The Latest on MS Clinical Trials

Clinical trials are the process we use to test new treatments. Most of the drugs and treatments we use exist because of clinical trials. These trials are an important step in the approval process for any new drug or treatment. They are used to make sure the new treatment is safe and effective. There are many clinical trials in progress for multiple sclerosis (MS).^1,2

MS clinical trials

MS is an autoimmune disease. This means it is caused by the immune system mistakenly attacking the body. With MS, the immune system attacks the myelin on nerves. Most new trials look at ways to reduce symptoms or disease progression. They do this by targeting the immune system.^1,3

Clinical trials for MS are typically divided by the type of MS they focus on:^1,3

• Relapsing-remitting MS (RRMS) – RRMS is when MS goes through cycles of stopping before advancing again. Trials for this form of MS tend to be more common and successful.
• Primary progressive MS (PPMS) – PPMS is when MS actively advances without relapse. Trials for this form of MS have not been as successful. But new methods show promise.

There are many different active clinical trials at any time for MS. The clinicaltrials.gov website lists clinical trials active in the United States. At any given time, the website can list hundreds of active MS trials. Here, we have provided details about a few trials that are in progress.^4,5

Biologics and biosimilars

Biologics are drugs made from living cells. One type of biologic is called a monoclonal antibody. Monoclonal antibodies are clones of antibodies that our bodies naturally produce. These clones are made in labs. They work by helping your body target cells that are involved in immune reactions.^3,6,7

In recent years, many monoclonal antibodies have been in trials for MS. These include:^3,6,7

• Ocrelizumab (Ocrevus®)
• Rituximab (Rituxan®)
• Ublituximab (Briumvi™)
• Ofatumumab (Kesimpta®)

Thanks to clinical trials, many monoclonal antibody drugs have been approved to treat MS, including Ocrevus, Briumvi, and Kesimpta.^3,6,7

Biosimilars are drugs that are very similar to a specific biologic. But because biologics are so complex, biosimilars are not identical to them. A biosimilar has to be as effective as its similar biologic. Recently, natalizumab-sztn (Tyruko®) was approved as a biosimilar to natalizumab (Tysabri®).^7,8

Vaccines

A special type of vaccine called an inverse vaccine is being investigated for MS. Typical vaccines work by teaching the immune system to attack specific targets. Inverse vaccines work by tricking the immune system into not attacking something. In the case of MS, the inverse vaccine would keep the immune system from attacking myelin. The vaccine in trials is called ANK-700.⁷

Primary progressive MS

Most treatments that are studied for RRMS do not work for PPMS. But some trials on treatments for PPMS have shown promising results.^7,9

Ocrevus was previously approved for PPMS. But it was only tested for people with certain ages and disease progression. New trials have shown it is safe and effective for a wider range of people.⁷

CAR T-cell therapy changes a patient's T cells so the T cells get rid of disease-causing B cells in the body. A new CAR T-cell therapy clinical trial recently received fast-track status from the US Food and Drug Administration (FDA). This clinical trial is testing KYV-101.⁹

KYV-101 is a CAR T-cell therapy for people with PPMS who have not responded to other MS treatments. It will target CD19, a protein expressed on the surface of B cells. CD19 is associated with many autoimmune diseases, including MS. Researchers are hopeful that this treatment can bring much-needed relief to those with PPMS.⁹

Stem cells and bone marrow transplants

Stem cell therapy is a treatment that uses stem cells. Stem cells are special cells that can turn into many different types of cells in the body. The use of stem cells for the treatment of MS is an up-and-coming research area. There are different stem cell treatments in clinical trials. For example, some involve using stem cells to form new cells to make or repair myelin.^3,10

Bone marrow transplants are studied for using your own stem cells to fight MS. This approach aims to “reboot” an overactive immune system. First, your healthy stem cells are taken. Then the body’s immune system is destroyed with drugs like chemotherapy. Once the immune cells are depleted, the stem cells are put back into the body. Then they can help develop a new healthy immune system.^3,10

Combination treatments

Combination treatments involve more than one treatment. Combination treatments and testing can be difficult. Two drugs can interact in unexpected ways. It can also be hard to prove which treatment is working in a study.¹

One study looked at women with MS. The women took estriol, a pregnancy hormone, along with the approved MS drug glatiramer acetate (CopaxoneⓇ). The combination therapy resulted in lower levels of certain substances in the women’s blood. These substances indicate nerve damage.¹¹

Participation and representation

Clinical trials are the best method for discovering new, safe, and effective treatments for MS. Women and people of color tend to be underrepresented in clinical trials. Learning how you can participate in trials is the best way to help make sure treatments and therapies apply to all people.^1,4

If you are interested in clinical trials, you can find out which studies you may qualify for near you.