CardioCell LLC has just received the FDA's investigational new drug (IND) approval for a United States-based, Phase IIa clinical study using its allogeneic stem-cell therapy to treat subjects with chronic heart failure (CHF), which generates more than 1 million hospitalizations annually. Preparation is underway to initiate the study, "A Phase IIa, Single-Blind, Placebo-Controlled, Crossover, Multi-Center, Randomized Study to Assess the Safety, Tolerability and Preliminary Efficacy of a Single Intravenous Dose of Ischemia-Tolerant Allogeneic Mesenchymal Bone Marrow Cells to Subjects With Heart Failure of Non-Ischemic Etiology," at Emory University, Northwestern University and the University of Pennsylvania in May 2014.
"With the FDA's IND approval, CardioCell is pleased to proceed with a Phase IIa CHF clinical trial based on the safety data reported in previous clinical trials using our unique, hypoxically grown stem cells," says Dr. Sergey Sikora, Ph.D., CardioCell's president and CEO. "At the study's conclusion we will understand if our therapy produces signs of improvement in a population of patients with dilated CHF, a condition largely unaddressed by current therapies. Dilated CHF is characterized by a viable but non-functioning myocardium in which cardiomyocytes are alive but are not contracting as they should. We hope that unique properties of our itMSCs will transition patients' cardiomyocytes from viable to functioning, eventually improving or restoring heart function."
Only CardioCell's CHF therapies feature itMSCs, which are exclusively licensed from CardioCell's parent company Stemedica Cell Technologies Inc. Unlike all other MSCs - which are grown under normoxic conditions - Stemedica's bone-marrow-derived, allogeneic itMSCs are unique because they are grown under hypoxic conditions that more closely resemble the environment in which they live in the body. Compared to other MSCs, itMSCs secrete higher levels of growth factors usually associated with angiogenesis and healing. The current CHF study has been designed to help determine if CardioCell's itMSC-based therapies stimulate the transition of viable but non-functioning myocardium into functioning myocardium.
"Many mechanisms contribute to the cardiac-dysfunction characteristic of dilated cardiomyopathy (DCM)," says MedStar Heart Institute's Director of Translational and Vascular Biology Research and CardioCell's Scientific Advisory Board Chair Dr. Stephen Epstein. "They include the progressive death of myocytes, chronic inflammation, and myocardial ischemia (the latter probably due to disease of the microvasculature); both inflammation and ischemia contribute to functional impairment of still-viable myocardium. Although past trials have tested the efficacy of different stem cells in patients with DCM, CardioCell's itMSCs, grown under chronic hypoxic conditions, are unique. As compared to stem cells grown under normoxic conditions, they express higher levels of factors that could exert beneficial effects on the mechanisms contributing to myocardial dysfunction and disease progression. This study, therefore, provides an exciting opportunity to test the potential of these itMSCs to attenuate or eliminate these mechanisms and, in so doing, improve patient outcomes."
CardioCell is making significant progress in clinical trials for CHF, which represents the single largest cause of natural death in the United States. According to the American Heart Association, approximately 5.5 million individuals suffer from CHF, and it generates more than 1 million hospitalizations annually, with a striking 50-percent mortality and re-admission rate after 60-90 days post-discharge. CardioCell has developed an itMSC-based treatment for the type of dilated CHF that is not related to coronary artery disease, and its treatment could apply to approximately 35 percent of CHF patients. This treatment will be conducted via intravenous administration, which is non-invasive and, potentially, can be performed in outpatient facilities.