A single-center, open-label, phase 1 dose-escalation study evaluated the 5-year safety and tolerability of bilateral intraputaminal convection-enhanced delivery (CED) of AAV2-GDNF in 13 adults with advanced Parkinson's disease across three dose cohorts (6 low, 6 medium, 1 high dose).
Over 5 years, 562 adverse events were recorded; only 45 were possibly or probably drug-related, and none of the 13 serious adverse events were attributed to the study drug. Mean putaminal coverage was 26% ± 10%. Exploratory clinical outcomes did not change significantly from baseline to final follow-up.
- Very small sample (n=13) with no control arm, limiting any efficacy conclusions. - Putaminal coverage averaged only 26%, suggesting incomplete delivery that may have blunted any therapeutic signal. - Single high-dose participant prevents dose-response analysis at the upper range.
Real-time image-guided CED of AAV2-GDNF into the putamen appears safe and well tolerated over 5 years in advanced PD, with no protocol-defined stopping events. However, with modest putaminal coverage and no control group, efficacy remains unestablished—this therapy is not yet ready for clinical practice outside of trials.
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