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Taking Optogenetics into the Human Brain: Opportunities and Challenges in Clinical Trial Design

Lookup NU author(s): Michael White, Dr Michael Mackay, Professor Roger Whittaker



This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).


Optogenetics, the use of light to control the activity of suitably sensitized cells, has led to major advances in the field of basic neuroscience since it first emerged in 2005. Already, the technique has entered clinical trials for conditions such as Retinitis Pigmentosa. A major focus of interest is the use of optogenetics within the brain, where the ability to precisely control the activity of specific subsets of neurons could lead to novel treatments for a wide range of disorders from epilepsy to schizophrenia. However, since any therapy would require both the use of gene therapy techniques to introduce non-human proteins, and implantable electronic devices to provide optical stimulation, applying this technique in the brain presents a unique set of obstacles and challenges. This review looks at the reasons why researchers are exploring the use of optogenetics within the brain. It then explores the challenges facing scientists, engineers and clinicians wanting to take this technology from the lab into the first human brain, discussing different possibilities for a first-in-human clinical trial from a sponsor, patient and regulatory perspective.

Publication metadata

Author(s): White M, Mackay M, Whittaker RG

Publication type: Review

Publication status: Published

Journal: Open Access Journal of Clinical Trials

Year: 2020

Volume: 12

Pages: 33-41

Online publication date: 25/08/2020

Acceptance date: 07/07/2020

ISSN (electronic): 1179-1519


DOI: 10.2147/OAJCT.S259702