The Canavan trial signals a new phase in a 10-year offensive that gene therapy researchers have waged against neurodegenerative disorders. Previously limited mostly to cell-culture and animal experiments, the scientists are now poised or starting to take their protocols and reagents to the clinic.
In April, Mark H. Tuszynski, a neuroscientist at the University of California, San Diego, initiated an eight-subject trial in which he infects cultured fibroblasts with a recombinant virus and then injects the fibroblasts into Alzheimer's brains. Last summer, NIH's Recombinant DNA Advisory Committee (RAC) held a public meeting on a gene-therapy protocol for Parkinson's disease. In October 2000, a workshop at the National Institute of Neurological Disorders and Stroke (NINDS) hailed advances in gene therapy research on the lysosomal storage diseases, which include Tay-Sachs. And in July, five NIH institutes issued a Request for Applications (RFA-NS-02-007) to accelerate clinical use of gene-transfer methods developed by basic ...
