Gilenya (TDI-132 or fingolimod) is a therapy currently being researched for the treatment of amyotrophic lateral sclerosis (ALS) by the ALS Therapy Development Institute (ALS TDI).

Gilenya was developed by Novartis and is approved for the treatment of multiple sclerosis (MS), another neurodegenerative disorder.

How Gilenya works

ALS is a progressive and fatal neurodegenerative disease, where the damage and death of motor neurons (nerve cells that control the movement of muscles) lead to the loss of muscle control and paralysis. The disease’s cause is unknown, but it is believed that overactivity of the immune system can contribute to its progression. The immune system uses inflammation as a protective mechanism, but if this is activated inappropriately it can damage the body’s tissues and organs. In ALS, the disease’s progression often coincides with inflammation in the nervous system.

Gilenya is a sphingosine 1-phosphate (S1P) receptor modulator, or a molecule that controls the function of the S1P receptor, which is required for immune cells (or lymphocytes) to leave the lymph nodes, where they are made and enter circulation. Once in the bloodstream, these immune cells can travel to the target tissue (such as the nervous system in ALS) and trigger inflammation.

Gilenya acts to block signaling from the S1P receptor, trapping the immune cells in the lymph nodes and therefore preventing them from reaching the nervous system to cause inflammation. By reducing inflammation, it is thought that Gilenya will slow the progression of ALS.

Preclinical trials in mouse models of ALS demonstrated that Gilenya is able to significantly extend survival and improve symptoms.

Gilenya in clinical trials for ALS

A Phase 2a clinical trial, (NCT01786174), investigating the safety and tolerability of Gilenya compared to a placebo in patients with ALS has been completed. The randomized, double-blind, placebo-controlled trial enrolled 30 patients who were assigned to take either Gilenya or a placebo daily for four weeks. The patients were monitored for adverse events during the treatment period, and during a follow-up appointment at week 8. The trial met its primary endpoint, and results suggest that the treatment was well-tolerated in ALS patients. No serious adverse events were reported.

The next step in research includes moving to Phase 2b and Phase 3 clinical trials to assess the potential clinical benefits and long-term safety of Gilenya in people with ALS.

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