Lancet: Infusing autologous cardiosphere-derived stem cells after MI is safe

Infusion of cardiosphere-derived stem cells (CDCs) into patients who had had heart attacks can help regenerate healthy heart muscle, according to a prospective, randomized trial published Feb. 13 in The Lancet.

Between May 5, 2009, and Dec 16, 2010, in the CADUCEUS (CArdiosphere-Derived aUtologous stem CElls to reverse ventricUlar dySfunction) study, Eduardo Marbán, MD, PhD, of the Cedars-Sinai Heart Institute in Los Angeles, and colleagues assessed 25 patients, average age 53 years, who experienced an MI two to four weeks prior (with left ventricular ejection fraction of 25 to 45 percent).

The researchers treated patients at Cedars-Sinai Heart Institute and at Johns Hopkins Hospital in Baltimore. Of these, eight received standard care while 17 received infusions of CDCs, which are cardiac stem cells created using the patient's own heart tissue.

The primary endpoint was the proportion of patients at six months who died due to ventricular tachycardia, ventricular fibrillation, sudden unexpected death or had an MI after cell infusion, new cardiac tumor formation on MRI, or a major adverse cardiac event—defined as composite of death and hospital admission for heart failure or non-fatal recurrent MI.

The procedure was minimally invasive and involved removing pieces of living heart muscle around half the size of a raisin using a catheter under local anesthetic; this tissue was then used to create the supply of cardiac stem cells. Each patient received an infusion of around 12 to 25 million of his or her own stem cells during a second minimally invasive procedure.

Patients who had the stem cell infusion saw their scar size drop from 24 percent to 12 percent of the heart on average (a reduction by about 50 percent), while controls saw no reduction in scar size. Changes in end-diastolic volume, end-systolic volume and left ventricular ejection fraction did not differ between groups by six months.

Four patients (24 percent) in the stem cell group had serious adverse events compared with one control (13 percent), although of the four events in the stem cell group, only one was regarded as possibly related to the treatment.

In the CADUCEUS trial, 17 patients underwent endomyocardial biopsy sampling about four weeks after MI to generate autologous CDCs that were subsequently injected into the infarct-related artery after about five weeks; outcomes were compared with those for eight patients who received conventional medical therapy, according to the accompanying editorialists, Chung-Wah Siu, MD, and Hung-Fat Tse, MD, of the cardiology division of the University of Hong Kong, Queen Mary Hospital, in Hong Kong, China.

“No acute complications related to biopsy sampling or cell delivery were noted,” Siu and Tse wrote. “At six months and 12 months, the rates of serious adverse effects and arrhythmias did not differ between patients in the CDC group and controls. Nevertheless, one patient developed recurrent MI and four patients received additional cardiac interventions (including one patient who had coronary revascularization and three who needed implantation of prophylactic defibrillators) in the CDC group.”

"This discovery challenges the conventional wisdom that, once established, cardiac scarring is permanent and that, once lost, healthy heart muscle cannot be restored," explained the study authors. 

The study authors concluded that “intracoronary infusion of autologous CDCs after MI is safe, warranting the expansion of such therapy to phase II study. The unprecedented increases we noted in viable myocardium, which are consistent with therapeutic regeneration, merit further assessment of clinical outcomes.”

In an accompanying commentary, Siu and Tse wrote: "These findings suggest that this therapeutic approach is feasible and has the potential to provide a treatment strategy for cardiac regeneration after MI. … [The] trial will hopefully alert researchers and clinicians to the potential of cardiac regeneration as a new paradigm for treatment after MI.”

The U.S. National Heart, Lung and Blood Institute and Cedars-Sinai Board of Governors Heart Stem Cell Center funded the trial.

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