Gene therapy using BDNF to treat Alzheimer's disease
A Clinical Trial of AAV2-BDNF Gene Therapy in Alzheimer's Disease
This study is testing a new gene therapy that delivers a helpful protein directly into the brain to see if it can improve memory and brain function in people with Alzheimer's Disease or Mild Cognitive Impairment, and it’s designed for those who want to explore new treatment options.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-10867308 on NIH RePORTER |
What this research studies
This research investigates the use of BDNF gene therapy to treat Alzheimer's Disease (AD) and Mild Cognitive Impairment (MCI). The therapy aims to reduce neuronal loss and enhance neuronal function by delivering BDNF directly into the brain using advanced gene delivery techniques. Patients will receive the treatment through a minimally invasive procedure guided by real-time MRI, allowing for precise targeting of the entorhinal cortex. The trial will involve 12 participants, with a focus on assessing the safety and potential efficacy of this innovative approach.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Alzheimer's Disease or Mild Cognitive Impairment.
Not a fit: Patients with other forms of dementia or significant comorbidities that may interfere with the treatment are unlikely to benefit from this research.
Why it matters
Potential benefit: If successful, this research could slow or even reverse cognitive decline in patients with Alzheimer's Disease and Mild Cognitive Impairment.
How similar studies have performed: Previous animal studies have shown promising results with BDNF therapy, but this trial represents a novel first-in-human approach.
Where this research is happening
La Jolla, United States
- University of California, San Diego — La Jolla, United States (Active)
Researchers
- Principal investigator: Tuszynski, Mark H. — University of California, San Diego
- Study coordinator: Tuszynski, Mark H.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.