What’s in the pipeline of treatments for amyotrophic lateral sclerosis?
29 Mar 2009
Amyotrophic lateral sclerosis (ALS, also called Lou Gehrig's disease) is a rapidly progressing, motor neuron disease characterized by the gradual degeneration and death of motor neurons. When muscles in the diaphragm and chest wall fail, patients lose the ability to breathe without ventilatory support. Most people with ALS die from respiratory failure. Approximately 120, 000 new cases are diagnosed each year. No cure has yet been found for ALS. There is one FDA approved drug, riluzole, that modestly slows the progression of ALS.

Quite a number of approaches to treat ALS are being investigated in clinical trials: of the 20 different approaches, the majority (16) are small molecule based treatments, followed by biologics such as proteins, peptides and antibodies and more recently cell-based therapies have emerged for clinical testing. Among the targets being clinically tested are glutamate based therapies, antioxidants, cellular energy regulators, immunomodulators and neuroprotective/neurotrophic therapies. Biologic therapies study the neuroprotective effects of VEGF or are directed at the misfolded superoxide dismutase (SOD-1) among other approaches. Cellular therapies intent to induce regeneration of neuronal cells and tissues.