Initial data from the first ever trial to evaluate autologous cardiac stem cell (CSC) transplants in humans suggests that the treatment improves left ventricular (LV) systolic function by an average of 12% over one year, and reduces infarct size in patients with severe heart failure due to ischemic heart disease. The trial investigators say the results triple the 4% average improvement that they had projected and calls for the start of larger Phase II trials.
Stage A of the ongoing open-label Phase I SCIPIO (Stem Cell Infusion in Patients with Ischemic cardiOmyopathy) study, by investigators at the University of Louisville and Brigham and Women’s Hospital, is evaluating CSC transplantation in patients with severe heart failure secondary to ischemic cardiomyopathy. The target population includes patients who underwent coronary artery bypass grafting (CABG), had LV ejection fraction (EF) of less than or equal to 40%, and a previous myocardial infarction.
Treated patients were administered with about a million autologous CSCs by intracoronary infusion, at a mean of 113 days after CABG. To generate the cardiac stem cells, tissue from the right atrial appendage was harvested from the patients at the time of CABG, and CSCs were isolated and expanded at the Brigham and Women’s Hospital.
Data from 14 of 16 patients assigned to the treatment group, and seven from the control group (best supportive care), have now been published in The Lancet to coincide with data presentation at the American Heart Association’s Scientific Sessions meeting in Orlando, FL. The reported data showed that autologous CSC transplantation led to an increase in LVEF from 30.3% before CSC infusion to 38.5% at four months after infusion. In contrast, the LVEF of seven control patients didn’t change over eight months. The benefits of CSC transplantation was even more pronounced at one year in eight evaluated patients, for whom LVEF increased by 12.3 ejection fraction units compared with baseline. In the seven treated patients evaluated using MRI, infarct size was also shown to have decreased by 24% at 4 months, and 30% at one year.
The trial has been led by Roberto Bolli, M.D., at the University of Louisville and Piero Anversa, Ph.D., at Brigham and Women's Hospital/Harvard Medical School in Boston. "The results are striking," Dr. Bolli states. "While we do not yet know why the improvement occurs, we have no doubt now that ejection fraction increased and scarring decreased. If these results hold up in future studies, I believe this could be the biggest revolution in cardiovascular medicine in my lifetime."
The published paper in The Lancet is titled "Cardiac stem cells in patients with ischaemic cardiomyopathy (SCIPIO): initial results of a randomised Phase I trial.”