Ralph H. Johnson VA Medical Center
Cardiac stem cell clinical trial to help Veterans
The Ralph H. Johnson VA Medical Center, in partnership with the Medical University of South Carolina (MUSC), began a new cardiac stem cell clinical trial for Veterans and civilians affected by heart failure with preserved ejection fraction, a common but typically difficult to treat heart disease. The trial is one of five research projects funded through a $10 million Department of Defense grant, initiated by the Congressionally Directed Medical Research Program, awarded to Cedars-Sinai Heart Institute in Los Angeles.
In March 2016, a study conducted at Cedars-Sinai was published in the Journal of the American College of Cardiology Basic and Translational Science, demonstrating that weeks after infusions of cardiosphere-derived cells (CDCs), fibrosis in the hearts of laboratory rats with hypertension and heart failure with preserved ejection fraction was “melted away like hot water on a sugar cube,” said Michael Zile, M.D., Charleston VAMC Cardiology physician and principal investigator of the clinical trial in Charleston.
“This novel therapy, if successful, will be useful for our Veterans, and improve the quality of their life and their survival.” - Michael Zile, M.D.
Following the publication of the study, the FDA approved Cedars-Sinai’s proposal for the clinical trial as a collaborative effort between the Ralph H. Johnson VA Medical Center, MUSC, the Cedars-Sinai Heart Institute and Capricor, a Los Angeles-based biotechnology company.
“The goal is to attract as many Veterans as possible to this trial because this is a common, important and devastating disease process that our veterans suffer from,” said Zile. “This novel therapy, if successful, will be useful for our Veterans, and improve the quality of their life and their survival.”
The trial will began in January 2017 and aims to enroll 40 patients. Twenty patients will receive the stem cells and 20 patients will be given a placebo. The studies will be conducted by VA employees with the assistance of two DOD research coordinators who are funded directly through the grant. As part of the close affiliation agreement between the Ralph H. Johnson VA Medical Center and MUSC, all Veteran and civilian participants will receive treatment at MUSC for patient convenience.
With approximately three million people - more than half the nation’s total heart failure patients - suffering from this type of heart failure in the U.S., this clinical trial has the potential to positively affect millions of people. Patients with this condition experience fibrosis, similar to scaring, in the heart muscles which in turn prevents the pumping chambers from filling with blood properly. This disease is devastating; 50 percent of those suffering from the disease die within five years of the initial diagnosis and each patient has a 50 percent chance they will be hospitalized with heart failure every six months.
“At this moment there are no specific, effective therapies in heart failure preserved ejection fraction that will reduce the disability these patients have, reduce the chance they’ll be admitted to the hospital for treatment, or increase their survival rate,” Zile said. “It’s for this reason that Dr. Sheldon Litwin [a trial collaborator, University adjunct professor and director of the VA Echo Lab] and I are making efforts to explore these novel therapies. There is an enormous unmet need in this area of cardiology in 2016.”
During the trial, CDCs will be injected inside of patients’ coronary arteries and migrate into the heart muscle. Zile explained that the trial in Charleston is pivotal to the other four, more basic, studies funded through the grant because it tests the utility of CDC therapy in humans.
“We know CDCs do not raise significant safety concerns because more than 100 patients have been given these stem cells for treatment of other diseases,” he said. “The question here is, are they an effective treatment for heart failure with preserved ejection fraction? We hope that the CDCs deliver an effective treatment that corrects what caused the heart to fail in the first place.”
Individual patients are monitored closely with face-to-face appointments for six months, and follow-ups via phone for the duration of the three-year study.