for people ages 50-80 (full criteria)
at San Diego, California and other locations
study started
estimated completion
Principal Investigator
by Mark Tuszynski



This is a first-in-human clinical trial to test whether a protein administered into the brain continuously by gene therapy, Brain-Derived Neurotrophic Factor (BDNF), will slow or prevent cell loss in the brains of people affected by Alzheimer's disease and Mild Cognitive Impairment. The protein may also activate cells in the brain that have not yet deteriorated. Gene therapy refers to the use of a harmless virus to have brain cells make the potentially protective protein, BDNF.

Official Title

A Phase I Study to Assess the Safety, Tolerability and Preliminary Efficacy of AAV2-BDNF [Adeno-Associated Virus (AAV)-Based, Vector-Mediated Delivery of Human Brain Derived Neurotrophic Factor] in Subjects With Early Alzheimer's Disease and Mild Cognitive Impairment


This is an open label Phase I clinical trial of AAV2-BDNF gene therapy for early Alzheimer's Disease (AD) and Mild Cognitive Impairment (MCI) in 12 participants. BDNF is a nervous system growth factor that regulates neuronal function in key memory circuits of the brain (the entorhinal cortex and hippocampus). BDNF reduces cell loss, stimulates cell function, and builds new connections (synapses) between brain cells in animal models. This clinical trial will use techniques of gene therapy because the candidate therapeutic protein, BDNF, does not cross the blood brain barrier (BBB). Two previous clinical programs of Nerve Growth Factor (NGF) gene therapy for AD and Neurturin gene therapy for Parkinson's disease in over 120 patients provided evidence that degenerating neurons respond to growth factors with classic "trophic" responses in the human brain. Participants will undergo one gene transfer procedure. Thus, dosing is performed only once, and repeat dosing or daily medications are not expected to be required. 12 participants will be enrolled in this Phase I trial, 6 with early AD and 6 with MCI.


Alzheimer's Disease Mild Cognitive Impairment Alzheimer Disease Cognitive Dysfunction Mitogens AAV2-BDNF Gene Therapy Gene transfer of AAV2-BDNF


You can join if…

Open to people ages 50-80

  • diagnosis of probable Alzheimer's Disease (AD) within 3 years of memory loss
  • diagnosis of Mild Cognitive Impairment
  • age between 50-80 years
  • resident of San Diego or Orange Counties in California, or Ohio.
  • primary language English with no aphasia (no communication impairment)

You CAN'T join if...

• seizure disorder


  • University of California - San Diego accepting new patients
    San Diego California 92093-0626 United States
  • Case Western Reserve University not yet accepting patients
    Cleveland Ohio 44106 United States

Lead Scientist at UCSD


accepting new patients
Start Date
Completion Date
Mark Tuszynski
Phase 1 research study
Study Type
Expecting 12 study participants
Last Updated