September 5, 2018

Lighting a Path to Precision Medicine



Today the Niskanen Center released a new research paper — Lighting a Path to Precision Medicine: Regulatory and Policy Implications of Optogenetic Technology — looking at the emerging science and potential commercial applications of optogenetics. Using light-activated proteins (opsins) embedded in certain cells, optogenetics allows for targeted and controlled activations of these specific cells. As the paper notes, opsin gene expression “offers an unprecedented ability to control specific cell types with precise timing, opening the door to real-time therapeutic control of complex physiological functions.”

The paper describes specific examples of neurological and psychiatric diseases and conditions that could one day benefit from advancements in optogenetic therapies. These include epilepsy, drug addiction, Alzheimer’s, Parkinson’s, spinal cord injuries, and a variety of mood disorders, such as anxiety and depression. While certain safety and efficacy limitations abound, this is a rapidly developing field that could soon be ripe for commercial applications. Indeed, a number of optogenetic therapies for Retinitis pigmentosa, a form of blindness, have already been approved by the Food and Drug Administration (FDA) for clinical trials in humans, and at least one company is currently in the process of attempting to approve its optogenetic treatment for pain therapy.

Optogenetics promises an innovative new means of delivering precision medical treatment for those suffering from a variety of diseases and disorders, and flexible FDA guidance can play an invaluable role in supporting pathways to market approval.

From the executive summary:

Optogenetics is a bioengineering technology that allows for precisely timed control of brain cells using light. The technology involves adapting genes of light-responsive proteins found in microbial species such as algae to uses in animal and human tissues. Recently, the U.S. Food and Drug Administration has authorized clinical trials for the first-ever human use of optogenetic gene therapy, for treatment of a form of blindness. New biotechnology startups have sprung up with plans to bring optogenetics to the U.S. health care market. Wide-ranging clinical applications, from unique therapies for neurological and psychiatric disorders to new solutions for cardiac problems, make this technology an innovative player in the next generation of precision medicine.

This paper begins with a review of the latest scientific developments in optogenetics, followed by a discussion of the safety and effectiveness issues in clinical optogenetic applications and the complexities of the existing FDA regulatory pathways for approval of optogenetic therapy. The paper concludes by addressing larger social and policy questions surrounding the future use of optogenetics for human-enhancement purposes. Optogenetics is a potentially revolutionary medical technology and the current regulatory and policy landscape may determine its ultimate reach.

Read the full research paper here.