At the California Institute for Regenerative Medicine (CIRM), we support cardiovascular research from Discovery through Clinical to advance treatments for heart failure and other forms of heart disease — the world’s leading cause of death.
New Study to Prevent heart Failure
CIRM recently awarded $6 million to Arjun Deb, MD, and his team at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. Their work aims to prevent heart failure after heart attacks. In preclinical studies, their therapy reduced scar formation without increasing the risk of heart rupture — a major step forward in post–heart attack care.
Heart disease causes one‑third of deaths worldwide. After a heart attack, the heart forms scar tissue to maintain structure, but this inflexible tissue weakens the heart’s ability to pump blood. As a result, many patients develop heart failure, and half do not survive beyond five years. New therapies are urgently needed.
The UCLA team— led by Deb who is also a professor of medicine and molecular, cell and developmental biology— targets a protein called ENPP1, which drives inflammation and scar formation after a heart attack. Their experimental monoclonal antibody blocks ENPP1 and is designed to enhance the heart’s natural repair mechanisms. Findings published in Cell Reports Medicine show that a single dose improved heart repair in mice, reduced tissue damage, and limited the development of heart failure. Only 5% of treated mice developed severe heart failure, compared with 52% of the control group.
Tissue Repair
This approach could become the first therapy to directly promote tissue repair after a heart attack, rather than simply prevent further damage. By influencing cellular cross‑talk, the antibody benefits multiple cell types involved in healing, including muscle cells, endothelial cells, and fibroblasts.
Early data show that the therapy reduces scarring without raising the risk of heart rupture, though more work is needed to understand long‑term effects of ENPP1 inhibition, including potential impacts on bone.
Deb’s team plans to submit an Investigational New Drug (IND) application to the FDA this winter and begin first‑in‑human trials in early 2025. The initial study will test a single dose administered soon after a heart attack to boost repair during the critical early phase of healing.
The team is also exploring whether this therapy could help repair other organs, since many tissue‑repair mechanisms are shared across the body.
The therapeutic candidate is covered by a patent held by the Regents of the University of California. It has not yet been tested in humans or approved by the FDA.
Read the full press release by Ani Vahradyan on the UCLA website.