Editorial: The Designation of Human Embryonic Stem Cell Cardiac Therapy Derivatives for Human Trials

Editorial: The Designation of Human Embryonic Stem Cell Cardiac Therapy Derivatives for Human Trials

Dr. Parsons, founder of San Diego Regenerative Medicine Institute and Xcelthera, INC., has discussed scientific breakthroughs in human embryonic stem cell (hESC) research in her two recent Editorials, titled “Cellular medicine for the heart - the pharmacologic utility and capacity of human cardiac stem cells” at J. Clinic. Exp. Cardiology 2013;S11-e001 (doi: 10.4172/2155-9880.S11-e001) & “Reviving cell-based regenerative medicine for heart reconstitution with efficiency in deriving cardiac elements from pluripotent human embryonic stem cells” at Cardiol. Pharmacol 2013;2(3):e112 (doi: 10.4172/2329-6607.1000e112). Such breakthrough developments have demonstrated the direct pharmacologic utility and capacity of hESC cell therapy derivatives for human CNS and myocardium regeneration, thus, presented the hESC cell therapy derivatives as a powerful pharmacologic agent of cellular entity for CNS and heart repair.

Cardiovascular disease is a major health problem and the leading cause of death in the Western world. About 600,000 people die of heart disease in the United States every year–that’s 1 in every 4 deaths. The estimated costs of cardiovascular disease for the overall US population are approximately $190 billion annually. Currently, there is no treatment option or compound drug of molecular entity that can change the prognosis of cardiovascular disease. Given the limited capacity of the heart for self-repair or renewal, cell-based therapy represents a promising therapeutic approach closest to provide a cure to restore normal heart tissue and function for heart disease and failure. However, traditional sources of cells for therapy in existing markets have been adult stem cells isolated from tissues or artificially reprogrammed from adult cells, which all have the historical shortcomings of limited capacity for renewal and repair, accelerated aging, and immune-rejection following transplantation. In addition, artificially reprogrammed adult cells have the major drawbacks of extremely low efficiencies and genetic defects associated with high risks of cancers, which have severely limited their utility as viable therapeutic approaches. In the adult heart, the mature contracting cardiac muscle cells, known as cardiomyocytes, are terminally differentiated and unable to regenerate. There is no scientific evidence that adult stem/precursor/progenitor cells derived from mature tissues, such as bone marrow, cord blood, umbilical cord, mesenchymal stem cells, patients’ heart tissue, placenta, or fat tissue, are able to give rise to the contractile heart muscle cells following transplantation into the heart. Despite numerous reports about cell populations expressing stem/precursor/progenitor cell markers identified in the adult hearts, the minuscule quantities and growing evidences indicating that they are not genuine heart cells and that they give rise predominantly to non-functional smooth muscle cells rather than functional contractile cardiomyocytes have caused skepticism if they can potentially be harnessed for cardiac repair. Although a vast sum of government and private funding has been spent on looking for adult alternates, such as reprogramming and trans-differentiation of fibroblasts or mature tissues, so far, only human cardiac stem/precursor/progenitor cells derived from embryo-originated hESCs have shown such cellular pharmacologic utility and capacity adequate for myocardium regeneration in pharmaceutical development of stem cell therapy for the damaged heart.

Recent milestone advances and medical innovations in hESC research enable high efficient direct conversion of non-functional pluripotent hESCs into a large supply of clinical-grade high purity functional human neuronal cells or heart muscle cells for developing safe and effective stem cell therapies as treatments or cures for a wide range of neurological and cardiovascular diseases. Currently, these hESC neuronal and cardiomyocyte cell therapy derivatives are the only available human cell sources in commercial scales with adequate cellular pharmacologic utility and capacity to regenerate CNS neurons and contractile heart muscles, vital for CNS and heart repair in the clinical setting. Transforming non-functional pluripotent hESCs into fate-restricted functional human cell therapy derivatives or products allows moving stem cell research from current studies in animals towards human trials.