Longeveron Gains FDA Approval to Begin Phase 2 Stem Cell Trial for Children with Heart Disease
Longeveron will evaluate if it's stem cell candidate, laromestrocel, could target pediatric dilated cardiomyopathy
Laromestrocel is an allogeneic (donor-derived) cell therapy derived from mesenchymal stem cells (MSCs). It is designed to potentially exert anti-inflammatory, pro-vascular, and regenerative effects in cardiovascular tissue.
Pediatric dilated cardiomyopathy is a severe progressive condition characterized by enlargement or dilation of one or more heart chambers, weakening the heart’s ability to pump blood effectively. It is the most common form of cardiomyopathy in children, accounting for 50-60% of pediatric cardiomyopathy cases. Approximately 100,000 children worldwide are affected by pediatric cardiomyopathies, and nearly 40% of children diagnosed with DCM either die or require a heart transplant within two years of diagnosis. Currently, effective treatment options for this condition are limited.
Barry Byrne, M.D., Ph.D., Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida, stated: “Current treatment for DCM focuses on managing symptoms, improving heart function, and preventing complications rather than addressing the underlying cause or causes. Many therapeutic agents with known efficacy in adults lack the same evidence in children. Longeveron’s innovative stem cell therapy approach, with the possibility for stem cells to repair damaged heart tissue, is a potential groundbreaking development in the treatment of children with cardiovascular diseases.”
In most cases, the exact cause of DCM remains unknown (idiopathic cardiomyopathy). It is reported to be more common in boys and particularly prevalent among infants under one year old.
Longeveron is currently pursuing laromestrocel clinical development in multiple indications, including hypoplastic left heart syndrome (HLHS), Alzheimer’s disease, and aging-related frailty. The therapy has received multiple FDA designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease designations for HLHS, and Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations for Alzheimer’s disease.
