Scientists are now able to create cardiac heart muscle cells from patients with heart disease. But cells alone aren’t enough to fully study cardiac disorders — especially rhythm disorders that require the activity of multiple cells assembled into tissues.
William Pu, MD, of Boston Children’s Hospital’s Heart Center and his team are honing the art of modeling heart disease in a dish. With an accurate lab model, they hope to test drug therapies without posing a risk to living patients (or even live animals).
Together with researchers at Harvard’s Wyss Institute, Pu’s lab recently modeled a rare rhythm disorder called catecholaminergic polymorphic ventricular tachycardia (CPVT). CPVT is a dangerous disease in which the heart’s rhythm can suddenly jolt abnormally without warning. Undetectable on a resting electrocardiogram (EKG), CPVT does not affect patients at rest. However, exercise or emotional upset trigger high levels of adrenaline, which can lead to life-threatening arrhythmia, cardiac arrest and possibly sudden death. …
From a series on researchers and innovators at Boston Children’s Hospital.
With all of the recent buzz about precision medicine, it’s no wonder that William Pu, MD is gaining recognition for his innovative application of stem cell science and gene therapy to study Barth syndrome, a type of heart disease that severely weakens heart muscle. Pu’s research was recently recognized by the American Heart Association as one of the top ten cardiovascular disease research advances of 2014.
Can you describe your work and its potential impact on patient care?
We modeled a form of heart-muscle disease in a dish. To do this, we converted skin cells from patients with a genetic heart muscle disease into stem cells, which we then instructed to turned into cardiomyocytes (heart-muscle cells) that have the genetic defect. We then worked closely with bioengineers to fashion the cells into contracting tissues, a “heart-on-a-chip.”
How was the idea that sparked this innovation born?
This innovation combined the fantastic, ground-breaking advances from many other scientists. It is always best to stand on the shoulders of giants. …