• Press Release

Mount Sinai Researchers Take Part in Phase 2 Clinical Trial Testing Gene Therapy for Alzheimer's Disease Patients

Mount Sinai School of Medicine is one of 12 sites participating in the Phase 2 study of neurological interventions for Alzheimer's disease research.

  • New York, NY
  • (November 16, 2009)

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 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.

About the Mount Sinai Health System

Mount Sinai Health System is one of the largest academic medical systems in the New York metro area, with more than 43,000 employees working across eight hospitals, over 400 outpatient practices, nearly 300 labs, a school of nursing, and a leading school of medicine and graduate education. Mount Sinai advances health for all people, everywhere, by taking on the most complex health care challenges of our time — discovering and applying new scientific learning and knowledge; developing safer, more effective treatments; educating the next generation of medical leaders and innovators; and supporting local communities by delivering high-quality care to all who need it.

Through the integration of its hospitals, labs, and schools, Mount Sinai offers comprehensive health care solutions from birth through geriatrics, leveraging innovative approaches such as artificial intelligence and informatics while keeping patients’ medical and emotional needs at the center of all treatment. The Health System includes approximately 7,300 primary and specialty care physicians; 13 joint-venture outpatient surgery centers throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and more than 30 affiliated community health centers. We are consistently ranked by U.S. News & World Report's Best Hospitals, receiving high "Honor Roll" status, and are highly ranked: No. 1 in Geriatrics and top 20 in Cardiology/Heart Surgery, Diabetes/Endocrinology, Gastroenterology/GI Surgery, Neurology/Neurosurgery, Orthopedics, Pulmonology/Lung Surgery, Rehabilitation, and Urology. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 12 in Ophthalmology. U.S. News & World Report’s “Best Children’s Hospitals” ranks Mount Sinai Kravis Children's Hospital among the country’s best in several pediatric specialties.

For more information, visit https://www.mountsinai.org or find Mount Sinai on Facebook, Twitter and YouTube.

Related Articles