New gene therapy for Parkinson’s disease: GDNF gene

New clinical trial (NCT04167540) aims to establish expression of GDNF gene in the brain, by delivering adeno-associated viruses (AAV2) to putamen. The gene produces protein called glial cell-line-derived neutrotrophic factor. It is hypothesized to enhance dopamine production in neural cells, leading to at least less pronounced symptoms.

The study will enroll 12 patient under 70 years of age. As it is the first phase trial, main outcomes are limited to safety and dosing. Single injection forces clinicians to use high viral loads to achieve desired results, which in turn can cause an adverse immunological response.

Completion is expected in December 2022.

This is at least the third study of GDNF insertion in gene therapies. Another, highly similar trial, plans to use GDNF in advanced Parkinson’s disease (NCT01621581) and ends in February 2022. There is also a study of GDNF in amyotrophic lateral sclerosis (NCT02943850) which nears completion, but its results are not available yet.

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