Clinical Trials for Multiple Sclerosis (MS)

Medically reviewed by
Evelyn O. Berman, MD
, Assistant Professor of Neurology and Pediatrics at University of Rochester, Rochester, NY  | Written by 
Emily Wagner

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Editor's note: This article contains information that goes out-of-date quickly. While we strive to maintain currency, research trends can sometimes move faster than our editorial team. We still feel this information is important for those considering participation in clinical trials. Please consult with your doctor or the investigative team running any particular research study for the most up-to-date information.
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Before a new treatment can be prescribed to people living with multiple sclerosis (MS), it must first go through the clinical trials process. Clinical trials are studies that last months to years that use healthy volunteers or people with a certain condition to see whether a new drug is safe and effective.

Current trials investigating MS treatments are focused on both new and existing treatments used in other autoimmune diseases. These include small molecule inhibitors, monoclonal antibodies, and stem cell transplants.

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What is Multiple Sclerosis?Treatments for Multiple Sclerosis

Current Clinical Trials for Multiple Sclerosis

MS clinical trials are conducted by researchers and doctors to find better ways to treat the disease. These trials may be studying new treatments or they may be repurposing existing therapies that work well in other autoimmune or neurological diseases. The drugs and therapies discussed below are just a fraction of the current clinical trials for MS, representing a variety of treatment strategies.

Bruton’s Tyrosine Kinase (BTK) Inhibitors

Bruton’s tyrosine kinase (BTK) is a protein known to be critical for the function of B cells. These are specialized immune cells that are responsible for making antibodies, which help the immune system fight off infection. BTK inhibitors are drugs designed to block the protein, preventing B cells from functioning properly. These drugs are mainly used to treat certain blood cancers, such as leukemias and lymphomas.1

However, recent research has found that BTK may also play a role in MS. There are several BTK inhibitors being studied as new disease-modifying therapies (DMTs), which are used to slow nerve damage and disease progression in MS. BTK inhibitors are more selective to the types of B cells they block, meaning they’ll likely have fewer side effects compared to other DMTs such as ocrelizumab (Ocrevus®).

Newer BTK inhibitors being studied for treating MS also have another advantage compared to older DMTs — they can cross the blood-brain barrier (BBB). This means that these drugs can be more effective in the central nervous system (CNS) and act in the areas they need to. MS causes lesions (scarring) in the brain and spinal cord. BTK inhibitors may be able to stop B cells from attacking the fatty coating on nerve cells (myelin), which causes MS symptoms.2,3

There are currently four BTK inhibitors in clinical trials for treating MS:

  • Evobrutinib — two clinical trials are currently recruiting participants with relapsing-remitting MS (RRMS) for phase 3 studies comparing the drug to teriflunomide (Aubagio®)4,5
  • Fenebrutinib — three clinical trials are currently recruiting participants with primary progressive MS (PPMS) or RRMS for phase 2 and 3 studies comparing the drug to placebo, Aubagio®, or ocrelizumab (Ocrevus®)6-8
  • Orelabrutinib — one clinical trial is currently recruiting participants with RRMS for a phase 2 study comparing the drug to placebo9
  • Tolebrutinib — two clinical trials are currently recruiting participants with PPMS or secondary progressive MS (SPMS) for phase 3 studies comparing the drug to placebo10,11

Other New Therapies for Multiple Sclerosis

Researchers are also investigating other new treatments, including a monoclonal antibody (mAb) drug and lipoic acid.

Ublituximab is a mAb currently being studied for treating RRMS, in addition to blood cancers. A recent study published in August 2022 from the phase 3 clinical trials for ublituximab showed that the therapy reduced relapse rates and the number of brain lesions seen with magnetic resonance imaging (MRI) when compared to placebo or Aubagio®.12 Currently, an extension study for ublituximab is enrolling participants by invitation for those who have completed another active (not recruiting) study of the drug.13,14

Lipoic acid is an antioxidant also being studied for treating MS. Studies show that lipoic acid helps prevent immune cells from crossing the BBB to create inflammation in the CNS. There’s currently one active (not recruiting) phase 2 clinical trial investigating lipoic acid against a placebo in treating progressive forms of MS.15,16

Clinically Available Therapies for Multiple Sclerosis

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Often, therapies already approved by the U.S. Food and Drug Administration (FDA) for one disease are studied for use in treating another disease. For example, many MS clinical trials are currently looking at treatments used in diabetes.

Metformin is a medication originally approved to treat type 2 diabetes (T2D) to help control blood sugar levels. Now, it’s being investigated as an MS treatment due to its antioxidant and anti-inflammatory effects. There’s currently one phase 1 clinical trial recruiting participants with progressive forms of MS to compare metformin to placebo.17,18

Stem cell therapy uses cells from a donor or a person’s own bone marrow to help treat certain diseases. Specifically, mesenchymal stem cells (MSCs) are being studied for treating MS and other neurological conditions. MSCs have the potential to become cells used to build bone, cartilage, or fat. There’s currently one phase 2 clinical trial recruiting participants with RRMS to compare treatment with their own MSCs to placebo.19,20

Enrolling in MS Clinical Trials

Before you can join an MS clinical trial, there are a few things to take into consideration. First, you must decide whether you want to take part in a clinical trial. Many people join these studies when they’ve run out of treatment options, while others join to help advance science and new therapies. No matter your reasons for joining, it’s important to consider what the best choice is for you.21

Eligibility for MS Clinical Trials

Once you’ve decided you’d like to participate in a clinical trial, you can talk to your doctor about trials you’re eligible for. Study investigators select inclusion and exclusion criteria, which determine who can and can’t participate in a trial.

