Discovery and Validation of Novel Targets for Safe and Effective Treatment of Pain
The Research Need
Pain is a major factor in many acute and chronic conditions, but only a small number of new therapeutics for pain advance from Phase 1 clinical trials to approval by the U.S. Food and Drug Administration. There is an urgent need for innovative approaches to find and validate new biological targets for treating pain.
About the Program
This program seeks to accelerate the scientific discovery and validation of treatment targets for acute and chronic pain conditions in order to reduce reliance on opioid medications and speed the development of effective pain medications that have little or no abuse liability. This research enables basic discovery of biological targets in the peripheral and central nervous systems, as well as in the immune and other body systems involved in detecting and transmitting painful signals across diseases. This research also explores understudied genes and proteins (the druggable genome and proteome) associated with pain, pain perception, and opioid use disorder that might serve as markers or targets for treatment.
The program conducts rigorous validation of identified pain targets to ensure they have minimal side effects and little to no abuse or addiction liability. Validation approaches include use of knockout animals or human tissue, as well as reproduction of experiments by an independent laboratory. Research in the program also identifies and validates small molecules and biologics such as antibodies and cell-based therapies that could lead to translational research to develop pain therapeutics.
Within this program, the Program to Reveal and Evaluate Cells-to-Gene Information that Specify Intricacies, Origins, and the Nature of Human Pain (PRECISION Human Pain) network will capitalize on recent technological advances to understand the cellular and molecular characteristics of human tissues involved in pain processing. This information will be used to validate human therapeutic targets using a variety of cellular assay systems and reproducibility testing in multiple laboratories. This cutting-edge research approach will enable future translational research and the development of non-addictive pain therapies for human pain conditions.
To date, through the Helping to End Addiction Long-term® Initiative, or NIH HEAL Initiative®, NIH has funded 47 awards for this program, totaling $90.3 million.