Evidence map›Paper›PMID 42091584›Full record

ReviewJournal of Parkinson's disease2026

Clinical GDNF delivery methods for Parkinson's disease.

Matthias Luz, Massimo S Fiandaca, Krystof S Bankiewicz

Abstract readReview
In one paragraph

Review in Journal of Parkinson's disease, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

3 authors.

Matthias LuzML Clinical Solutions, Mannheim, Germany.ORCID 0000-0001-6646-3655
Massimo S FiandacaGene Therapy Institute, The Ohio State University, Columbus, OH, USA.ORCID 0000-0002-8376-1934
Krystof S BankiewiczGene Therapy Institute, The Ohio State University, Columbus, OH, USA.ORCID 0000-0002-0424-3566

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Intracerebral delivery of glial cell line-derived neurotrophic factor (GDNF) therapeutics continues to show promise, especially in Parkinson's disease (PD). However, randomized, placebo-controlled clinical trials using GDNF protein have been inconclusive to date. A major sham-surgery controlled trial using GDNF gene therapy commenced in 2024. In this review we aim to update the reader on the evolution and current state of the art of intracerebral delivery methods for GDNF protein and gene therapy in PD clinical trials. Our intent is to increase the awareness for the importance, subtleties, and pitfalls of intracerebral delivery when reviewing current available results for these therapies and their prospects going forward. We will compare and contrast GDNF protein infusion versus gene therapy strategies and define specific anatomical and physiological details in trial participants that continue to challenge clinicians and investigators attempting to maximize therapeutic coverage of the target putamen. Despite a growing consensus that convection-enhanced delivery (CED) is the optimal intracerebral infusion strategy for localized administration of therapeutics in general and gene therapy products in particular, there is less agreement on the need for related methods, such as the co-infusion of a gadolinium contrast agent and use of intraoperative magnetic resonance imaging (iMRI) to visualize the therapeutic distribution for optimizing target coverage. Whereas certain debates will continue, most investigators and clinicians respond positively to well-designed trials providing clean, conclusive results. Information included in this review is intended to provide additional insights to interested readers, allowing better assessment of details associated with upcoming GDNF therapeutic investigations.Plain language summary titleClinical Delivery Methods for Treatments Based on Glial Cell Line-Derived Neurotrophic Factor (GDNF) in Parkinson's Disease.

Indexed as

Drug Delivery SystemsGenetic TherapyGlial Cell Line-Derived Neurotrophic FactorParkinson DiseaseAnimalsGene Therapy AgentsHumansGDNF protein, humanGlial Cell Line-Derived Neurotrophic FactorAAV2adeno-associated viral vector serotype 2Convection-enhanced deliveryGDNFgene therapyglial cell line-derived neurotrophic factorintraputaminalParkinson's diseaseprotein therapy

Identifiers

PMID42091584
PMCPMC13435179

What OpenQuestion holds

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LicenceCC BY-NC
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.