Researchers Working to Advance ExRNA Biomarkers and Therapies

Cells in the human body release a type of signaling molecule called extracellular RNA (exRNA) that can travel through body fluids to communicate information to distant cells. NIH-funded researchers are exploring the use of exRNAs as biomarkers, or indicators of the presence, absence or stage of a disease. In addition, researchers hope to use exRNAs to develop molecular treatments for many diseases.

NCATS has announced it will spearhead the second phase of several NIH ExRNA Communication program projects to test and validate exRNA molecules for their potential as disease biomarkers and treatments. The first phase of these NCATS projects focused on discovery and feasibility. The NCATS projects are funded by the NIH Common Fund.

“Although still in its early days, exRNA communication is an example of a game-changing discovery that could revolutionize the field of translational science,” said NCATS Director Christopher P. Austin, M.D.

ExRNA molecules have many biological functions and may play an important role in a wide range of diseases, including different types of cancer and neurological disorders, among others.

Researchers are using exRNA biomarkers in some of the projects to diagnose a variety of conditions and to predict their progression in patients. Others are exploring new ways to use exRNAs as clinical therapeutic treatments for illnesses. For example, therapeutic exRNA molecules could be delivered inside exosomes ― tiny particles produced by cells that can contain and transport exRNA ― to targets for many diseases. Specific focus areas include:

  • Alzheimer’s disease;
  • Multiple sclerosis;
  • Kidney disease;
  • Brain injury;
  • Pregnancy complications;
  • Heart disease, heart attack and stroke; and
  • Liver, stomach and brain cancers.

“During the first two years of the program, we confirmed that exRNA is not just a bystander molecule, but that it exerts important effects in the body,” said Danilo A. Tagle, Ph.D., M.S., associate director for special initiatives at NCATS. “We hope to use exRNA biomarkers and therapies to potentially address a wide range of diseases and improve the health and well-being of patients.”

The funded exRNA researchers disseminate data and resources via the exRNA Research Portal to keep the scientific community informed of the most recent developments in the field. In addition to NCATS, the NIH team leading the exRNA communication effort includes the National Cancer Institute; National Heart, Lung, and Blood Institute; and National Institute on Drug Abuse.

Learn more about NCATS’ participation in the ExRNA Communication program. The NIH Common Fund provides more information about the exRNA consortium.


Posted September 2015