Breakthrough Drug Shows Promise for Kidney Repair

A drug originally developed to heal heart tissue after a heart attack may also hold the key to repairing damaged kidneys, according to new research from UCLA.

Researchers at UCLA have found that a drug called AD-NP1, initially designed to repair heart tissue after a heart attack, may also aid kidney tissue repair and regeneration. The drug works by blocking the protein ENPP1, which disrupts healing and prevents full recovery of internal organs. In experiments with mice suffering kidney injury, blocking ENPP1 led to improved kidney function and reduced scarring within just seven days of treatment.

The Science Behind the Breakthrough

The study, published in Cell Stem Cell, builds on prior work by UCLA cardiovascular scientist Arjun Deb. Previously, Deb's team discovered that injured kidney tissue produces ENPP1, initiating a metabolic chain that impairs energy production and cell function, hindering repair. Blocking ENPP1 in heart tissue had earlier been shown to improve healing, suggesting a common mechanism across organs.

Evidence from Human and Animal Studies

Kidney biopsies from people with chronic kidney disease showed higher ENPP1 expression compared to healthy tissue. Experiments with mice involved a toxic diet and drugs that cause kidney damage. Mice unable to produce ENPP1 showed significantly lower levels of serum creatinine, BUN, and cystatin C—key markers of kidney dysfunction—after four weeks, compared to control mice.

Following kidney damage, normal mice given AD-NP1 showed improved kidney function and reduced scarring after seven days.

How the Drug Works

The drug AD-NP1 is a monoclonal antibody engineered to target human ENPP1, mimicking the function of natural antibodies. It was developed entirely at UCLA with public funding.

Path to Clinical Use

AD-NP1 was approved by the FDA for a Phase 1 clinical trial in humans for heart conditions in September. Phase 1 trials assess safety, dosing, and metabolism. Deb plans to apply for trials in the kidneys as well.

Funding

The research was funded by the National Institutes of Health, the California Institute of Regenerative Medicine, and the Department of Defense.