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Published on: August 25, 2017
Clinical trials in neurological disorders using AAV vectors: promises and challenges
Ronald J Mandel1, Corinna Burger
1University of Florida College of Medicine, Department of Neuroscience, PO Box 100244, Gainesville, FL 32610, USA. rmandel@ufl.edu
Abstract:
Currently, there are five phase I clinical trials of recombinant adeno-associated viral vectors for the treatment of neurological disorders that are approved or likely to be approved shortly. Two trials are testing different strategies to treat Parkinson's disease (PD), the third trial is aimed at treating Canavan's disease, a pediatric leukodystrophy, the fourth trial targets Alzheimer's disease (AD), and the fifth will attempt to target the lysosomal storage disorder, Batten's disease. All four clinical trials rely on the de novo expression of an enzyme or a trophic factor to correct neuropathology. Ironically, the theories used to choose enzymes for the two PD trials were widely divergent, whereas the enzymatic strategy used for one of the PD trials and the Canavan's trial have remarkable similarities. Other gene therapy treatment strategies for PD and other disorders, such as amyotrophic lateral sclerosis, are also on the horizon.
Insights
Gene therapy using recombinant adeno-associated viral vectors shows promise for neurological disorders like Parkinson's disease and Alzheimer's disease. Clinical trials are exploring enzyme replacement strategies to correct neuropathology.
Area of Science:
- Neurology
- Gene Therapy
- Biotechnology
Background:
- Recombinant adeno-associated viral vectors are emerging as a promising tool for treating neurological disorders.
- Several phase I clinical trials are underway or nearing approval for conditions including Parkinson's disease, Canavan's disease, Alzheimer's disease, and Batten's disease.
Purpose of the Study:
- To review the current landscape of gene therapy clinical trials utilizing recombinant adeno-associated viral vectors for neurological conditions.
- To highlight the diverse enzymatic strategies being employed and their underlying rationales.
Main Methods:
- Analysis of ongoing and upcoming phase I clinical trials for neurological disorders.
- Examination of the gene therapy approaches, focusing on enzyme or trophic factor replacement.
Main Results:
- Five phase I clinical trials are identified, targeting Parkinson's disease, Canavan's disease, Alzheimer's disease, and Batten's disease.
- The trials primarily use de novo expression of enzymes or trophic factors to address neuropathology.
- Divergent theoretical bases exist for enzyme selection in Parkinson's disease trials, with some overlap noted between a Parkinson's trial and the Canavan's disease trial.
Conclusions:
- Recombinant adeno-associated viral vector gene therapy is advancing into clinical trials for a range of severe neurological disorders.
- Enzyme replacement strategies are a common therapeutic approach, though the specific rationales can vary significantly.
- Gene therapy holds potential for treating other neurological conditions, including amyotrophic lateral sclerosis.
