Heart Disease Regenerative Medicine Team Planning Award
The present planning grant application lays the groundwork for a collaborative heart disease regenerative medicine team. We plan to develop sequential preclinical and clinical investigations directed at regenerative stem cell-based approaches to treating the following major cardiovascular diseases: heart attack and its sequelae; congestive heart failure; and heart block requiring a pacemaker. The basic foundation necessary to justify focused preclinical studies has been achieved by members of the collaborative team and by investigators elsewhere. We will plan detailed milestone-driven studies aimed at direct clinical applications in the above-mentioned cardiovascular diseases. Autologous adult cardiac stem cells (CSCs) will be used to treat myocardial infarction as well as cardiomyopathy. Cell delivery will be via catheters like those used to perform angioplasties on diseased coronary arteries. This work needs minimal additional preclinical work in large animals in order to be ready for regulatory filings in anticipation of human studies. To treat heart block, we will develop human embryonic stem cell (hESC)-derived biological pacemakers as a clinical product. Work to date has demonstrated feasibility at the proof-of-concept level, but preclinical studies in large animals will be required in preparation for an IND filing. We request funding to plan and establish detailed protocols to accomplish these studies, which will utilize existing facilities, and will leverage the resources and expertise of the collaborative team. Anticipated team members include experts in stem cell biology, clinical cardiologists and surgeons, imaging physicians, and leaders in clinical trial design. The planning grant will be used to obtain appropriate consultative expertise, to support collaborative team meetings, and to carve out time for the PI to prepare the definitive proposal.
Ceders-Sinai Medical Center
Disease Team Planning