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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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
Find Clinical Trials Near Me
Interested in clinical trials? Simply search by postal code and type of condition to see what’s going on in your area.
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