Research seeking therapies for ALS is advancing at a rapid pace, and there are more potential treatments in clinical trials than ever before. Here, we look at six drugs currently in clinical trials for ALS: arimoclomol, Gilenya, ozanezumab, pyrimethamine, rasagiline and tirasemtiv.
How to use the interactive graphic: Select each drug name to view a brief description of the corresponding clinical trial. For even more detailed information about the drug trial, click on the drug name at the bottom of the brief description.
Gilenya (fingolimod, TDI132)
This drug already is approved by the U.S. Food and Drug Administration for the treatment of some forms of multiple sclerosis.
How it works: Gilenya, developed by Novartis, works by dampening the body's immune response. A growing body of evidence suggests that malfunction of the immune system contributes to the complex ALS disease process. Previous studies conducted in the SOD1 ALS research mouse have shown that blocking the activation of certain parts of the immune system slows disease progression and improves survival.
Purpose of trial: The study will determine whether Gilenya is safe and tolerable in people with ALS.
Read more about Gilenya.
How it works: Ozanezumab, developed by GlaxoSmithKline, is thought to work by helping protect neuromuscular junctions, where muscles interact with nerves. The drug may help repair damaged axons, the long fibers that carry signals to and from nerve cells in the brain and spinal cord.
Purpose of trial: Assess efficacy and safety of intravenous (injected into a vein) ozanezumab compared to placebo in people with ALS.
Read more about Ozanezumab.
How it works: Arimoclomol, developed by CytRx Corporation, is believed to work by increasing production of the so-called "chaperone" molecules heat-shock protein 70 and heat-shock protein 90. These molecules aid in cell repair by helping newly synthesized proteins fold correctly, or by detecting proteins that are misfolded — and therefore potentially toxic — and refolding them into their correct shapes.
Purpose of trial: To see if arimoclomol has a beneficial effect on functional ability, disease progression and longevity in people with ALS.
Read more about Arimoclomol.
How it works: Tirasemtiv, developed by Cytokinetics, is designed to increase the sensitivity of muscle fibers to calcium, resulting in a potential increase in muscle force generation.
Purpose of trial: To evaluate the safety, tolerability and effectiveness of tirasemtiv when taken with or without riluzole by people with ALS. (Riluzole — brand name Rilutek — is the only drug approved by U.S. Food and Drug Administration to treat ALS.)
Read more about Tirasemtiv.
This drug is approved by the U.S. Food and Drug Administration for the treatment of Parkinson’s disease; it is marketed under the brand name Azilect.
How it works: Rasagiline, which belongs to a class of drugs called monoamine oxidase B inhibitors, has demonstrated neuroprotective properties in cell culture studies and in the SOD1 research mouse model of ALS.
Purpose of trial: To determine if rasagiline is safe and if it slows disease progression in people with ALS.
Read more about Rasagiline.
This drug is approved by the U.S. Food and Drug Administration for the treatment of malaria and toxoplasmosis; it is marketed under the brand name Daraprim.
How it works: Pyrimethamine reduces levels of SOD1 protein. Although mutations in the SOD1 gene primarily are associated with the familial form of ALS, toxicity from misfolded SOD1 protein has been seen in both familial and sporadic (in which there is no known family history) ALS.
Purpose of trial: To evaluate the safety and tolerability, and effect of pyrimethamine on SOD1 protein levels in people with familial ALS.
Read more about Pyrimethamine.
Note: The effect of these drugs on people with ALS is still unknown. Individuals are strongly advised not to use these drugs to treat ALS until they have been proven safe and effective, and have been approved for use in ALS. Always consult with your physician before adding any medications to your treatment plan.