Inclusion criteria are a list of requirements that must be met in order to participate in a trial. In MS trials, the criteria differ slightly between studies. Some examples include:8

  • Adult male and female subjects ages 18 to 65 years old
  • Diagnosis of PPMS according to the revised 2017 McDonald criteria
  • Disability progression in the 12 months before screening

Exclusion criteria are a list of conditions or treatments that disqualify you from participating in a study. Examples include:8

  • Having an infection at screening, such as tuberculosis or hepatitis B or C
  • History of cancer
  • Having another condition that is treated with immunosuppressants or corticosteroids that can interfere with the study results

Though exclusion criteria limit clinical trial participation, these requirements are in place to help keep people safe who may otherwise experience unwanted side effects or become sick from treatment.21

Last updated: 11/06/2023Last medically reviewed: 04/04/2023

Medical Disclaimer: The information provided in this article is not a substitute for the advice of qualified healthcare professionals. While we strive to publish accurate information, it is not possible to cover all potential scenarios, including drug or treatment effects, interactions, or usage. You should not rely solely on this article to determine whether a particular treatment, drug, or clinical trial is suitable for you or any other individual. Always consult a healthcare professional before starting or changing any treatments.

Sources

  1. Singh SP, Dammeijer F, Hendriks RW. Role of Bruton’s tyrosine kinase in B cells and malignancies. Mol Cancer. 2018;17(1):57. 
  2. MS Society. Under the Microscope: What is the Potential of BTK Inhibitors? January 4, 2022. Accessed from: https://www.mssociety.org.uk/research/latest-research/research-blog/under-microscope-what-potential-btk-inhibitors 
  3. Garcia-Merino A. Bruton’s tyrosine kinase inhibitors: A new generation of promising agents for multiple sclerosis therapy. Cells. 2021;10(10):2560.
  4. ClinicalTrials.gov. Study of Evobrutinib in Participants with RMS (evolutionRMS 1). October 6, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT04338022
  5. ClinicalTrials.gov. Study of Evobrutinib in Participants with RMS (evolutionRMS 2). October 3, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT04338061
  6. ClinicalTrials.gov. A Study to Investigate the Efficacy of Fenebrutinib in Relapsing Multiple Sclerosis (RMS) (FENopta). October 14, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT05119569
  7. ClinicalTrials.gov. Study To Evaluate The Efficacy Of Fenebrutinib Compared With Teriflunomide In Relapsing Multiple Sclerosis (RMS) (FENhance). October 14, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT04586023
  8. ClinicalTrials.gov. A Study To Evaluate The Efficacy And Safety Of Fenebrutinib Compared With Ocrelizumab In Adult Participants With Primary Progressive Multiple Sclerosis (FENtrepid). September 29, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT04544449
  9. ClinicalTrials.gov. A Phase 2 Study of Orelabrutinib in Patients With Relapsing-Remitting Multiple Sclerosis. May 18, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT04711148
  10. ClinicalTrials.gov. Nonrelapsing Secondary Progressive Multiple Sclerosis (NRSPMS) Study of Bruton’s Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (HERCULES). October 3, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT04411641
  11. ClinicalTrials.gov. Primary Progressive Multiple Sclerosis (PPMS) Study of Bruton’s Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (PERSEUS). October 3, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT04458051 
  12. Steinman L, Fox E, Hartung H, et al. Ublituximab versus teriflunomide in relapsing multiple sclerosis. N Engl J Med. 2022;387(8):704-714. 
  13. ClinicalTrials.gov. An Extension Study of Ublituximab in Participants With Relapsing Multiple Sclerosis. October 20, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT04130997 
  14. ClinicalTrials.gov. An Extension of the TG1101-RMS201 Trial. October 20, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT03381170 
  15. Xie H, Yang X, Cao Y, et al. Role of lipoic acid in multiple sclerosis. CNS Neurosci Ther. 2022;28(3):319-331. 
  16. ClinicalTrials.gov. Lipoic Acid for Progressive Multiple Sclerosis (MS) (LAPMS). March 14, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT03161028 
  17. Dziedzic A, Saluk-Bijak J, Miller E, Bijak M. Metformin as a potential agent in the treatment of multiple sclerosis. Int J Mol Sci. 2020;21(17):5957. 
  18. ClinicalTrials.gov. Metformin Treatment in Progressive Multiple Sclerosis. May 6, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT05349474 
  19. Gugliandolo A, Bramanti P, Mazzon E. Mesenchymal stem cells in multiple sclerosis: Recent evidence from pre-clinical to clinical studies. Int J Mol Sci. 2020;21(22):8662. 
  20. ClinicalTrials.gov. Randomized Double-Blind Phase 2 Efficacy and Safety of Autologous HB-MSCs vs Placebo for Treatment of Multiple Sclerosis (HBMS01). May 24, 2022. Accessed from: https://clinicaltrials.gov/ct2/show/NCT05116540 
  21. Jalusic KO, Ellenberger D, Rommer P, et al. Effect of applying inclusion and exclusion criteria of phase III clinical trials to multiple sclerosis patients in routine clinical care. Mult Scler. 2021;27(12):1852-1863.
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