multiple sclerosis (MS) is a chronic inflammatory disease in which the body’s own immune system attacks and destroys the protective sheath that coats nerve cells, called myelin. This disrupts normal communication between the brain and the rest of the body.
While there is currently no cure for MS, there are many disease-modifying therapies, lifestyle changes, and experimental treatments designed to adequately control the disease.Over the past few decades, one such experimental treatment has been called hematopoietic stem cell transplantation (HSCT) has been at the forefront of advancing MS research.
This article reviews this novel MS therapy, its advantages and disadvantages, and the need for further research.
What is HSCT treatment?
Originally studied as a treatment option for certain types of blood cancers, after decades of research, HSCT provides strong evidence for use in people with MS.
The goal of HSCT is to potentially “reboot” or reset the body’s immune system. Since MS is an autoimmune disease, the body’s immune system is attacked by itself. HSCT aims to eliminate miscommunication between the brain and immune system, reducing or stopping it altogether demyelination occur.
How is autologous HSCT different from other types of HSCT?
Autologous HSCT (aHSCT) refers to stem cells derived from a person’s own body, as opposed to allogeneic HSCT, in which the stem cells are derived from another person.
How it can help MS symptoms
By essentially eliminating the body’s immune system and reintroducing stem cells, HSCT is designed to significantly reduce or completely stop the demyelination process associated with MS. This can stop the disease from progressing, but it can also improve sometimes debilitating neurological symptoms.
Common MS symptoms include, but are not limited to:
- cognitive dysfunction
- muscle spasms or tremors
- Numbness, tingling, or burning sensation
- balance problem
- Urinary and bowel dysfunction
In some disease states, multiple sclerosis can progress so rapidly that severe disability can result.
In a 2016 study, Nearly 70% of those who underwent HSCT showed no new MS disease activity after three years. The study also found significant neurological recovery in HSCT recipients throughout the trial and follow-up. This further suggests that HSCT may not only prevent new disease activity, but may also help repair pre-existing nerve damage.
HSCT and DMT treatment
While HSCT is considered a therapeutic option with the potential to at least temporarily halt disease activity and progression, more research is needed. Treatment also needs to be made more accessible to patients.
While research is underway around the world, a variety of disease-modifying therapies (called DMTs) are used to help manage MS symptoms and slow the progression of the disease.
The National Multiple Sclerosis Society has reviewed all relevant data and treatments and strongly recommends starting DMT immediately after diagnosis. Discovered that continuous use of DMT can:
- Reduce the number of new recurrences
- Disability progresses slowly
- Reduces new inflammation within the central nervous system
DMT comes in one of three ways, oral, injectable, or intravenous medication.
Conventional DMT, most common interferon beta (Avonex and Betalone) and glatiramer acetate (Copason), helps relieve some MS symptoms through a variety of mechanisms, including by inhibiting immune cell activation and reducing the immune system’s inflammatory response.
pros and cons
As with any new therapy, both the affected individuals and the treating healthcare provider need to carefully weigh the risks and benefits.
Potential to completely stop disease progression over a long period of time
A person’s own stem cells can be used, no external donor required
Can reduce or even reverse some neurological impairments
Not yet approved by the U.S. Food and Drug Administration (FDA) for off-trial MS treatment
increased risk of infection
Linked to rare death
According to the National Multiple Sclerosis Society, aHSCT may be a beneficial treatment option for people who:
- have relapsing-remitting MS (stable periods between symptomatic periods)
- Have MS for less than 10 years
- under 50 years old
- have new lesions Relapse on MRI or despite appropriate disease-modifying therapy
- Inability to take effective disease-modifying therapy
HSCT has been found to be most effective in highly active patients with relapsing MS.
How HSCT Medicine Works
This treatment is a multi-step process, usually done only once. While procedures may vary from center to center, the process is relatively as follows:
- People with MS are treated with drugs that help release stem cells from the bone marrow into the blood.
- The stem cells are then collected and frozen until the patient is ready for infusion.
- While the stem cells are in a waiting state, the patient must receive medication, such as chemotherapy or immunosuppressants, to try to suppress the body’s immune system.
- Once the patient’s immune system has been primed, the stem cells can be introduced into the body through an intravenous infusion.
- Then, as they recover, they are monitored very closely.
Potential Risks and Complications
Although HSCT is very promising as a treatment option for early-stage MS, it has potential risks and complications. These can include:
- increased risk of infection
- allergic reaction
- low neutrophils counting
Notably, many of the adverse effects found in HSCT studies of MS patients were associated with chemotherapy prior to transplantation. Research is ongoing to see if potential immunosuppressive approaches are just as effective, with fewer associated risks and adverse effects.
MS is a complex disease and its management requires collaboration between MS treating specialists and affected individuals. Working together to develop an appropriate treatment plan ensures that people with MS have a say in their care.
When conventional treatments fail to adequately control the disease, some new options may be worth discussing.
Clinical trials near you
There are currently a number of HSCT clinical trials for MS in the world, and participants are being actively recruited. These studies can be found at ClinicalTrials.gov. To see if you are eligible for one of the studies, please contact your MS specialist to discuss.
What HSCT means for the future of MS research
Researchers around the world are working to find new treatments for potentially disabling diseases such as MS. Through the studies conducted to date, HSCT has demonstrated that it deserves further study.
HSCT has the potential to halt MS disease activity, but more is needed to understand how long such outcomes can last, what other factors can be modified to reduce side effects for patients, and more.
MS is a complex disease, but thankfully there are many successful treatment options available. HSCT may be an option to consider in people whose disease and its symptoms are not effectively controlled by conventional disease-modifying therapies. Research to date has found that HSCT has halted MS disease activity and, in some cases, even reversed previous damage. Although not FDA-approved for widespread use in MS patients, clinical trials are ongoing.
If you or someone you know has been diagnosed with MS within the past 10 years and is struggling to manage the disease, HSCT may be an option to consider. You must tell your healthcare provider about all treatment options you are interested in. Contact your provider to find out if you are eligible for a clinical trial using HSCT for aggressive and relapsing MS.
Frequently Asked Questions
Is HSCT approved for MS FDA?
Drugs and procedures for HSCT are approved by the FDA, and HSCT is approved to treat certain types of blood or bone marrow cancer. However, HSCT is currently not approved for the treatment of MS and is considered experimental. Therefore, ongoing trials need to provide more evidence for potential future approval.
Do all MS types respond to HSCT?
Available evidence suggests that HSCT may be most beneficial for people with aggressive and highly active MS or relapsing forms of MS.
What is the cost of HSCT for MS?
Since HSCT does require hospitalization, the average cost is about $150,000. Costs vary depending on the provider and what insurance the patient may have.
Is HSCT the same as chemotherapy?
No, HSCT is not the same as chemotherapy. Before HSCT, however, a person must be treated with chemotherapeutic agents to prime the immune system for stem cell transplantation.
What is the success rate of MS HSCT?
The current success rates of HSCT in MS vary from study to study, further emphasizing the need for further research. That being said, studies to date have had high success rates in stopping new disease progression in MS patients.