanded Disability Status Scale (EDSS) is a tool used by healthcare professionals to measure and monitor the level of disability in individuals with neurological disorders. It assesses a person’s ability to perform daily activities, focusing on walking ability and other functions such as vision, coordination, and speech. The scale ranges from 0 (normal function) to 10 (death due to disease) and provides a clear way to understand changes in disability over time.
The EDSS is widely used to assess the progression of disability in people with multiple sclerosis (MS). It helps monitor the impact of the disease on mobility and neurological function, guides treatment decisions, and monitors the effectiveness of treatments. Regular EDSS assessments are important to understanding how MS develops and how it affects a person’s quality of life.
Frequently Asked Questions About MS Stem Cell Treatment
What are the results of stem cell therapy for relapsing MS?
Clinical studies have shown that HSCT can reduce relapses. Some people experience stabilization of symptoms and/or improvement in their disability. However, these improvements are not always permanent.
What are the side effects of HSCT?
HSCT has the highest risk of serious side effects compared with disease-modifying therapies (DMTs). Side effects that may occur include:
- Increased risk of developing infections in the long term
- Increased risk of developing cancer and autoimmune conditions such as thyroiditis
- Early menopause
- Infertility problems
The chemotherapy that is part of the HSCT procedure also has its own side effects. These include an increased risk of bleeding and bruising, fatigue, loss of appetite and hair loss.
If someone has a high level of disability before the transplant, chemotherapy can do more harm than good. Chemotherapy treatments can cause your mobility to deteriorate and the way your nerves work.
If you have a myeloablative type of HSCT, where the immune system is completely destroyed, the side effects of chemotherapy are more likely to be serious.
Some people have died as a result of HSCT. Since 2005, 1 in 330 people who have had HSCT have died from it. This risk is: It may increase in people who are elderly, have high EDSS scores or have certain other conditions.
How should pregnancy, breastfeeding and birth control be during HSCT?
Your doctor will discuss all the disadvantages with you in detail and indicate what precautions should be taken when undergoing HSCT.
The chemotherapy drugs you receive with HSCT can damage the unborn baby, ovaries and testicles. This damage is usually permanent and prevents you from having children (infertility). It can also cause early menopause in women. Hormone replacement therapy (HRT) can help with menopause symptoms but cannot stop infertility.
You can freeze your eggs or sperm before having HSCT. You can then have children using procedures such as IVF (in vitro fertilization).
High levels of chemotherapy drugs can pass into breast milk. Therefore, breastfeeding should be avoided during HSCT.
If you have periods, they may stop during chemotherapy, but you may still be producing eggs, so you can get pregnant.
You should continue to use reliable birth control, as chemotherapy drugs can harm an unborn baby. It takes about 6 to 12 months for periods to return to normal. It is important to use birth control during this time.
Since chemotherapy damages sperm, male patients should ask their doctor about what type of birth control they should use.
Sources:
- nationalmssociety.org
- mssociety.org.uk
- The New York Stem Cell Foundation – nyscf.org
- Patient iPSC models reveal glia-intrinsic phenotypes in multiple sclerosis, Clayton, Benjamin L.L. et al., Cell Stem Cell, Volume 31, Issue 11, 1701 – 1713.e8