Researchers to Test First Gene Therapy For Alzheimer’s Patients
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Mount Sinai School of Medicine is one of 12 sites nationwide participating in the first Phase 2 clinical trial to test gene therapy treatment for Alzheimer’s disease. The study is the first multicenter neurosurgical intervention in Alzheimer’s research in the U.S.
The experimental treatment utilizes a viral-based gene transfer system, CERE-110, that makes Nerve Growth Factor (NGF), a naturally occurring protein that helps maintain nerve cell survival in the brain. CERE-110 has been previously studied in animals, where it reversed brain degeneration in aged monkeys and rats. For this study, CERE-110, will be injected by a neurosurgeon directly into the nucleus basalis of Meynert (NBM) of the brain, an area where neuronal death occurs in Alzheimer’s patients.
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.
A Phase 1 study in Alzheimer’s patients has been conducted at Rush University in Chicago and the University of California San Diego, where researchers observed increases in brain metabolism in several cortical regions of the brain at 6- and 12-month follow-up in some of the participants. 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.
Participants in the Phase 2 study will be randomly placed into one of two treatment groups, with half receiving CERE-110 via neurosurgery and half receiving placebo surgery without any cranial injections. Once the study is completed, and if the results are promising, participants in the placebo group will be eligible to be treated with CERE-110. All participants will receive a thorough medical examination and cognitive testing. In addition, participants will be closely monitored by a team of physicians for the duration of the two-year study. Participants will also be encouraged to participate in long-term follow-up.
The study, to be conducted at 12 sites throughout the country, is the first multicenter neurosurgical intervention in Alzheimer’s research in the United States. The local study at Mount Sinai will involve approximately four to six volunteers between the ages of 50 and 80 with mild to moderate Alzheimer’s symptoms. People seeking more information about participating in the study should call Mount Sinai at (212) 659-8885.
More information about this Phase 2 trial can be found on the ADCS website at http://www.adcs.org/Studies/NGF.aspx , and at the NIA’s Alzheimer’s Disease Education and Referral Center (ADEAR) website at http://www.alzheimers.org/clinicaltrials/fullrec.asp?PrimaryKey=308.
The study is sponsored by the Alzheimer’s Disease Cooperative Study (ADCS) through a grant from the National Institute on Aging (a part of the NIH) in association with Ceregene, Inc, which developed and will provide the active agent (CERE-110).
About The Mount Sinai Medical Center
The Mount Sinai Medical Center encompasses The Mount Sinai Hospital and Mount Sinai School of Medicine. The Mount Sinai Hospital is one of the nation’s oldest, largest and most-respected voluntary hospitals. Founded in 1852, Mount Sinai today is a 1,171-bed tertiary-care teaching facility that is internationally acclaimed for excellence in clinical care. Last year, nearly 50,000 people were treated at Mount Sinai as inpatients, and there were nearly 450,000 outpatient visits to the Medical Center.
Mount Sinai School of Medicine is internationally recognized as a leader in groundbreaking clinical and basic science research, as well as having an innovative approach to medical education. With a faculty of more than 3,400 in 38 clinical and basic science departments and centers, Mount Sinai ranks among the top 20 medical schools in receipt of National Institute of Health (NIH) grants.
Source: The Mount Sinai Medical Center
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