Recent Progress in Advancing Gene Therapy for Parkinson Disease: Russell Lonser, MD


The director for the Gene Therapy Institute at The Ohio State University talked about the latest developments in gene therapy for patients with Parkinson disease. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

"I think what gene therapy provides us is a new opportunity to really target the specific problem of the disease at its core."

AB-1005 (AskBio), an investigational adeno-associated viral vector serotype 2 (AAV2) gene therapy containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, was recently assessed in a phase 1b trial (NCT04167540) for Parkinson disease (PD) treatment. The trial primarily looked at safety and potential clinical effect of AB-1005, with other secondary outcomes that assessed motor and non-motor function, as well as brain dopaminergic network integrity through DaTSCAN. New data from the phase 1b study showed that the therapy met its primary end point of successful putamen coverage and was safe over an 18-month period.

In the study, which follows patients for up to 5 years post administration, scheduled 6-month postoperative MRIs revealed findings of asymptomatic unilateral T1 hypointensity adjacent to 3 of the putaminal infusion trajectories. Although the study was small scale, with only 11 patients included, those with mild (n = 6) and moderate (n = 5) forms of PD experienced improvements in ON and OFF time with AB-1005.1 Presented at the 2024 American Academy of Neurology (AAN) Annual Meeting, held April 13-18, in Denver, Colorado, neurosurgical delivery of AB-1005 resulted in putamen coverage of 63% (±2%), exceeding the goal of greater than 50% coverage. Bilateral infusions of the agent in the putamen (up to 1.8 mL) were well tolerated, with no serious adverse events associated with the gene therapy or contrast agent.

Russell Lonser, MD, director for the Gene Therapy Institute and chair of the department of neurological surgery at The Ohio State University, recently sat down with NeurologyLive® in an interview to discuss the key challenges in optimizing gene therapy for PD, particularly regarding target selection and gene choice. He also spoke about how findings from the recent phase 1b trial further contribute to the understanding of the potential of gene therapy's efficacy and safety in treating PD. In addition, Lonser talked about the potential implications of gene therapy advancements for the future treatment landscape of neurological disorders beyond PD.

1. AskBio presents 18-month phase 1b trial results of AB-1005 gene therapy for patients with Parkinson disease. News release. AskBio. April 16, 2024. Accessed May 6, 2024.
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