$63 million NIH grant helps UF, national consortium explore cell regeneration therapies for heart disease

April 2, 2012

GAINESVILLE, Fla. — University of Florida researchers and colleagues at six other institutions have received a $63 million, seven-year grant from the NIH National Heart, Lung and Blood Institute to develop heart disease therapies that use a patient’s own bone marrow and heart cells to generate new healthy heart cells and restore function.

“The work has the potential to change the paradigm from the management of patients with heart disease, which right now is aimed at prevention and slowing progression,” said UF principal investigator Dr. Carl J. Pepine, a professor and eminent scholar emeritus of cardiovascular medicine. “This has the ability to move treatment into the regenerative medicine field.”

Heart disease, also called cardiovascular disease, is the leading cause of death in the U.S., killing more than 600,000 men and women a year, according to the Centers for Disease Control and Prevention. More than 27 million Americans are estimated to be living with heart disease, which encompasses a range of abnormalities of the heart and blood vessels such as heart failure, narrowed arteries, irregular heartbeat and heart defects present from birth. Those conditions can, in turn, lead to chest pain, heart attack and stroke.

The new grant is to the Cardiovascular Cell Therapy Research Network, or CCTRN, a national clinical trial consortium that, in addition to UF, includes the University of Miami, Indiana University, Stanford University, Texas Heart Institute, the University of Louisville and the Minneapolis Heart Institute. Originally funded in 2007, the network received the first federal funding for cooperative studies of so-called adult stem cells, in which patients are treated with cells taken from their own bodies. The UF-led team includes satellite study sites at UF College of Medicine-Jacksonville, Orlando Health and Pepin Heart Hospital in Tampa, Fla.

The network’s large size allows recruitment of hundreds of patients over relatively short periods for important clinical trials. Member researchers are specialists in a wide range of fields, including stem cell processing, bone marrow transplantation, interventional cardiology, MRI, EKG, cardiac anesthesia, pharmacology and gene and cell therapy.

“Stem cell therapy holds great promise for treating heart disease, and researchers involved in CCTRN are helping determine how these promising therapies might be most beneficial to patients,” said Sonia Skarlatos, deputy director of the division of cardiovascular sciences in the National Heart, Lung and Blood Institute. “This new round of funding is an important step in helping to improve cardiovascular health.”

The award, of which UF’s portion totals more than $5 million, allows the researchers to build on findings from rigorous randomized clinical trials funded by an earlier five-year grant to the network. The researchers previously found that in patients who had heart failure and/or chest pain, called angina pectoris, but were not eligible for standard surgical treatment to improve blood flow, delivering two types of stem cells from the patient’s own bone marrow resulted in a small but notable improvement in the heart’s ability to pump oxygen-rich blood around the body.

In other studies, the researchers sought to determine the optimal time for delivering therapeutic stem cells to patients after a heart attack. They administered the therapy in the first and third week after a heart attack, respectively. Giving the therapy in the third week yielded no benefit, and conclusions from the first-week study are pending.

This pioneering work in adult stem cell therapy for cardiovascular disease has laid the groundwork and set the standards for translating insights from basic stem cell biology research into clinical application.

With the new funding, the researchers will work to identify new kinds of stem cells that can be used for therapy. They will explore patients’ bone marrow and hearts to find cells ultimately capable of becoming new heart cells. The research will also extend to new categories of patients, including those who have a condition called peripheral vascular disease, in which narrowed arteries limit blood flow to the limbs. The effectiveness of stem cell therapy will also be evaluated in patients with heart failure or weak hearts and who have implanted mechanical pumps called left ventricular assist devices that help send blood around the body.

“What we are doing today is going to change cardiovascular medicine 10 years from now,” said Stanford University principal investigator Dr. John Cooke. “We are going to have better therapies for heart failure, myocardial ischemia and arrythmia. We’re going to have regenerative therapies for vascular disease and myocardial disease. These new therapies will have the ability to extend the quantity and quality of human life and lift the burden of cardiovascular disease from humanity.”