The ALS Therapy Development Institute, a nonprofit biotech, will be launching a phase II clinical trial on TDI 132 (fingolimod)—currently used as treatment for multiple sclerosis—as a potential treatment for ALS (Lou Gehrig's disease).
The primary purpose of this phase II clinical trial will be to determine the safety and tolerability of TDI 132 (fingolimod/Gilenya) in people living with ALS today. The study will provide important information on dosing and safety for a subsequent efficacy study.
"We hope to learn from this study whether or not TDI 132 is safe to test in people living with ALS. Participants will be evaluated over a month-long exposure to the drug, and we'll be monitoring their response closely," said Merit Cudkowicz, M.D., director of the neurology clinical trials unit at Massachusetts General Hospital and one of the principal investigators.
ALS Therapy researchers first began preclinical testing TDI 132 in 2011 for its ability to block certain immune cells from entering the central nervous system where they can cause activities that result in damage to motor neurons. The institute has confirmed that TDI 132 significantly alters the trafficking of these immune cells through the bloodstream, resulting in fewer of them infiltrating into and damaging the nervous system.
Further experiments at ALS Therapy showed treatment with TDI 132 resulted in positive outcomes on several disease measures in preclinical studies in the SOD1 mouse model. A key partner of ALS Therapy, the Muscular Dystrophy Association, provided significant funding for some of that preclinical work, as did numerous other private donors.
ALS Therapy will be working with the Northeast ALS Consortium (NEALS) to assist with clinical trial design and to oversee the execution of the clinical trial.