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NIH trial shows safety and feasibility of cell therapy for heart failure

A clinical trial shows that cell-based therapy can be safe and feasible for patients with chronic heart failure—patients who currently have limited treatment options. The researchers also reported lower rates of death and hospitalization as well as improvements in quality of life for some.

Presented November 13, 2020 at the American Heart Association annual meeting and published April 3, 2021 in the European Heart Journal, the findings report the outcomes of CONCERT-HF, a multicenter trial funded by the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health.

The CONCERT-HF trial sought to determine if c-kit+ cardiac progenitor cells derived from the heart, either alone or in combination with mesenchymal stromal cells derived from bone marrow, are safe and can improve cardiac function and clinical outcomes for patients with advanced heart failure.

Researchers recruited 125 patients with chronic heart failure and randomly assigned them to one of four groups. Then, they harvested the cells from the patients’ hearts and bone marrow, to manufacture the two different cell products that would be injected back into the patients’ diseased hearts.

Each group received c-kit+ cardiac progenitor cells, mesenchymal stromal cells, a combination of both cell products, or a placebo. All the study participants were followed up for 12 months after the procedure.

“This study demonstrated that it was feasible and safe to harvest the two different types of progenitor cells from patients, expand them in a rigorously controlled manufacturing facility, and administer them directly into the patients’ heart muscle,” said Ray F. Ebert, Ph.D., a coauthor and program officer at NHLBI’s Division of Cardiovascular Sciences.

Notably, patients treated with the c-kit+ cells alone had a significantly lower rate of major adverse cardiac events (death, hospitalization, and worsening heart failure) when compared with the placebo group. Researchers observed a similar but not statistically significant difference in the group that received the combination treatment.

Study participants who received bone marrow-derived cells, either alone or in combination with c-kit+ cells, reported significant improvements in their quality of life when compared with the placebo group.

“CONCERT-HF is the first randomized clinical trial to report a potential clinical benefit of c-kit+ cells in patients with chronic ischemic heart failure,” Ebert said.

Various physical measures of cardiac function such as ejection fraction or end diastolic volume were not significantly improved by any of the treatments, a finding that suggests that the beneficial effects resulted from the anti-inflammatory or other beneficial factors released by the cells, according to the researchers.

“This was an important trial looking to answer a question of life and death for patients with heart failure, about half of whom will die within five years of receiving a diagnosis, despite significant medical and surgical advances,” said David Goff, M.D., Ph.D., director of NHLBI’s Division of Cardiovascular Sciences. “Now comes the time to carefully analyze the findings to understand the impact and potential of c-kit+ cells to inform future research decisions. Rethinking the path of studies based on new findings is the essence of the scientific process.”

In October 2018, NHLBI paused the CONCERT-HF trial, out of an abundance of caution, after the retraction of journal articles in related fields of cell therapy raised concerns about the trial’s scientific foundations. The CONCERT-HF’s Data and Safety Monitoring Board (DSMB) conducted an independent reassessment of the data and recommended that the study continue. Visit the CONCERT-HF Study page for more information.

Bolli, R., et al. A Phase II Study of Autologous Mesenchymal Stromal Cells and c-kit Positive Cardiac 2 Cells, Alone or in Combination, in Patients with Ischemic Heart Failure: The CCTRN 3 CONCERT-HF Trial. European Heart Journal. DOI:  10.1002/ejhf.2178

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