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Abstracts from the International Congress of Parkinson’s and Movement Disorders.

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GDNF Gene Therapy for Parkinson’s Disease (PD): Preliminary Safety and Clinical Findings from a Phase Ib Study

A. van Laar, C. Christine, A. Merola, N. Phielipp, B. Elder, P. Larson, N. Stoicea, W. San Sebastian, M. Fiandaca, A. Kells, K. Bankiewicz (Research Triangle Park, USA)

Meeting: 2022 International Congress

Abstract Number: 406

Keywords: Glial-derived neurotrophic factor(GDNF), Parkinson’s, Stereotactic neurosurgery

Category: Therapy in Movement Disorders: Gene and Cell-Based Therapies

Objective: Assess the safety and tolerability of intraputaminal AAV2-GDNF in mild to moderate PD

Background: Glial cell line-derived neurotrophic factor (GDNF) is critical for dopaminergic neuron development and survival. The neurorestorative potential of GDNF is evaluated in this Phase Ib trial, using a single high AAV2-GDNF vector dose and increased putaminal coverage to test the safety and possible clinical benefit in 2 PD cohorts on levodopa treatment. An earlier study of lower doses in advanced PD showed safety and stabilization of symptoms for up to 5 years.

Method: Six PD participants in the “Mild Cohort” (MDS-UPDRS III OFF <32; PD diagnosis 2.1±0.6 years; mean LED 541mg/day) and 4 in the “Moderate Cohort” (MDS-UPDRS III 33-60; PD diagnosis 8.3 ± 0.7 years; mean LED 840mg/day) underwent MRI-monitored, direct intracranial convective delivery of AAV2-GDNF (3.3E+12 vg/mL) to cover >50% of each putamen (≤1.8mL per putamen). Safety assessments, including MRIs at 6- and 18-months, and impact of AAV2-GDNF gene delivery on PD symptomatology was measured via neurological assessments (MDS-UPDRS, PD Motor Diary, NMSS, PDQ-39), and changes in LED, are ongoing. Data reported as mean absolute change±SEM.

Results: The neurosurgical procedure was well tolerated and all 10 participants have completed 9-18 months of clinical follow-up. Putaminal coverage was 63%±2% and 6-month MRIs showed expected post-surgical changes. No SAEs were associated with AAV2-GDNF. Reported AEs primarily occurred peri-operatively or were related to underlying PD.

Mild cohort: At the 6-month assessments compared to pre-treatment scores: MDS-UPDRS III in OFF state -2.7±2.3 pts, MDS-UPDRS II 0.8±1.4pts, Diary OFF time 0.8±0.8hrs with similarly unchanged scores on PDQ-39 and NMSS.

Moderate cohort: The 6-month assessments: MDS-UPDRS III in OFF state -13.0±3.7pts, MDS-UPDRS II -2.3±4.3pts, Diary OFF time -3.4±0.7hrs. Improvements were noted on PDQ-39 and NMSS.

Conclusion: A positive safety profile is emerging from this Phase Ib study of AAV2-GDNF in 2 cohorts of levodopa treated PD subjects. Although the placebo effect limits interpretation of small open-label studies, these preliminary findings show stabilization in the Mild Cohort and possible early improvements in the Moderate Cohort. Further longitudinal evaluation and a controlled study is planned to confirm these initial findings.

To cite this abstract in AMA style:

A. van Laar, C. Christine, A. Merola, N. Phielipp, B. Elder, P. Larson, N. Stoicea, W. San Sebastian, M. Fiandaca, A. Kells, K. Bankiewicz. GDNF Gene Therapy for Parkinson’s Disease (PD): Preliminary Safety and Clinical Findings from a Phase Ib Study [abstract]. Mov Disord. 2022; 37 (suppl 2). https://www.mdsabstracts.org/abstract/gdnf-gene-therapy-for-parkinsons-disease-pd-preliminary-safety-and-clinical-findings-from-a-phase-ib-study/. Accessed June 24, 2025.
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