UCLA researchers have received clearance from the U.S. Food and Drug Administration to begin clinical trials of a new drug, AD-NP1, aimed at regenerating heart tissue after injury. The drug was developed by Arjun Deb, a professor at UCLA and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research.
Deb’s research identified that after a heart attack, levels of the protein ENPP1 increase in heart tissue, which disrupts energy production in cells and impedes repair. By blocking ENPP1 with the monoclonal antibody AD-NP1, his team found improved tissue repair and reduced scar formation in animal studies.
The project was funded entirely by public sources, including the National Institutes of Health, Department of Defense, and California Institute for Regenerative Medicine. No private companies or investors were involved in its development.
“This work has been entirely funded by taxpayer dollars, and done entirely within the University of California research ecosystem,” said Deb. “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.”
Monoclonal antibodies like AD-NP1 are engineered to target specific proteins—in this case, human ENPP1—without affecting other proteins. According to Deb: “Much like people eat food to get energy, cells also require energy to multiply and grow and function, and this is more critical when the tissue is injured.”
He added: “That is what we saw: increased ENPP1 expression interfered with critical pathways that are needed for a cell to derive energy,” noting that animal tests showed stronger heart muscle contraction when treated with AD-NP1.
Deb’s approach does not use stem cells but instead aims to optimize natural repair mechanisms within tissues. He believes AD-NP1 could also benefit other organs following acute injury due to shared cellular energy pathways.
If clinical trials confirm its effectiveness in humans as seen in animals, AD-NP1 could lead to new therapies for preventing organ function decline after injury.
“Cardiovascular disease is still the leading cause of death in the U.S. and around the world,” said Deb. “All Americans want to lead healthier and longer disease-free lives. 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.”
