Cell Based Therapy for Congenital Cardiomyopathy Using Endogenous Cardiac Stem Cells

Doctor's Name: 
Sunjay Kaushal, M.D., PhD
Hospital/Institution: 
Children’s Memorial Hospital, Chicago, IL

Even though surgical repair of congenital heart disease patients has been highly successful, in many of these patients loss of cardiac function occurs, which ultimately may lead to heart transplantation or death. Currently, the treatment options are limited, with no good long term solutions. Cell based therapy is now a plausible intervention to recover heart function, as seen in the clinical trials of adult ischemic heart disease patients. To date, no cell based therapies have been applied to children with lethal heart function loss.

Recent evidence from the laboratory at Children’s Memorial Hospital and others have identified a population of cells within the heart itself, called cardiac stem cells, which may also be a unique cell source to stimulate heart muscle cell growth ( regeneration ). Cardiac stem cells have the potential to be implanted in the same individual that they were harvested from and improve that individuals cardiac function. This research proposes to determine whether cardiac stem cells can improve cardiac function in a drug induced ( doxorubicin ) cardiomyopathy model in mice that replicates cardiomyopathy  seen in congenital heart patients. Specifically, after the drug induced heart loss occurs in mice, the researchers will inject the cardiac stem cells directly into the heart and then look for improvement of cardiac function by various studies. This project will be an important first step in generating pre-clinical laboratory data to determine whether this can be a promising new therapy to reduce pediatric heart failure and improve life expectancy for these clinically challenged cardiac patients.

Award Date 1: 
2008
Award Amount 1: 
$100,000
Award Date 2: 
2009
Award Amount 2: 
$100,000