The green light has been given for Mark Tuszynski and his colleagues from the University of California, San Diego, the Salk Institute, and UC Davis to start a Phase I (safety) trial of nerve growth factor (NGF) gene therapy for early onset Alzheimer's. Eight patients will be enrolled to receive autologous transplants of skin fibroblasts engineered to produce large amounts of NGF. The experiment targets the nucleus basalis of Meynert, a basal forebrain area that sends cholinergic projections to the hippocampus and cortex.
The NIH's Recombinant DNA Advisory Committee gave approval for the trial in part based on promising results from a study in monkeys reported in the September 14 issue of the Proceedings of the National Academy of Sciences. Tuszynski and his colleagues described significant atrophy of cholinergic neurons in the basal forebrain of aged monkeys, and they were able to reverse that degeneration with transplants of tissue engineered to generate elevated levels of NGF.
For the human safety trial, the researchers are enrolling only healthy patients, free of aphasia symptoms, who have a firm diagnosis but are in the early stages of early-onset disease (Mini Mental State Exam score 21 to 28). Inquiries should be directed to Mark Tuszynski, University of California, San Diego, La Jolla, CA 92093-0626, USA.—Hakon Heimer
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- Smith DE, Roberts J, Gage FH, Tuszynski MH. Age-associated neuronal atrophy occurs in the primate brain and is reversible by growth factor gene therapy. Proc Natl Acad Sci U S A. 1999 Sep 14;96(19):10893-8. PubMed.