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Mark H Tuszynski1,2, Douglas W Scharre3, Gabriel C Leger1

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This Phase 1 clinical trial shows AAV2-BDNF gene therapy is safe for mild Alzheimer's disease (AD) and mild cognitive impairment (MCI). Treated patients show restored brain metabolism, suggesting a potential neuro-restorative therapy.

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Area of Science:

  • Neuroscience
  • Gene Therapy
  • Neurology

Background:

  • Brain-Derived Neurotrophic Factor (BDNF) is crucial for neuronal survival, function, and cognitive processes.
  • Animal models of Alzheimer's disease (AD) show BDNF gene delivery improves cognitive performance.
  • Previous studies demonstrated BDNF's benefits in preclinical models, supporting its advancement to human trials.

Purpose of the Study:

  • To evaluate the safety and efficacy of AAV2-BDNF gene delivery in patients with biomarker-supported mild AD and Mild Cognitive Impairment (MCI).
  • To assess the impact of AAV2-BDNF gene therapy on cognitive function and brain metabolism in AD and MCI patients.

Main Methods:

  • A Phase 1 clinical trial involving MRI-guided injection of AAV2-BDNF into the entorhinal cortex.
  • Recruitment of 12 patients (6 mild AD, 6 MCI) monitored over 2 years.
  • Serial cognitive testing and Fluorodeoxyglucose Positron Emission Tomography (FDG-PET) scans for monitoring.

Main Results:

  • AAV2-BDNF gene therapy was administered to 6 mild AD patients with follow-up up to 18 months.
  • No serious adverse events related to the AAV2-BDNF treatment were reported.
  • FDG-PET scans in three patients treated for at least 6 months showed increased cortical metabolism in the entorhinal cortex, reversing the typical AD decline.

Conclusions:

  • AAV2-BDNF gene therapy delivered to the entorhinal cortex is safe in mild AD patients.
  • The treatment restored metabolic activity in the targeted brain region, as evidenced by FDG-PET scans.
  • BDNF gene therapy presents a potential neuro-restorative approach for AD and MCI by preventing neuronal loss and enhancing function.