Augie Nieto’s fight against amyotrophic lateral sclerosis will open a new front at UCI Medical Center in Orange, with an upcoming clinical trial for a drug under development by a research institute he has led as chairman since 2007.
The Cambridge, Mass.-based ALS Therapy Development Institute said last week that the Food and Drug Administration granted it permission to conduct a clinical trial of a drug called TDI-132 for amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease.
ALS is a progressive neurodegenerative disease that leads to paralysis through damage of motor neurons in the spinal cord and brain. It is currently incurable, and the average patient survives only two to five years following diagnosis.
Nieto is a Corona del Mar resident who founded and served as chief executive of exercise bicycle maker Life Fitness, eventually selling his stake to Lake Forest, Ill.-based Brunswick Corp. as part of $310 million.
TDI-132 is marketed by Switzerland-based Novartis AG under the name of Gilenya for treating some forms of multiple sclerosis. Preclinical research by the ALS Therapy Development Institute showed Gilenya to have a positive impact on disease progression.
The clinical trial at UCI Medical Center and other locations will aim to determine whether Gilenya can be given safely to ALS patients. That requires an extensive clinical trial, and UCI researchers will be joined in the effort by colleagues at three other institutions.
“Seeing TDI-132 enter into clinical trial for ALS gives me hope that people living with ALS may soon be able to fight back,” Nieto said in a press release.
Nieto was diagnosed with ALS almost eight years ago and is now confined to a wheelchair. He and his wife, Lynne, have been supporting the ALS Therapy Development Institute, one of a number of efforts they’ve undertaken to battle ALS under the banner of Augie’s Quest, a nonprofit foundation that is part of the Muscular Dystrophy Association and has raised more than $35 million.
The research institute in Cambridge, which also is a nonprofit, has raised about $100 million since it was founded in 1999, and grown to more than 50 employees, with about 30 drug development staff members and 26,000 square feet of laboratory space.
The institute holds patents and stakes in the drugs under its development, which points to the potential for eventual milestone payments and royalties that would put the foundation and research on a self-funded footing. It is the sole funding sponsor of the trial.
The institute has numerous other efforts in its pipeline, including dozens of small molecules, protein biologics and gene therapies. It collaborates with academic institutions and businesses, including the Muscular Dystrophy Association and Weston, Mass.-based Biogen Idec Inc.
UCI Medical Center, a teaching hospital in Orange, is one of the current enrollment sites for the trial.
The trial will look at the safety and tolerability of Gilenya in people who have ALS.
Other Sites
Institute researchers started preclinical testing of Gilenya in 2011 to see if the drug can block certain immune cells from entering the brain and spinal cord, where they can cause activities that result to motor neuron damage.
Other enrollment sites include Massachusetts General Hospital in Boston, Georgia Health Sciences University in Augusta, Ga., and the Methodist Neurological Institute in Houston.
