Nerve Growth Factor Study

Legacy Study (Closed for Enrollment)

A Phase II clinical study of Ceregene’s CERE-110, a gene therapy product designed to deliver nerve growth factor (NGF) to the brain for the treatment of Alzheimer’s disease (AD) is currently underway.

This Phase II study is a randomized, double blind, placebo-controlled trial and employs gene therapy to deliver nerve growth factor (NGF) directly into the brain. The rationale behind this study is that NGF is known to promote survival of certain neurons, called cholinergic neurons, that degenerate in AD, and therefore may provide sustained functioning of these neurons. Direct delivery of CERE-110 into the brain aims to selectively target the Nucleus Basalis of Meynert (NBM), where cholinergic neuronal degeneration occurs in AD.

A Phase I study at Rush University and the University of California San Diego indicated that a single administration of the therapy was generally safe and well tolerated. The Phase I participants underwent cognitive testing, measures of activities of daily living, MRI scans and PET (positron emission tomography) scans. Increases in brain metabolism were observed in several cortical regions of the brain at six months and 12 months in some of the subjects, as compared to other severity-matched individuals with AD, suggesting a potential reversal of patterns typically observed in AD.

This Phase II study will examine the safety and effectiveness of NGF on Alzheimer’s disease in 50 patients at 11 research sites throughout the United States. All eligible participants will be randomized equally to one of two treatment groups: half of the subjects will initially receive placebo surgery, but no administration of gene therapy. In the other half, CERE-110 will be injected into the NBM of the brain. At the completion of the trial, subjects in the placebo arm will be given the opportunity to switch to the active treatment protocol if the efficacy and safety data are supportive.

To be eligible for this study participants must:

  • Have mild to moderate AD with an MMSE score of 17 to 26
  • Be in good general health
  • Be on standard of care medications (acetylcholinesterase inhibitors and/or memantine) for AD at least three months prior to entry.
  • Have a reliable study partner to accompany all study visits

Any of the following will exclude participation:

  • Significant neurological disease other than AD
  • Depression, major psychiatric disorders and behavioral problems
  • Alcohol or substance abuse within the last two years
  • Systemic cancer within the last three years
  • Any significant systemic illness or unstable medical condition

Participants will be screened at the research sites to determine if they are eligible for the study. To learn how to participate contact one of the above centers or contact the NIA’s Alzheimer’s Disease Education and Referral (ADEAR) Center at 1-800-438-4380 or by email to To view a list of the research sites or for information on dementia and aging, go to

Clinical Study Research Sites Participating in the NGF Study:

Site City State Contact Telephone
University of Alabama, Birmingham Birmingham AL Jackie Vaughn (205)934-6126
Banner Sun Health Research Institute Sun City AZ Sherye Sirrel (623)832-6522
University of California, Los Angeles Los Angeles CA Celine Ossinalde (310)794-6191
Georgetown University Washington DC Kelly Behan (202)687-0413
Emory University Atlanta GA Tamara Attis (404)712-6914
Duke Health Center Durham NC Debra Heydt (919) 668-2843
Mount Sinai School of Medicine New York NY Kelly Pun (212)241-1514
Case Western Reserve University Cleveland OH Parianne Fatica (216)464-6474
Roper St. Francis Hospital Charleston SC Arthur Williams (843)724-2302 ext 6375
Medical University of South Carolina North Charleston SC Arthur Williams (843)724-2302 ext 6375
University of Utah Salt Lake City UT Josh Amaro (801)581-3986