“This is a very important advance in the field of cell therapy and in the management of heart failure. It suggests that a treatment, given only once, can produce long-term beneficial effects on the quality of life and prognosis of these patients,” said Roberto Bolli, director of the UofL Institute of Molecular Cardiology, who led the study at UofL. “The results pave the way for a larger, Phase 3 trial of cell therapy in heart failure.”

UofL led enrollment among seven institutions participating in CONCERT-HF, a Phase 2 clinical trial testing the safety, feasibility and efficacy of two types of adult cells, used alone and in combination, in patients with heart failure.

CONCERT-HF evaluated the use of two types of cells – autologous mesenchymal stromal cells (MSCs) and c-kit positive cardiac cells (CPCs) – alone or in combination, in patients with heart failure caused by chronic ischemic cardiomyopathy, a decrease in heart pumping effectiveness due to heart attacks and a lack of blood getting to the heart. Autologous MSCs are derived from the patient’s bone marrow and CPCs are from the patient’s heart tissue. Both are known as “autologous” cells because they come from the same patient in whom they are returned for the treatment.

In the study, patients treated with CPC cells alone had a significant reduction in major adverse cardiac events, particularly hospitalization. Patients treated with MSC cells alone and with a combination of both types of cells experienced significantly improved quality of life compared with patients who received no treatment. Quality of life was assessed using patient responses to the Minnesota Living with Heart Failure Questionnaire, which gauges the degree to which physical, emotional and socioeconomic effects of heart failure adversely affect the patient’s life and the extent to which they prevented the patient from living as they wanted to live.

“The results of this trial show for the first time that cell therapy reduces hospitalization for heart failure and improves clinical outcome, providing a cogent rationale for undertaking a pivotal Phase 3 trial” that could be the next step on the pathway to FDA approval, Bolli said.

The results of the were published in the . The Phase 2, randomized, placebo-controlled trial, funded by NIH National Heart, Lung and Blood Institute, was conducted by the Cardiovascular Cell Therapy Research Network, a network of clinical trial researchers involved in cell therapy for heart disease that includes UofL. UofL led enrollment in the study, accounting for 25% of the 125 trial participants.

Bolli’s expertise and long career in successful cardiac research led to the establishment of the CCTRN center at UofL in 2011. This consortium of leading cardiovascular research organizations includes Stanford University, the University of Miami, Indiana University, the Texas Heart Institute, the University of Florida and the Minneapolis Heart Institute Foundation, in addition to UofL. The School of Public Health at the University of Texas Health Science Center at Houston serves as the data coordinating center.

Bolli is a pioneer in research using adult stem cells for cardiac disease. Over the past two decades, he has shown that CPCs are beneficial in many preclinical models of heart failure, thus paving the way for the CONCERT-HF trial. He also led the recent – the first study to show safety and potential efficacy of cell therapy in cancer survivors with heart failure caused by anticancer therapy.

The summit, a high-profile event within the meeting of approximately 25,000 clinicians and researchers from around the world, brings together top international experts who will present the latest and most exciting work in stem cells, cell therapy and cardiac regeneration.

The association meeting is Nov. 11-15; the summit will be held on Nov. 14.

Bolli, director of UofL’s Institute of Molecular Cardiology and scientific director of the Cardiovascular Innovation Institute at UofL, was asked by the association to organize the event this year. He is well-known in the field of stem cell research, and last month he and his team at UofL received a $13.8 million award from the National Institutes of Health to study a promising new type of adult cardiac stem cell that has the potential to treat heart failure.

Bolli also serves as editor of Circulation Research, an official journal of the American Heart Association that is considered the world’s leading journal on basic and translational research in cardiovascular medicine.

He will give the opening introduction, as well as an overview before each part of the summit, which will have two sessions in the morning and two in the afternoon.

“I’m honored to be selected to lead the summit this year,” Bolli said. “It reaffirms UofL’s leadership in the field of cell therapy for cardiovascular disease. This will be an outstanding program and I expect it to be well-received.”

The announcement on Friday was made by Gregory Postel, MD, interim president of UofL, and the study’s principal investigator, Roberto Bolli, MD, director of UofL’s Institute of Molecular Cardiology. Bolli also serves as scientific director of the Cardiovascular Innovation Institute at UofL and as a professor and chief of the Division of Cardiovascular Medicine at the School of Medicine.

“This is a prestigious grant reflecting the magnitude of the work being conducted here,” Postel said. “Being awarded this grant is a huge, huge accomplishment.”

Bolli thanked the National Heart, Lung and Blood Institute and the NIH for their support. “It is critical that we have this type of support for the important research programs that we carry out, which can help patients around the world,” he said.

Heart failure affects millions of people, and the most common cause is a heart attack. When a person suffers a heart attack, part of the heart muscle dies from lack of oxygen and is replaced with scar tissue, which does not contract. Because of the loss of muscle, the heart becomes weaker and less able to pump.

Until now, conventional treatments for heart failure have consisted of surgery or medications, which can alleviate symptoms but do not cure the disease. In contrast, Bolli’s focus has been on how to repair the heart itself and actually cure heart failure using a patient’s own stem cells. It is an approach that could revolutionize the treatment of heart disease.

The NIH grant is a continuation of a Program Project Grant (PPG) that Bolli and his team were originally awarded in 2005. The overall goal of this PPG is the use of stem cells to repair the damage caused by a heart attack by regenerating heart muscle in the area that died, replacing the scar tissue with new muscle and thereby making the heart stronger and able to pump more blood.

A PPG is a cluster of several projects with a common focus relating to one theme, in this case, the use of adult stem cells to repair the heart. It involves a collaboration among different investigators working as a team, a collaboration that otherwise might not be able to occur without funding.

The latest round of funding comes after Bolli and his colleagues discovered a new population of adult stem cells, called CMCs, in the heart three years ago.

“CMCs seem to be more effective,” Bolli said. “In addition to showing more promise than those we have used in the past, these cells also offer several advantages in that they can be produced more easily, faster, more consistently and in larger numbers than other adult stem cells, which have proven tricky.”

He said this would make them easier to apply for widespread use, as specialized labs to isolate the cells would not be needed as with other types of adult stem cells.

Bolli and his team want to find out what CMCs will do when transplanted into a diseased heart in mice and pigs, ultimately laying the groundwork for clinical trials in patients.

On Friday, Postel noted that the NIH didn’t just approve UofL’s grant application – a long, multistep process involving more than a dozen reviewers who are experts in the field – it funded the project with a perfect score and rare high praise. In fact, the committee reviewing the application concluded Bolli’s program was, “exceptional,” with “significant translational impact, an exceptional leader and investigative team and an exceptional environment.”

“We are continually striving for new and better ways to treat heart disease,” Bolli said. “I’m confident we are not that far from a cure.”

Watch the announcement below: