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Parkinson's Disease: Treatment

Parkinson's Disease: Treatment

Neurodegenerative disorders, such as Parkinson's Disease (PD), involve the gradual and irreversible destruction of neurons in particular brain areas. These disorders exhibit standard features like proteinopathies, selective vulnerability of some neurons, and an interaction of intrinsic properties, genetics, and environmental influences in neural injury.
Parkinson's Disease is primarily a result of the loss of dopaminergic neurons in the substantia nigra pars compacta. The cornerstone of its...

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Neurodegenerative disorders are progressive diseases that cause irreversible damage and loss to neurons in specific brain areas. Examples of these disorders include Parkinson's disease, Alzheimer's disease, Multiple Sclerosis (MS), and Amyotrophic Lateral Sclerosis (ALS). These disorders share characteristics such as proteinopathies, selective neuronal vulnerability, and a complex interplay between genetic and environmental factors. The primary therapeutic goal for these conditions is to...

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Updated: May 8, 2026

Ole Isacson: Development of New Therapies for Parkinson's Disease

Ole Isacson: Development of New Therapies for Parkinson's Disease

Published on: April 29, 2007

Clinical GDNF delivery methods for Parkinson's disease.

Matthias Luz¹, Massimo S Fiandaca², Krystof S Bankiewicz²

¹ML Clinical Solutions, Mannheim, Germany.

Journal of Parkinson'S Disease

|May 6, 2026

Summary

Glial cell line-derived neurotrophic factor (GDNF) delivery for Parkinson's disease (PD) shows promise but requires optimized intracerebral methods. This review details GDNF protein and gene therapy delivery techniques, focusing on convection-enhanced delivery (CED) for improved treatment outcomes.

Keywords:

AAV2 Convection-enhanced delivery GDNF Parkinson's disease adeno-associated viral vector serotype 2 gene therapy glial cell line-derived neurotrophic factor intraputaminal protein therapy

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Last Updated: May 8, 2026

Ole Isacson: Development of New Therapies for Parkinson's Disease

Ole Isacson: Development of New Therapies for Parkinson's Disease

Published on: April 29, 2007

Generation of Induced Neural Stem Cells from Peripheral Mononuclear Cells and Differentiation Toward Dopaminergic Neuron Precursors for Transplantation Studies

Generation of Induced Neural Stem Cells from Peripheral Mononuclear Cells and Differentiation Toward Dopaminergic Neuron Precursors for Transplantation Studies

Published on: July 11, 2019

Area of Science:

Neuroscience
Biomedical Engineering
Gene Therapy

Background:

Intracerebral glial cell line-derived neurotrophic factor (GDNF) delivery is a promising therapeutic strategy for Parkinson's disease (PD).
Previous clinical trials using GDNF protein have yielded inconclusive results, necessitating advancements in delivery methods.
A new sham-surgery controlled trial for GDNF gene therapy began in 2024, highlighting the ongoing evolution of PD treatments.

Purpose of the Study:

To provide an updated review of intracerebral delivery methods for GDNF protein and gene therapy in PD clinical trials.
To emphasize the critical role of delivery techniques in interpreting trial results and future prospects.
To compare and contrast GDNF protein infusion versus gene therapy strategies, addressing challenges in targeting the putamen.

Main Methods:

Review of current literature and clinical trial data on intracerebral GDNF delivery for PD.
Comparison of convection-enhanced delivery (CED) for protein and gene therapy administration.
Discussion of anatomical and physiological challenges in achieving optimal therapeutic coverage of the putamen.

Main Results:

Convection-enhanced delivery (CED) is widely considered the optimal strategy for localized intracerebral therapeutic administration.
Debate continues regarding the necessity of co-infusion with gadolinium contrast agents and intraoperative MRI (iMRI) for visualizing distribution.
Well-designed trials with clear results are crucial for advancing GDNF-based therapies for PD.

Conclusions:

Optimizing intracerebral delivery methods is paramount for the success of GDNF-based therapies in Parkinson's disease.
Understanding the nuances of delivery techniques, such as CED, is essential for evaluating current and future clinical trial outcomes.
Further research and well-designed trials are needed to fully elucidate the potential of GDNF in treating PD.