December 13, 2007 

Possible ALS Drug Granted
Orphan Status by the FDA

A compound that could one day extend the lifespan of people living with an hereditary form of ALS, Lou Gehrig’s Disease, has been granted orphan drug status by the U.S. Food and Drug Administration.

Funding for study of second-generation antisense has been provided through The Association’s TREAT ALS (Translational Research Advancing Therapy for ALS) program to study the potential effectiveness of the drug on primates.

Approximately 5% to 10% of people with ALS have the hereditary form of the disease, called familial ALS. This approach targets the 2% of ALS carrying mutations in SOD1. Advancing this technology may have future implications for other forms of ALS as other abnormal proteins in the disease are discovered.

The Orphan Drug Act provides for economic incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the United States.

Before the drug can be tested on human beings in clinical trials, it is being tested for safety on primates by investigators Timothy Miller, M.D., Ph.D. and Don Cleveland, Ph.D., of the Ludwig Institute, University of California, San Diego and Richard Smith, M.D., of the Center for Neurologic Study plan. Isis will then need to apply to the FDA for IND (Investigating New Drugs) status if these tests are satisfactory.

“We are extremely pleased that this promising drug has received orphan status,” said Dr. Lucie Bruijn, science director and vice president of The Association. “This effort is a prime example of how important the TREAT ALS program is to support studies from the laboratory into pre-clinical development.”

The second-generation antisense, also referred to by the company as ISIS 333611, works by inhibiting Cu/Zn superoxide dismutase (SOD1), a molecule that is associated with familial ALS. In preclinical studies, the drug extended the lives of rats that show many features of ALS.

“ALS is a terrible disease in need of innovative and efficacious new therapies. We are pleased that ISIS 333611 has been granted orphan drug status for this small but important patient population,” said C. Frank Bennett, Ph.D., senior vice president of research at Isis Pharmaceuticals Inc.

TREAT ALS is aimed at developing one or more drugs in the next decade that will prevent, halt or significantly slow the course of the disease. This ambitious and pioneering effort focuses on accelerated drug discovery and clinical trials to bring new therapies from the bench to the bedside.

At the forefront in the battle against Lou Gehrig’s Disease, The Association directs and funds a comprehensive global research program to find a cure and treatments for this devastating disease.