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2017 Part the Cloud: Translational Research Funding for Alzheimer’s Disease (PTC)

A Clinical Trial of BDNF Gene Therapy in Alzheimer’s Disease

This Phase I clinical trial will test the safety of a novel therapy to prevent nerve cell loss in Alzheimer’s disease

Mark Tuszynski, M.D., Ph.D.
University of California, San Diego - Health Sciences
La Jolla, CA - United States


BDNF (brain-derived neurotrophic factor) is a protein that occurs naturally in the brain and is essential for the health and survival of nerve cells. It has been challenging to develop BDNF as a drug therapy because it does not enter the brain from the bloodstream. Mark Tuszynski, M.D., Ph.D and colleagues have been optimizing a novel technique for delivering the BDNF gene directly into the brain. This gene therapy technique requires only a single injection and leads to prolonged increases in brain BDNF levels.

In previous studies, Dr. Tuszynski has shown that the BDNF gene therapy improves the survival and function of brain cells in Alzheimer’s-like mice and non-human primates. In particular it protects nerve cells that are susceptible to dying in Alzheimer’s disease. This mechanism differs from many other treatments being developed for Alzheimer’s because it targets the survival of nerve cells rather than the removal of beta-amyloid (“plaques”) and tau protein (“tangles”) from the brain.

Research Plan

For their current study, Dr. Tuszynski and colleagues will conduct a Phase I clinical trial of BDNF gene therapy in people with early Alzheimer’s disease. The researchers will use magnetic resonance imaging (MRI) to guide injection of the BDNF gene into specific brain regions affected by Alzheimer’s disease. The study participants will undergo brain imaging and cognitive testing to examine changes in brain structure and function, and will be followed for 2 years to determine any long-term side effects of the treatment.


This study represents the first human clinical trial of BDNF gene therapy for Alzheimer’s disease. It is an essential step in determining the long-term safety of BDNF gene therapy and will inform the development of larger clinical trials that will study its effectiveness as a treatment to slow, halt or prevent the progression of Alzheimer’s disease.

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