Funded entirely by taxpayer dollars, this first-of-its-kind drug targets cellular metabolism to promote tissue repair of multiple organs
By Holly Ober | Oct 06, 2025 Clinical Trials Research
Key Takeaways
https://stemcell.ucla.edu/news/fda-clea ... cal-trials
Internal organ tissues often don’t heal after illness or injury and lose some function, such as the heart after a heart attack. UCLA cardiologists have identified a protein that interferes with healing.
Funded entirely by federal and state grants, the researchers developed a drug to block this protein and promote tissue regeneration.
The FDA has now granted approval to begin Phase I clinical trials of the first-in-class drug for tissue repair, called AD-NP1, in humans.
The body’s tissues can get injured in many ways, but while some injuries heal perfectly, others don’t heal at all. A cut in the skin, for example, usually heals all on its own, while internal organs, such as the heart after a heart attack or the kidney after an acute injury, remain damaged, leading to diminished function. Most tissues of the body repair themselves using the same processes, but until now, drugs that target these pathways to enhance tissue repair in slow-healing organs have not been identified. That’s about to change.
UCLA cardiovascular scientist Arjun Deb discovered that heart tissue samples taken from mice and humans after a heart attack had increased levels of a protein called ENPP1. Deb and his scientific team observed that increased levels of ENPP1 initiated a metabolic chain of events that disrupted energy generation and function of multiple cells in the injured region of the organ, impeding tissue repair. The researchers found that blocking the production of ENPP1 enhanced heart repair and reduced the formation of scar tissue, thereby improving heart function.