Experimental Therapies for Multiple Sclerosis (MS)

Multiple sclerosis (MS) is an autoimmune disease that affects the central nervous system. MS eats away at the myelin sheath that protects nerve fibers. MS-related damage to myelin disrupts communication between the brain and the rest of the body. There isn't a cure for MS, but promising new treatments are constantly being developed.

This article reviews the latest experimental treatments being developed for MS as well as recently approved therapies.

Doctor discusses treatment with woman with multiple sclerosis

Deepak Sethi / Getty Images

Experimental Therapies for MS

There are a number of experimental therapies being explored for MS treatment.

Masitinib

Masitinib is a tyrosine kinase inhibitor that targets overly active immune cells known to play a role in progressive MS.

A 2022 study found evidence that people with primary progressive multiple sclerosis and inactive secondary progressive MS benefited from taking masitinib orally.

Simvastatin

Simvastatin is a prescription drug typically used to lower low density lipoprotein (LDL) cholesterol, considered "bad" cholesterol. Although some research suggests that this drug improves MS symptoms by lowering cholesterol, a 2019 study found that simvastatin may slow MS progression via mechanisms not linked to cholesterol reduction.

Ibudilast

MN-166 (ibudilast) is an experimental anti-inflammatory MS treatment that has been shown to slow MS-related brain atrophy (wasting). It was given "Fast Track" designation by the Food and Drug Administration (FDA) in 2016.

Researchers are still testing the safety and efficacy of oral ibudilast in phase 3 clinical trials.

Lipoic Acid

Lipoic acid is an oral antioxidant therapy that may help to maintain brain volume by slowing the rate of whole-brain atrophy (wasting) in secondary progressive MS.

Stem Cell Therapies

Hematopoietic stem cell transplantation (HSCT) is an experimental treatment that may help some people with relapsing-remitting multiple sclerosis (RRMS).

Stem cell therapies are not FDA-approved and can be risky. Unfortunately, the potential benefits of this treatment may be overhyped by offshore locations that fail to disclose the risk of mortality associated with HSCT.

Tcelna

Tcelna is a T-cell immunotherapy. Hypothetically, this medication is tailored to each person's myelin condition and reduces the destructive activity of myelin-reactive T-cells (MRTC).

However, numerous studies and clinical trials into the effectiveness of immunomodulatory treatment strategies for MS, such as tcelna, have failed or ended with inconclusive results.

Temelimab

Temelimab is a monoclonal antibody therapy that targets a human retrovirus (HERV-W-Env) that may be associated with the development of certain autoimmune disorders such as MS.

Phase 2 trials suggest that temelimab has anti-neurodegenerative effects (can prevent degeneration of nerves in the brain) and may help people with progressive MS.

Rituximab

Rituximab is a monoclonal antibody that targets CD20 protein molecules found on the surface of B cells.

Rituximab is FDA-approved to treat cancers such as leukemia and non-Hodgkin’s lymphoma. It isn't approved to treat MS, but accumulating clinical evidence suggests that rituximab is an effective disease-modifying therapy (DMT) for relapsing and progressive MS.

Minocycline

Minocycline is an antibiotic that's been used to treat bacterial infections since the 1970s. Recently, researchers have discovered that it also has anti-inflammatory and neuroprotective properties, characteristics that help protect nerve cells from damage.

A 2017 clinical trial found that minocycline lowered the risk of conversion to multiple sclerosis after someone has their first demyelinating event, also known as clinically isolated syndrome.

NDC-1308

NDC-1308 is an experimental drug that repairs the myelin sheath of demyelinated nerve fibers and may slow the progression of MS. Animal models suggest that NDC-1308 increases remyelinating activity but human studies are needed to see if this small molecule can help people with MS.

Other Treatments

Although the underlying cause of MS is unknown, emerging evidence suggests that the Epstein-Barr virus (EBV) may have some influence. A 2022 analysis found a high prevalence of people having been infected with EBV also developing multiple sclerosis.

Another study found a mechanistic link for the association between MS and Epstein-Barr. This research could lead to the development of new MS treatments that target EBV-infected cells.

There is also research to suggest that adequate intake of vitamin D can reduce the risk of MS development and improve disease activity. However, more research is needed to formally recommend vitamin D supplementation as a treatment for MS.

How Long Does It Take to Get Experimental MS Treatments Approved?

Getting an experimental therapy approved for use in patients is a lengthy process that typically involves three phases of clinical trials, taking on average between 10 to 15 years.

Recently Approved DMTs

Eight disease-modifying therapies have been approved since 2017. Below is a timeline of the DMTs approved between March 2017 and March 2021, in reverse chronological order:

  • Ponvory (ponesimod) Approved: March 18, 2021
  • Kesimpta (ofatumumab) Approved: August 20, 2020
  • Bafiertam (monomethyl fumarate) Approved: April 28, 2020
  • Zeposia (ozanimod) Approved: March 25, 2020
  • Vumerity (diroximel fumarate) Approved: October 29, 2019
  • Mavenclad (cladribine) Approved: March 29, 2019
  • Mayzent (siponimod) Approved: March 26, 2019
  • Ocrevus (ocrelizumab) Approved: March 28, 2017

Treating Multiple Sclerosis

DMTs are used to slow the progression of MS. DMTs for treating multiple sclerosis fall into these three categories:

  • Oral therapies: Mayzent and Zeposia are popular oral treatments, among others.
  • Injectables: Copaxone (glatiramer acetate) and Rebif (interferon beta-1a) are commonly used injectables.
  • Intravenous (IV) infusions: Ocrevus (ocrelizumab) is one of the newer FDA-approved infusion therapies.

How Close Is a Cure?

Finding a cure for MS has been a decades-long medical quest, with each year showing more promise of coming closer to a cure.

Studies published at the beginning of 2022 suggest that designing antiviral drugs capable of blocking the Epstein-Barr virus could lead to treatments, procedures, or a vaccine against EBV that may help prevent MS development.

Summary

Multiple sclerosis is a mysterious disease. It's still unclear exactly what causes MS, and there is still no cure. Fortunately, there are over a dozen FDA-approved drugs that can slow or halt disease progression. Experimental treatment options—mainly medications—are continuously being developed.

The link between MS and Epstein-Barr virus shows promise for breakthrough treatments that might even lead to a cure.

A Word From Verywell

Experimental therapies give us hope that someday there may be a way to prevent or cure MS. Existing treatments can slow or halt disease progression and improve your quality of life, but emerging treatments show promise for reversing MS-related degeneration. That said, it's always wise to proceed with caution and to speak with a healthcare provider before trying any therapy that isn't FDA-approved.

Frequently Asked Questions

  • What is the newest treatment for MS?

    Fifteen drugs have been approved by the Food and Drug Administration for MS treatment since 2004, including Ponvory (ponesimod), approved on March 18, 2021.

  • Are we getting close to a cure for MS?

    Yes, we may be getting closer to a cure for MS. After discovering a causal link between Epstein-Barr virus and MS in January 2022, some scientists speculate that antiviral drugs designed to target EBV-infected cells could lead to a cure for MS.

  • What is the importance of experimental treatment?

    Experimental therapies for MS bring a cure within closer reach. Although existing FDA-approved drugs can help slow or halt MS's progression, emerging treatments move the ball forward. Groundbreaking discoveries give people with MS hope that something new might reverse the disease and heal their bodies. 

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