The Sanford Consortium for Regenerative Medicine’s Scientific Symposium and the Regen Med Partnering Forum at Stem Cell Meeting on the Mesa VIII

The Scientific Symposium at Stem Cell Meeting on the Mesa (SCMOM) aims to advance scientific progress through the collaboration of over 16 researchers and academic leaders who were selected to present at the 8th annual conference in 2013. The focus of the meeting was the dissemination of scientific results to area researchers through seminar presentations, question and answers, a poster session and networking. Four panels and two keynote speakers centered their talks on translation of advances in regenerative medicine. The morning keynote speaker, Ann Tsukamoto, Ph.D., of StemCells, Inc., presented Human Neural Stem Cells from Discovery to Clinic. To close the meeting, keynote speaker Andrew McMahon, Ph.D. of University of Southern California spoke on Generation and Repair of the Kidney. The panels were Stem Cells and Aging, Drug Discovery and Endogenous Stem Cells, Tracking Stem Cells Using In Vivo Imaging, and Disease Modeling. Presentations documented the impact of stem cell research on the understanding of human biological mechanisms, amelioration of human health, and treatment of disease and trauma. For example, Larry Goldstein discussed how using iPS cells from patients can better help understand the genetic risk of neurodegenerative diseases like Alzheimer’s. He showed how the risk of manifesting Alzheimer’s disease is associated with a gene called SORL1, as well as many other genes also known to be risk factors. He discussed how assessing the risk from SORL1 can be better understood as a question of how SORL1 influences a positive response to BDNF. The implication was that for clinical trials that act by the BDNF pathway, study populations ought to be stratified on the basis of SORL1 genotype to make trial results more meaningful. In the drug discovery section, Luke Lairson discussed the fact that existing treatments for multiple sclerosis (MS) exclusively target immunological mechanisms. He discussed an alternative approach to this devastating disease, a therapeutic idea from regenerative medicine. This new approach tries to enhance neural remyelination and thus protect the nerves that MS damages. He wanted a compound that might differentiate the early versions of the cells that create the myelin sheath to protect the nerve ("OPCs)" to mature myelinating oligodendrocytes. Using high-throughput drug screening, he found that benztropine, an approved drug for Parkinson's disease, had the attributes he sought, and resulted in some of the in vitro and in vivo remylenation he was looking for. Dr. Robert Kass (Columbia University), gave further examples of the power of pluripotent stem cells to ameliorate a bad situation. In his example, a pediatric patient with Long QT syndrome could not be helped by the drugs used normally to improve the heart condition. In collaboration with the child’s cardiologists, iPSC from the patient were converted to cardiomyocytes (cardiac muscle cells). The iPS cells from the patient allowed the doctors and researchers to understand the specific genetic background of this patient that caused the bad, off-target effects of the drugs usually used to treat Long QT Syndrome. Notably, the iPSC-derived cardiomyocytes were then used to develop a therapy that was ultimately used to treat the patient. These cells could also be used to guide development of the right new drugs, based on existing ones, to treat such patients.