LOS ANGELES — The U.S. Food and Drug Administration (FDA) approved AD-NP1, a monoclonal antibody developed at the University of California Los Angeles (UCLA), for Phase 1 clinical trials in humans. AD-NP1 was developed to repair heart tissue following a heart attack, and Phase 1 trials evaluate the safety, dosing, and metabolism of investigational drugs.

AD-NP1 functions by blocking the ENPP1 protein, an entity that disrupts healing and prevents tissue recovery in internal organs. The monoclonal antibody was engineered to bind specifically to human ENPP1 and mimic the function of natural immune system antibodies. Injured kidneys produce the ENPP1 protein, which initiates a metabolic chain that disrupts cellular energy production and impairs tissue repair.

Research published in the journal Cell Stem Cell showed that blocking ENPP1 accelerates kidney tissue repair following injury in mice. Kidney biopsies from individuals with chronic kidney disease showed higher ENPP1 expression than biopsies from healthy tissue. Researchers administered kidney-damaging drugs to mice with ENPP1 gene knockouts and to normal control mice, with mice having induced kidney damage showing increased blood levels of serum creatinine, blood urea nitrogen, and cystatin C. These elevated levels indicate renal dysfunction.

After four weeks, mice lacking the ENPP1 gene exhibited lower levels of serum creatinine, blood urea nitrogen, and cystatin C than control mice. Researchers also administered AD-NP1 to normal mice with induced kidney damage. Seven days after receiving AD-NP1, normal mice showed improved kidney function metrics. Tissue inspection of mice treated with AD-NP1 showed reduced scarring and increased proliferation of kidney cells.

Arjun Deb, a professor of medicine and molecular, cell and developmental biology at UCLA, stated that the mechanisms observed in the heart were also applicable in the kidney. Deb said after injury, healthy cells around the damaged area attempted to proliferate, but the damaged area sent metabolic signals that prevented effective regeneration and repair. Deb directs a cardiovascular research laboratory at UCLA and is a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research. Researchers plan to submit applications for clinical trials evaluating AD-NP1 for kidney conditions. The development of AD-NP1 and the related research received funding from the National Institutes of Health, the California Institute of Regenerative Medicine, and the Department of Defense.

No independent assessment was available for this report.