The Pros and Cons of HSCT for MS

Multiple sclerosis (MS) is a chronic inflammatory disease in which the body's own immune system attacks and destroys the protective sheath covering nerve cells, known as myelin. This disrupts proper communication between the brain and other areas of the body.

While there is currently no cure for MS, there are numerous disease-modifying therapies, lifestyle modifications, and experimental treatments aimed at adequately managing the disease. Over the last few decades one such experimental treatment known as hematopoietic stem cell transplantation (HSCT) has been at the forefront of progressive MS research.

This article reviews this novel MS therapy, its pros and cons, and what further research needs to be done.

Neuron affected by MS

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What Is HSCT Treatment?

Originally studied as a treatment option for certain types of blood cancers, through decades of research, HSCT has provided 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 under attack from itself. HSCT aims to knock out the miscommunication between the brain and the immune system and decrease or completely halt the demyelination that occurs.

How Is Autologous HSCT Different From Other Types of HSCT?

Autologous HSCT (aHSCT) refers to stem cells that are derived from a person's own body, as opposed to allogeneic HSCT, in which the stem cells come from another individual.

How It Could Help MS Symptoms

By essentially wiping out the body's immune system and reintroducing stem cells, HSCT aims to significantly decrease or completely stop the demyelination process associated with MS. This can halt disease progression, but also improve sometimes debilitating neurological symptoms.

Common MS symptoms include but are not limited to:

  • Fatigue
  • Cognitive dysfunction
  • Muscle spasms or tremors
  • Numbness, tingling, or burning sensations
  • Balance issues
  • Urinary and bowel dysfunction

In some disease states, MS can be so progressive that it can lead to significant disability.

In a 2016 study, nearly 70% of people who received HSCT showed no new MS disease activity after three years. The study also found throughout the trial and follow-up that recipients of HSCT had a significant recovery in neurological function. This further illustrates that HSCT may not only stop new disease activity, but potentially help repair pre-existing neurological damage.

HSCT vs. DMT Treatment

While HSCT is regarded as a treatment option with the potential to at least temporarily suspend disease activity and progression, more research is needed. The treatment also needs to be more accessible to patients.

While research is underway all around the world, multiple disease-modifying treatments, known as DMTs, are used to help manage MS symptoms and also slow the progression of disease.

The National Multiple Sclerosis Society has reviewed all relevant data and treatments and strongly recommends the initiation of DMTs soon after diagnosis. Ongoing use of DMTs has been found to:

  • Reduce the number of new relapses
  • Slow progression of disability
  • Decrease new inflammation within the central nervous system

DMTs come in one of three ways, either as oral, injectable, or intravenous medications.

Conventional DMTs, most commonly interferon beta (Avonex and Betaseron) and glatiramer acetate (Copaxone), help alleviate some MS symptoms through a variety of mechanisms, including by inhibiting immune cell activation and decreasing the inflammatory response of the immune system.

Pros and Cons

As with any novel therapy, the risks and benefits need to be weighted carefully by both the affected individual and the treating healthcare providers.

Pros
  • Has the potential to fully halt disease progression for a prolonged period

  • A person's own stem cells can be used and no outside donor is required

  • Can decrease and even reverse some neurologic disability

Cons
  • Not yet approved by the Food and Drug Administration (FDA) for MS treatment outside of trials

  • Expensive

  • Increased risk of infection

  • Associated with rare cases of death

HSCT Eligibility

According to the National Multiple Sclerosis Society, aHSCT may be a beneficial treatment option for people who:

  • Have relapsing-remitting MS (periods of stability between periods of symptoms)
  • Have had MS for less than 10 years
  • Are younger than 50 years old
  • Have had new lesions on MRI or relapse in condition despite appropriate disease-modifying therapies
  • Are unable to take high-efficacy disease-modifying therapies

HSCT has been found to be most effective in people with highly active relapsing MS.

How HSCT Medicine Works

This treatment is a multi-step procedure that is typically performed only once. While the procedure may vary slightly from center to center, the process remains relatively as follows:

  1. Patients with MS are treated with medications that will help release stem cells from the bone marrow into the bloodstream.
  2. The stem cells are then collected and frozen until the patient is ready to be infused.
  3. While the stem cells are in waiting, the patient must undergo medication treatments such as chemotherapy or immunosuppressants in an attempt to suppress the body's immune system.
  4. Once the patient's immune system has been primed, the stem cells can be introduced back into the body through an intravenous infusion.
  5. The individual is then monitored very closely as they recover.

Potential Risks and Complications

While HSCT is quite promising as a treatment option for early MS, it comes with potential risk and complications. These can include:

  • Increased risk of infection
  • Allergic reaction
  • Fevers
  • Rash
  • Low neutrophil count
  • Death

Of note, many of the adverse reactions seen throughout studies of HSCT in people with MS were associated with the chemotherapy administered prior to the transplantation. There is currently research being done to see if potentially less immunosuppressive methods can be just as effective, with less associated risk and adverse reactions.

Getting Involved

MS is a complex disease whose management requires collaboration between the treating MS specialists and the individual affected. Collaborating on an appropriate treatment plan ensures that a person with MS has a say in their own care.

When conventional therapies are failing to adequately manage the disease, some new options may be worth discussing.

Clinical Trials Near You

There are currently several clinical trials on HSCT for MS around the world, which are actively recruiting participants. These studies can be found on ClinicalTrials.gov. To see if you may be eligible for inclusion in one of these studies, contact your MS specialist to discuss.

What HSCT Means for the Future of MS Research

Researchers around the world are working to find new therapies for treating potentially disabling diseases such as MS. Through studies performed to date, HSCT has proven that it warrants further research.

HSCT has the potential to stop MS disease activity in its tracks, but more needs to be learned about how long such results could last, what other factors can be modified for fewer side effects to the patient, and more.

Summary

MS is a complex disease, but thankfully there are a number of successful treatment options available. In people for whom conventional disease-modifying therapies are not effectively managing the disease and its symptoms, HSCT may be an option to consider. Studies to date have found that HSCT has halted MS disease activity and, in some cases, even reversed previous damage. Though not FDA-approved for widespread use in MS patients, clinical trials are ongoing.

A Word From Verywell

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. It's essential to inform your healthcare provider about all the treatment options you're interested in. Contact your provider to see if you may be eligible for enrollment in a clinical trial using HSCT to treat aggressive and relapsing MS.

Frequently Asked Questions

  • Is HSCT for MS FDA approved?

    The medications and procedure used for HSCT are FDA-approved and HSCT is approved for the treatment of certain types of blood or bone marrow cancers. However, HSCT is not currently approved for the treatment of MS and is considered experimental. Thus, ongoing trials need to provide more evidence for potential future approval.

  • Do all MS types respond to HSCT?

    Available evidence shows that HSCT may be most beneficial for people with aggressive and highly active MS or relapsing forms of MS.

  • How much does HSCT for MS cost?

    Because HSCT does require hospitalization, the average cost is around $150,000. Cost varies depending on the performing institution as well as insurance the patient may have.

  • Is HSCT the same thing as chemotherapy?

    No. HSCT is not the same thing as chemotherapy. However, before HSCT can occur, a person must be treated with chemotherapeutic agents to prime the immune system for the stem cell transplant.

  • What is the success rate of HSCT for MS?

    Current success rates of HSCT in MS vary from study to study, further emphasizing the need for additional research. That being said, studies to date have all yielded a high rate of success in halting new disease progression in people with MS.

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5 Sources
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read our editorial process to learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
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