The Facial Pain Research Foundation Announces New Gene Therapy Project

By Michael Pasternak

The Trustees of The Facial Pain Research Foundation (FPRF) have announced a Sixth Foundation funded research project to find the cures for trigeminal neuralgia and related neuropathic pain. To our knowledge it is the first scientific project using this gene therapy approach as a cure for trigeminal neuralgia and related neuropathic pain.  It continues the Foundation’s commitment to establish a well funded translational research continuum that is dedicated to identifying the mechanisms underlying neuropathic facial pain and to developing novel therapeutic strategies that will permanently stop the pain of TN and related neuropathic pain syndromes.

The FPRF has committed $250,000 to fund the two year research project “Evaluation of adeno-associated virus (AAV) constructs directed at pain pathway targets-a rapid approach for identification of effective therapies for trigeminal neuralgia”. The project will be located at the McKnight Brain Institute, University of Florida.  The Principal Investigators are Todd E. Golde, Ph.D., M.D. (Therapeutics) and John K. Neubert, D.D.S., Ph.D. (Behavioral Pain Testing).  Other Significant Contributors are Yona Levites, Ph.D. (Therapeutics) and Robert M. Caudle (Behavioral Pain Testing).

Dr. Golde is Director of the McKnight Brain Institute as well as the Director of the Center for Translational Research in Neurodegenerative Disease at UF.

Dr. Neubert at UF is the Principal Investigator of the FPRF project “In Search of a Cure: Finding the Brain Signature Centers that Cause Trigeminal Neuralgia”.

In summarizing this research, Dr. Golde states “This pain is known to be one of the worst pain conditions that a patient can suffer and has been called the “suicide disease”. Given the severity of this disorder, figuring out the cause becomes essential for finding the cure.  This project will investigate novel viral-based delivery therapeutics targeting specific targets within the pain processing network. We hypothesize that advances in specific gene-targeting vectors will more quickly identify and yield feasible therapies for the treatment of TN.  We have expertise in designing and testing such therapies using the animal models, providing a requisite step before considering use in patients.  This translational approach is important because a rapid screening approach is needed in order to identify candidate therapies in search of the cure for TN…The rationale for the proposed research is that improved and speedy identification of novel effective pain relievers will provide candidate therapeutics for treating TN patients in the next few years.”

Dr. Douglas Anderson, FPRF Director of Research and Trustee, stated that Dr. Golde’s project is innovative and well crafted.  He proposes to use Adeno Associated Virus, a state of the art viral vector transport system to deliver candidate genes that will either enhance or inhibit or replace malfunctioning genes.  Another big plus with Golde’s program is that he and his team already have significant expertise in designing and testing viral-based delivery systems in established animal pain models. Thus, in sum, the focus of this project is to design and synthesize a number of viral constructs directed at specific genes related to pain processing and then determine which of these constructs are the most efficacious in doing so initially in animals.”