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conferenceseries.com
October 26-27, 2016 Chicago, USA
Annual Congress on
Rare Diseases & Orphan Drugs
Volume 7, Issue 5 (Suppl) J Genet Syndr Gene Ther
ISSN: 2157-7412 JGSGT, an open access journal Rare Diseases 2016
October 26-27, 2016
A robust reprogramming approach to create viral-free and oncogene-free, orphan-disease specific induced
pluripotent stem cells from peripheral blood mononuclear cells
Alan B Moy1, 2
1The John Paul II Medical Research Institute, USA
2Cellular Engineering Technologies, USA
Innovations are needed to reduce the time, cost and failure rate of drug discovery. 58% of new molecular entities approved by
the FDA in the past ten years have utilized phenotypic screening; a drug screening process that uses patient-specific cultured
cells that reflect the patient’s diseased characteristics. Induced pluripotent stem cells (IPSC) are excellent patient-specific stem
cells for phenotypic drug screening platforms for orphan disease. Disease-specific IPSC has the advantage over prior conventional
approaches to better predict drug efficacy and safety and for patient stratification in clinical trials. Yet, one major hurdle in creating
disease-specific IPSC for infants and children has been developing reliable methods to reprogram target cells derived from peripheral
blood, which is the ideal minimally invasive approach to transform target cells. However, efficient reprogramming of peripheral
blood mononuclear cells has not been achieved without viral and the oncogenes, c-Myc and Lin28. These oncogenic transcriptional
factors and viral elements may alter the native phenotype of a patient’s cell and skew drug screening outcomes. We have solved
this problem by developing a combinatorial approach of small molecules and a novel episomal construct to reprogram adherent
cells and peripheral blood mononuclear cells. While the combinatorial approach is efficient for reprogramming adherent cells, the
approach requires an additional intermediate conversion of peripheral mononuclear cells (PMNC) into CD34+ hematopoietic stem
cells. PMNC were exposed to a defined tissue cultured media that converted PMNC into a sufficient number of hematopoietic stem
cells. Upon conversion to hematopoietic stem cells, IPSC reprogramming became feasible. Without an intermediate hematopoietic
stem cell conversion, we observed no IPSC colony formation. The opportunity now exists to develop a repository of IPSC for infants
and children suffering from orphan diseases from a simple venipuncture, which should accelerate hit to lead identification, drug
optimization and drug formulation.
Biography
Alan B Moy has established a successful career in academia, non-profits and industry. He has received his MD from Creighton University, completed his Internal
Medicine Residency at St. Louis University and Pulmonary Fellowship at the University of Iowa. He has served on Faculty at the University of Iowa College of
Medicine and College of Engineering with a research expertise in cellular and tissue engineering. He is the Founder and Scientific Director of the John Paul II
Medical Research Institute, a 501 (C)(3) and is the CEO and Co-Founder of Cellular Engineering Technologies, a leading stem cell manufacturing company. He
is listed in the Leading Physicians of the World by the International Association of Healthcare Professionals. His area of expertise includes pulmonary medicine,
cytoskeletal biology, vascular biology, tissue engineering and industrial stem cell manufacturing.
Alan B Moy, J Genet Syndr Gene Ther 2016, 7:5 (Suppl)