The Mussallem CHD Alliance is proud to support three research teams pursuing breakthroughs for people born with congenital heart defects.

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01 Imaging

Safer, more accessible heart imaging for the youngest patients, with scans that rely less on anesthesia.

MRI offers one of the most detailed and complete views of a human heart, but the majority of scanners in use today were designed for adults. MRI scanning for the smallest patients – infants and children – is no easy task.

For young patients who struggle to remain still, an MRI scan can take over an hour and frequently requires sedation, which adds risk, cost, and stress for families.

The Mussallem CHD Alliance is supporting the development of AI-powered technology by the Stanford Medicine Children’s Health pediatric radiology team, led by Shreyas Vasanawala MD/PhD. Vasanawala and his team are developing a low-cost, wearable insert that slides into any existing MRI machine and turns it into a high-performance pediatric imaging platform.

“If MRI turned into a quick walk-in procedure, it could be used much more frequently to speed up diagnoses,” Vasanawala says. “Making this powerful imaging technology more available would allow us to get answers for kids much more quickly.”

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02 Precision Medicine

Delivering a powerful heart medication exactly where it’s needed, without the systemic side-effects that limit it today.

Supraventricular tachycardia (SVT) is the most common heart-rhythm disorder in children and a particular danger for those born with CHD, whose hearts are less able to tolerate a sudden, sustained fast rhythm. The most effective medication for it is so toxic to the rest of the body that more than half of CHD patients can’t tolerate it.

Supported by the Mussallem CHD Alliance, Dr. William Goodyer and Dr. Henry Chubb, pediatric heart-rhythm specialists at at Stanford Medicine Children’s Health, are engineering a way to carry the medication directly to the upper chambers of the heart where the arrhythmia begins, sparing the rest of the body.

“This innovation could have enormous implications for children with CHD,” says Goodyer. “It could provide a game-changing alternative to how we treat rhythm disorders in kids.”

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03 Gene Therapy

A new CRISPR strategy for the genetic roots of CHD: one editor for thousands of patients, with groundwork that reaches beyond a single condition.

Around 40% of congenital heart defect cases involve genetic mutations. Because defects stem from hundreds of different types of mutations, building a custom gene-editing fix for each one is challenging.

With funding from the Mussallem CHD Alliance, Dr. Jesse Engreitz, an assistant professor of genetics at Stanford Medicine, is testing a solution: He is using CRISPR gene editing to develop a prototype therapy targeting a gene involved in one type of congenital heart disease. This approach is designed to rewrite DNA in heart cells and address the underlying genetic cause of disease.

“Our goal is to show first proof-of-concept that genetic surgery could mend broken hearts at the level of our DNA,” says Engreitz.

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