Researchers at Banner Sun Health Research Institute are about to start the first Phase 2 research study to test a gene transfer agent for Alzheimer's disease (AD). AD is a degenerative and ultimately fatal disorder affecting as many as five million Americans and that number is expected to soar to more than 11 million by 2040. For the first time in AD research, scientists are about to test the efficacy of a gene transfer therapy called CERE-110.
Previously, CERE-110 was studied in a small study to assess safety. These studies showed that CERE-110 can induce long-term production of Nerve Growth Factor (NGF) by brain cells. Researchers are now seeking 50 study participants with mild to moderate AD for a Phase 2 clinical study at 12 U.S. research sites, including Banner Sun Health research Institute.
The study utilizes a viral-based gene transfer system that makes NGF, a naturally occurring protein that maintains nerve cell survival in the brain. A neurosurgeon injects CERE-110 directly into the nucleus basalis of Meynert (NBM) of the brain, an area where neuron death occurs in AD.
"The reasoning behind this study is that NGF is known to support the survival and function of the neurons that deteriorate in AD," says Marwan Sabbagh MD, director of clinical research at Banner Sun Health Research Institute. "These neurons produce the chemical acetylcholine, which is important in memory and cognitive function; restoration of this system's function may improve memory in AD patients."
A Phase 1 study was conducted at University of California San Diego. The 10 subjects underwent cognitive testing, measures of activities of daily living, and MRI and PET (positron emission tomography) scans. Researchers observed increases in brain metabolism in several cortical regions of the brain at six months and 12 months in some of the participants, as compared to other severity-matched individuals with AD, suggesting a potential reversal of patterns typically observed in AD.
The Phase 2 Clinical Study
In this blinded study, all eligible participants will be randomized by chance to one of two treatment groups: half will receive CERE-110 and half will receive placebo (inactive drug). Once the study is completed, and if the results are promising, participants in the placebo group will be eligible to receive CERE-110.
The NGF study is being conducted by the Alzheimer's Disease Cooperative Study (ADCS), a nationwide consortium of research centers and clinics supported by the National Institute on Aging (NIA), part of the National Institutes of Health, and coordinated by the University of California San Diego. Ceregene, Inc., the study sponsor, is a San Diego-based biotechnology company focused on the delivery of nervous system growth factors via gene transfer for the treatment of neurodegenerative disorders. Ceregene is providing CERE-110 for the study.