Cardiovascular Regenerative Team
Heart failure affects 5 million patients in the U.S., representing the most common cause of hospital admission and resulting in 300,000 deaths annually. Despite aggressive treatment with advanced pharmacotherapies and implantable devices, the 5-year survival is only 50%. Cardiac transplantation is limited to 2,000 patients per year due to the lack of suitable donors. Therefore, a strong mandate exists for novel strategies to treat patients with end-stage heart failure. Recent investigations support cell therapy as a rational strategy to restore and regenerate the injured myocardium. However, clinical studies suggest that adult stem cell therapy provides only limited efficacy. The Stanford Cardiovascular program has a distinguished history that includes several firsts in transplantation and implantation: the first human heart and heart-lung transplants in the US and first human endovascular stent-graft for aortic aneurysm. The Stanford Cardiovascular Regenerative Team (SCRT) follows this tradition of translational research to focus on efficient, integrated collaboration across multiple disciplines within Stanford University and to accelerate clinical implementation of innovative ideas by fostering partnership between academia and industry. For the CIRM Disease Team grant, the SCRT proposes to directly test the utility of human embryonic stem cell (hESC)-based therapy for significant and sustained functional restoration of injured myocardium. Our approach is grounded in fundamental investigations performed by the team members. We propose a collaborative strategy based on novel discoveries in our laboratories that will culminate in the first Phase I safety trial of transplantation of hESC-derived cardiovascular stem cells for patients with end-stage heart failure. The specific aims are:
Derive clinical-grade hESCs using good manufacturing practice (GMP).
Differentiate, purify and characterize mesodermal cells for cardiovascular regeneration.
Deliver and monitor hESCs in large animal model.
Transplant GMP-hESC derived progenitor cells into the failing heart of patients awaiting heart transplantation.
The planning effort of SCRT will merge innovative approaches in human stem cell biology with the highest standards in clinical safety to address a fundamental yet very critical issue in translational research: safety of GMP-hESC derived progenitor cells in the patients with failing heart.
Disease Team Planning