// News Entry

Fall/Winter 2009

Mount Sinai Researchers to Test First Gene Therapy to Improve Brain Function In Alzheimer’s Patients

AD is a degenerative and ultimately fatal disorder affecting as many as five million Americans; that number is expected to soar to more than 11 million by 2040. Scientists are actively looking at new and more innovative ways to treat the disease. Now, for the first time in AD research, scientists are about to test the efficacy of a gene therapy called CERE-110.  Mount Sinai School of Medicine is one of 12 sites in the nation selected to participate in a multicenter clinical trial to examine the safety, efficacy, and benefits of a neurosurgical gene therapy technique for Alzheimer’s disease (AD).  Judy Neugroschl, M.D., an expert in dementia and Ron Alterman, M.D., an experienced neurosurgeon, will join efforts for this aggressive new approach to treat AD. Although neurosurgical techniques are used to treat other neurological disorders, there is no FDA approved surgical treatment for AD. In fact, relatively few studies have utilized this approach in AD research.

The experimental treatment utilizes a viral-based gene transfer system, CERE-110, that makes Nerve Growth Factor (NGF). NGF is a naturally occurring protein that helps maintain nerve cell survival in the brain. In animal studies, NGF has been shown to support the survival and function of the neurons that deteriorate in Alzheimer’s patients. These neurons produce the chemical acetylcholine, which is important in memory and cognitive function. The hope is that improvement of this system’s function may lead to better memory performance in Alzheimer’s patients.

CERE-110 is an experimental treatment that will be injected into a specific region of the brain that is affected by AD, the nucleus basalis of Meynert (NBM).  CERE-110 has been studied in animals and humans.  In aged monkeys and rats, CERE-110 reversed brain degeneration.  Thus, these studies demonstrated that CERE-110 can safely induce long-term production of Nerve Growth Factor (NGF) by brain cells. A Phase 1 study conducted with humans was performed at Rush University in Chicago and the University of California San Diego, where the treatment was found to be generally safe and well tolerated. 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.  This suggests a potential reversal of patterns typically observed in AD. With follow up ranging from six months to more than four years post-treatment, there have been no side effects thought to be caused by CERE-110.

The Phase 2 trial will be conducted at 12 U.S. sites, including Mount Sinai School of Medicine’s Alzheimer Disease Research Center.  The local study will involve approximately four to six volunteers between the ages of 50 and 80 with mild to moderate Alzheimer’s symptoms.

For more information about this study, please contact Judy Creighton, M.A., at 212-659-8886.

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