A patient-supported strategy for therapy development in amyotrophic lateral sclerosis (ALS)

Patient-supported foundations have had a significant impact on the scope of therapy development for the neurodegenerative disease, amyotrophic lateral sclerosis (ALS). In addition to sponsoring the investigation of potential curative therapies, the ALS Therapy Development Foundation (ALS-TDF) has also created an independent in vivo animal screening laboratory and is systematically evaluating the potential therapeutic benefit of drugs already approved by the FDA. A stepwise process of approved drug database development, in vitro drug screening using cell cultures, and 'theoretical' drug screening based on known drug mechanisms of action has narrowed the field from 1600 FDA-approved drugs to 90 candidates with potential therapeutic benefit. These drugs are now being evaluated in vivo using two animal models of ALS-the rat 'stroke' model and the transgenic SOD1 mouse model. Drug candidates showing significant benefit in these models compared with the current standard treatment for ALS will be selected for application in human patients.