UCLA’s investigational drug AD-NP1, designed to enhance tissue repair in the heart and other organs, has received clearance from the U.S. Food and Drug Administration (FDA) for human clinical trials. The drug was developed entirely within UCLA by a team led by Arjun Deb, professor of medicine and molecular, cell and developmental biology.
The research began when Deb’s team observed that after a heart attack, both mice and human heart tissue samples showed increased levels of the protein ENPP1. According to their findings, elevated ENPP1 disrupts energy production in cells at the injury site, which impedes healing. By blocking ENPP1 with a monoclonal antibody called AD-NP1, they saw improved tissue repair and reduced scar formation in animal models.
Deb emphasized the unique funding model behind this project: “This work has been entirely funded by taxpayer dollars, and done entirely within the University of California research ecosystem,” he said. “I have not taken a cent from any private donor or company to develop this drug. I hope this will form a model for future drug development at UCLA. This process has advantages of lower costs, potentially shorter development time and the principal investigator being in control of the science and having intellectual freedom with the development of the molecule, which is the most important of all.”
Unlike many academic discoveries that are licensed to biotech companies or spun off into startups for further development, AD-NP1 was developed exclusively at UCLA over seven years using grants from federal agencies such as the National Institutes of Health and Department of Defense as well as support from the California Institute for Regenerative Medicine.
Monoclonal antibodies like AD-NP1 are engineered to mimic natural immune system proteins but target specific molecules—in this case, only human ENPP1. Deb explained that interfering with ENPP1 helped maintain energy generation in heart muscle cells after injury: “That is what we saw: increased ENPP1 expression interfered with critical pathways that are needed for a cell to derive energy,” he said. “When AD-NP1 was used in animals, the heart muscle had more energy and contracted much more vigorously, preventing the development of heart failure.”
Deb also noted that because metabolic pathways involved in cellular energy production are common across different tissues, AD-NP1 could potentially aid recovery in other organs beyond just cardiac tissue.
His approach focuses on optimizing natural repair mechanisms rather than using stem cells: “Rather, you use the power of the body’s own repair system and optimize it to make it so much better,” Deb said.
Looking ahead to clinical trials in humans, Deb hopes positive results will pave the way for new therapies targeting organ damage following acute injuries or disease. He stated: “Cardiovascular disease is still the leading cause of death in the U.S. and around the world,” adding that rapid progress made at an academic institution demonstrates how public funding can drive innovation: “It’s a testament to the funding system we have in place in this country that within six or seven years, in an academic lab in a university setting, we have engineered a new drug that potentially could be helpful to many people with heart disease or other forms of organ injury.”
