Experimental Treatments for Multiple Sclerosis
There is no cure for multiple sclerosis (MS), but there are therapies approved to treat the disease and manage symptoms. A number of experimental therapies also are currently being tested in clinical trials, ranging from symptomatic treatments to those that target the underlying cause of the disease. Eventually, this research may even lead to a cure for MS.
Some of the therapies currently being developed to treat MS and its related symptoms are summarized below.
Amiselimod (formerly known as MT-1303) is an investigational sphingosine 1-phosphate (S1P) receptor modulator being evaluated for people with relapsing-remitting MS, inflammatory bowel disease, and other autoimmune diseases. It was last tested in Phase 2 trials.
ATL1102 is a therapeutic candidate for relapsing forms of MS. It is an antisense therapy designed to reduce levels of CD49d, potentially reducing inflammation and disease progression. It has been tested in Phase 2 trials in MS patients.
ATX-MS-1467 is a potential disease-modifying therapy for relapsing-remitting MS. The experimental treatment consists of four short peptides derived from the basic protein of myelin, which is a key autoantigen (a trigger for an auto-immune response) in MS. It was last studied in a Phase 2 trial.
CNM-Au8 is an experimental therapy being developed to treat patients with MS, as well as amyotrophic lateral sclerosis (ALS) and Parkinson’s disease. Preclinical studies have demonstrated that CNM-Au8 is able to protect motor neurons from severe damage and death. A Phase 2 trial is currently recruiting MS patients.
Evobrutinib is an investigational oral medication to treat relapsing MS. Evobrutinib blocks the BTK protein and prevents the activation and function of B-cells and subsequently curbs T-cell function and inflammation. Two Phase 3 trials are currently recruiting participants with relapsing forms of MS.
Fenebrutinib is an investigational oral therapy being tested in people with relapsing forms of MS and PPMS. An oral small molecule, it is designed to slow MS progression by preventing certain immune cells from driving the inflammation that damages nerve cells.
GA Depot is a long-acting formulation of glatiramer acetate that is under clinical investigation for relapsing forms of MS and PPMS. The active agent in GA Depot is a synthetic protein that’s designed to mimic a piece of myelin.
Ibudilast is a potential oral treatment for all forms of MS and other neurodegenerative disorders. The therapy has been marketed in Japan and Korea to treat post-stroke complications and bronchial asthma for more than two decades. Positive results from Phase 2 trials have been reported.
Laquinimod (also known as nerventra or ABR-215062) is an investigational therapy being developed to treat neurodegenerative diseases, including primary progressive and relapsing-remitting MS. It has been studied in Phase 3 trials in relapsing-remitting MS.
Lipoic acid (also known as alpha lipoic acid) is an antioxidant available as an over-the-counter supplement that is currently studied as a potential neuroprotector for people with MS. In animal models of MS, it has has been shown to reduce inflammation and degeneration of the optic nerve and spinal cord. A Phase 2 trial in progressive MS is currently recruiting.
Masitinib is an oral immunomodulatory medication being developed as a possible treatment for progressive forms of MS and other diseases. It belongs to a class of chemical compounds known as tyrosine kinase inhibitors. Positive results of a Phase 2b/3 trial were recently reported in primary progressive and non-active secondary progressive MS.
Minocycline is an oral antibiotic used to treat acne or bacterial infections, including respiratory and urinary tract infections. It is believed to have immunomodulatory, anti-inflammatory, and neuroprotective properties, all of which may have implications for MS. A Phase 3 trial is currently recruiting patients with clinically isolated syndrome.
Nabiximols is an oral spray containing two of the main compounds found in the cannabis plant that is approved in some countries for easing MS-related spasticity. While the therapy is approved under the brand name Sativex in 25 countries, it has not been approved for use in the U.S.
NDC-1308 is being developed to repair the myelin sheath of demyelinated axons (nerve fibers), a major cause of MS. It is specifically targeted to heal motor neuron damage in people with secondary progressive MS. It is in the preclinical phase of development.
Remibrutinib is an oral treatment that potently and selectively inhibits the Bruton’s tyrosine kinase enzyme, which plays a critical role in the inflammatory activity of certain immune cells such as B-cells and microglia. By blocking this protein, remibrutinib is expected to dampen the inflammatory activity that drives MS.
Rituximab is a monoclonal antibody currently being investigated as a potential therapy for MS. It is an approved treatment for various types of blood cancer, and is marketed under the name Rituxan in the U.S. and MabThera in Europe. It is currently used off-label for MS. A Phase 3 trial in relapsing-remitting MS is ongoing.
Simvastatin is an approved statin commonly used to lower cholesterol. It is being studied as a potential treatment to slow the progression of MS. Statins have been shown to potentially have anti-inflammatory and neuroprotective properties. Several trials are potentially recruiting patients to test the treatment.
Temelimab is a selective monoclonal antibody being developed for the treatment of MS. Studies have suggested that Temelimab may reduce the body’s inflammatory response. A Phase 2 trial is currently recruiting patients with relapsing forms of MS.
Tcelna is being developed as a personalized T-cell immunotherapy for people with secondary progressive MS. It is an autologous T-cell vaccine containing autoreactive T-cells. It was last studied in a Phase 2 trial.
Tolebrutinib is an investigational oral therapy being developed to treat relapsing and progressive forms of MS. It is an oral and selective small molecule inhibitor of the Bruton tyrosine kinase enzyme, which is critical for the activity of multiple immune cell types involved in MS progression.
Ublituximab (TGTX-1101) is an investigational anti-CD20 monoclonal antibody. Originally developed to treat blood cancers like non-Hodgkin’s lymphoma and chronic lymphocytic leukemia, ublituximab is also being studied as a potential infusion therapy for relapsing forms of MS.
Vidofludimus calcium, also known as IMU-838, is an experimental oral therapy with anti-inflammatory and neuroprotective properties that is being investigated for relapsing and progressive forms of MS.