The U.S. Food and Drug Administration (FDA) has cleared a new drug, AD-NP1, developed by UCLA researchers, for clinical trials in humans. The drug is designed to enhance tissue repair in the heart and potentially other organs after injury.
Arjun Deb, a professor at UCLA specializing in cardiovascular research, led the team that discovered elevated levels of the protein ENPP1 in heart tissue samples from both mice and humans following a heart attack. According to Deb, “This work has been entirely funded by taxpayer dollars, and done entirely within the University of California research ecosystem. 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.”
The researchers found that higher ENPP1 levels disrupt cellular energy production, hampering tissue repair after organ injury. By blocking ENPP1 with their engineered monoclonal antibody AD-NP1, they observed improved heart function and reduced scar tissue formation in animal studies.
Deb’s approach does not use stem cells but instead targets metabolic pathways to enhance the body’s natural repair mechanisms. “Rather, you use the power of the body’s own repair system and optimize it to make it so much better,” said Deb.
Monoclonal antibodies like AD-NP1 are designed in laboratories to mimic natural antibodies produced by the immune system. In this case, AD-NP1 was specifically engineered to target only human ENPP1.
“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,” said Deb.
He added that when AD-NP1 was used in animals recovering from cardiac injury, “the heart muscle had more energy and contracted much more vigorously, preventing the development of heart failure.”
Deb believes that because many organs share similar energy-generating pathways at the cellular level, AD-NP1 could eventually be used beyond cardiac treatment for other organ injuries as well.
“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.”
Clinical trials will determine whether these promising results seen in animal models can be replicated safely and effectively in humans.
