Name: 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.
Skeletal formula for Gilenya (courtesy of ChemSpider).
Type of trial: Phase 2 clinical trial. The Gilenya trial is supported by the ALS Therapy Development Institute (ALS TDI), of Cambridge, Mass. It was designed with, and will be overseen by, experts affiliated with the Northeast ALS Consortium (NEALS), an ALS research network.
- University of California, Irvine, Orange, Calif.
- Georgia Health Sciences University, Augusta, Ga.
- Massachusetts General Hospital, Boston
- Methodist Neurological Institute, Houston
- The primary outcome measure is safety, to be measured by the occurrence of adverse events and by clinical changes in vital signs, ophthalmologic (eye) examination, physical examination, electrocardiogram (measurement of electrical activity in the heart) and standard clinical laboratory blood tests.
- Secondary outcome measures are tolerability (the ability of trial participants to complete the entire four-week study); and measured effect on circulating immune system cells called lymphocytes.
- Other (exploratory) outcome measures include rate of decline on the ALSFRS-R (ALS Functional Rating Scale —Revised), and breathing function as measured by slow vital capacity, or SVC (the maximum volume of air that can be exhaled slowly after slow maximum inhalation).
Currently recruiting? Not yet recruiting.
Background info: In 2011, ALS TDI demonstrated that, in an ALS mouse model, Gilenya is able to reduce the number of lymphocytes circulating throughout the bloodstream. This, in turn, limits the number of these cells able to enter the central nervous system, where they are known to engage in interactions that result in damage to motor neurons (the muscle-controlling nerve cells lost in ALS). Mice treated with Gilenya showed good outcomes on several disease measures.
- Gilenya in Amyotrophic Lateral Sclerosis (ALS)
- For a Q&A about ALS TDI's upcoming trial, read: General Information About ALS TDI's Phase 2A Clinical Trial of TDI-132 (Gilenya).
- View a March 12, 2012, webinar, featuring ALS TDI CEO and Chief Scientific Officer Steve Perrin and James Berry, a clinical research fellow and MDA grantee at Massachusetts General Hospital, discussing Gilenya's development and advancement to ALS clinical trials. (You must log in or register.)
To read about the other ALS clinical trials:
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.
About Clinical Trials
A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.
MDA has no ability to influence who is chosen to participate in a clinical trial.
To learn more, see Learn About Clinical Studies and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to ClinicalTrials.gov.