Though the Phase I study conducted at the University of California, San Diego (UCSD) was small and designed to test safety and toxicity, the subjects may also show some early indications of reduction in the advancement of their disease, according to the study's principal investigator Mark Tuszynski, M.D., Ph.D., professor of neurosciences at the UCSD School of Medicine.
"These results are intriguing," said Tuszynski. "If these effects are borne out in larger, controlled trials, this could be a significant advance over existing therapies for Alzheimer's disease."
Eight volunteers diagnosed with early-stage Alzheimer's disease participated in this study, with the first surgery performed in April 2001 at UCSD's John M. and Sally B. Thornton Hospital. Patients' own skin cells were genetically modified in culture to produce Nerve Growth Factor (NGF), a naturally occurring protein that prevents cell death and stimulates cell function. The NGF-producing cells were then surgically implanted into a deep brain region where cholinergic cell degeneration occurs in Alzheimer's disease. The cholinergic system is important in memory and cognitive function. Patients received the implanted grafts in targeted areas on either the left, right or both sides of the brain.
This human trial was undertaken following extensive studies in primates conducted by Tuszynski and colleagues , which showed that grafting NGF-producing tissue into the brains of aged monkeys restored atrophied brain cells to near-normal size a
Contact: Leslie Franz
University of California - San Diego