A New Hope for Kids with a Rare Heart Condition

A big step has been taken in the fight against a serious heart condition in kids. A company is testing a new stem cell treatment called laromestrocel. This treatment is being studied to help children with hypoplastic left heart syndrome (HLHS), a rare and severe heart defect. The good news is that the company has finished enrolling all the kids needed for the trial. Results from this trial are expected by the third quarter of 2026. HLHS is a tough condition. Kids born with it have only one working heart ventricle. They need three open-heart surgeries to survive. Even with these surgeries, many kids don't make it to age 15 without a heart transplant. The new treatment, laromestrocel, is being tested to see if it can make things better for these kids. The trial is called ELPIS II. It follows a previous trial, ELPIS I, where kids who got the treatment had a 100% survival rate up to age five without needing a transplant. This is a big deal because, usually, about 20% of kids with HLHS don't make it that long. The trial is being done with help from the National Heart, Lung, and Blood Institute and grants from the National Institutes of Health. The U. S. Food and Drug Administration (FDA) has given the treatment some special statuses. These include Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation. This means the FDA thinks the treatment is important and worth fast-tracking. If the trial shows the treatment works, the company can apply for full approval. The company behind this treatment is called Longeveron. They make medicines that help the body heal itself. Laromestrocel is their main product. It's made from the bone marrow of young, healthy adults. The treatment has different ways of helping the body, like reducing inflammation and repairing tissues. Besides HLHS, the company is also testing the treatment for other conditions like Alzheimer's disease and aging-related frailty. The chief science officer of Longeveron, Dr. Joshua Hare, recently talked about this on National Public Radio. He discussed the seriousness of HLHS and the potential of the new treatment. The interview is available online for anyone who wants to learn more.