April 4, 2016, American College of Cardiology -- A new stem cell therapy significantly improved long-term health outcomes in patients with severe and end-stage heart failure in a study presented at the American College of Cardiology’s 65th Annual Scientific Session.

Among 109 patients randomized to receive the cell therapy or a placebo, those receiving the cell therapy, which involved extracting stem cells from a patient’s own bone marrow, showed a 37 percent lower rate of the trial’s primary endpoint, a composite of deaths, cardiovascular hospitalizations and clinic visits for sudden worsening of heart failure symptoms, over a 12-month period.

“To date, this is the largest double-blind, placebo-controlled stem cell trial for treatment of heart failure to be presented,” said Timothy Henry, M.D., director of cardiology at Cedars-Sinai Heart Institute and one of the study’s lead authors. “Based on these positive results, we are encouraged that this is an attractive potential therapy for patients with class III and class IV heart failure.”

Heart failure, which affects an estimated 5.1 million people in the United States, is a condition in which the heart progressively weakens and cannot pump enough blood to meet the body’s needs. Those with severe and end-stage heart failure—known as class III and IV heart failure on a scale developed by the New York Heart Association—often have no treatment options apart from a heart transplant or a left ventricular assist device, a heart pumping machine given to patients as a temporary measure while they await a heart transplant.

The study was a phase 2 clinical trial for a new stem cell therapy known as ixmyelocel-T. Using this technique, a doctor extracts a sample of bone marrow from a patient, processes it for two weeks to “enhance” it by increasing the number of beneficial stem cells, and then injects the processed bone marrow product into the same patient’s heart muscle. The goal of the procedure is to strengthen the heart by increasing the number of functioning heart muscle cells, an approach known broadly as regenerative therapy.

“We have a major unmet need for treating class III and IV heart failure,” Henry said. “I think this trial provides strong evidence that regenerative therapies are very promising for this group of people, who currently have limited options.”

The trial enrolled 109 patients with class III or IV heart failure resulting from ischemic cardiomyopathy, a type of heart failure that is related to restricted blood flow from a heart attack or coronary artery disease. Roughly half, 58 patients, were randomly assigned to receive intramyocardial ixmyelocel-T treatment, and 51 patients were assigned to receive a placebo. Patients in the control group underwent a bone marrow extraction and received a placebo injection two weeks later.

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