It is highly unlikely that a cure for ALS disease will come until we understand more about brain development, motor behavior and circuitry. Project ALS remains committed to supporting select basic research, as much of its 20 years of progress has resulted from the observations in this field. In fact, basic research has inspired many recent approaches to ALS therapies, including gene therapy, small molecule screenings, and stem cell approaches.
Project ALS enables communication between basic researchers and doctors. For example, a basic study in motor neuron biology in 2001, resulted in the generation of patient-specific motor neurons, a technique named Scientific Breakthrough of the Year by Time magazine in 2009. Doctors now routinely use a tool known as “ALS in a Dish”—a small model of an individual patient’s ALS—to screen for drugs.
Project ALS nurtures a network of scientists from academic centers including Columbia University, Harvard University, Boston Children’s Hospital, New York University, Massachusetts General Hospital, Johns Hopkins, The Salk Institute, Memorial Sloan Kettering Cancer Center, among others, that feed each other’s ideas and observations—and the Core. The Project ALS network shares one goal—to deliver drugs to human trial that will slow and/or stop ALS.