Intraputaminal Delivery of Adeno-Associated Virus Serotype 2-Glial Cell Line-Derived Neurotrophic Factor in Mild or Moderate Parkinson's Disease.

ABSTRACT:

Background

Glial cell line-derived neurotrophic factor (GDNF) is required for development and survival of dopaminergic neurons. A previous trial evaluating lower-dose adeno-associated virus serotype 2-GDNF (AAV2-GDNF) bilateral intraputaminal infusion in participants with advanced Parkinson's disease (PD) achieved 26% mean putaminal coverage and was associated with stable motor features with no unexpected adverse events (AEs) over 60 months.

Objective

We assessed safety and preliminary clinical outcomes of optimized bilateral intraputaminal infusion of a single, higher dose of AAV2-GDNF (product code AB-1005) for PD after 18 months.

Methods

This phase 1b single-arm, open-label clinical trial enrolled participants with mild (Movement Disorder Society-revised Unified Pa

SUBMITTER: Van Laar AD 

PROVIDER: S-EPMC12273615 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature