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Andrew P. McMahon, Ph.D., FRS | Keynote Speaker

Professor Andrew (Andy) McMahon is the W. M. Keck Provost and University Professor of Stem Cell Biology and Regenerative Medicine, and Biological Sciences, and Chair of Stem Cell Biology and Regenerative Medicine at USC. His group was the first to identify key signals coordinating cell interactions directing the assembly of several mammalian organ systems—research that led to a biotechnology startup and the first drug treatment for an invasive form of skin cancer. Current research focuses on understanding the development, injury, and repair of the mammalian kidney to prevent and treat renal disease.

Abstract: Developmental strategizing towards the treatment of kidney disease

An understanding of the developmental programs that build, maintain and repair our body systems provides the foundation for regenerative medicine. Our laboratory’s interest is the kidney. The kidney is an essential organ responsible for maintaining the normal balance of tissue fluids, removal of soluble waste products of metabolism, maintenance of blood cell composition and regulation of blood pressure, and control of bone biology. Over 750,000 Americans have end-stage renal disease. While a kidney transplant provides a many-year solution, there are insufficient transplantable kidneys to meet the need, and the temporary solution of dialysis is associated with high mortality and morbidity. New solutions are required for patients with kidney disease. Through the comparative analysis of mouse and human kidney development, we are gaining a comprehensive understanding of the programs that build a functional kidney. Pluripotent stem cell-derived kidney organoids provide a platform for generating functional kidney cell types, exploring regulatory processes and modeling disease. Using a mini-kidney organoid-directed polycystic kidney disease platform suited for pharmacological screens, we have identified small molecule pathway modulators that block kidney cyst formation and expansion. Improving the current kidney organoid model through the application of insight obtained from normal developmental programming and optimizing strategies for disease modeling and drug screening is a rational path to identifying and refining novel therapeutics for the treatment of kidney diseases.


 
 

Jennifer Woodell-May, Ph.D. |

Jennifer Woodell-May, Ph.D. is currently the Program Director of Advanced Osteoarthritis Therapies for the Biologics division of Zimmer Biomet in Warsaw, IN. She received her Ph.D. in Bioengineering from Clemson University in 2001, her M.S. in Bioengineering from Clemson in 1996, and a B.S. in Physics from Furman University in 1995. Since 2001, she has been in R&D in the Orthobiologics division for Biomet, Inc. and then exclusively for Biomet Biologics beginning in 2007. She manages research activities for currently marketed therapies as well as pre-clinical, clinical, and regulatory efforts for new technologies.

Jennifer’s research focuses are platelet-rich plasma, stem cells, demineralized bone matrix, and autologous anti-inflammatory agents. She has published several abstracts, papers, and a book chapter on the basic science and orthopedic uses for platelet-rich plasma. In addition, Jennifer is the 2019-2020 chair of the Women’s Leadership Forum of the Orthopaedic Research Society. She also is an Adjunct faculty for the Bioengineering Department of Clemson University and sits on the Clemson Bioengineering External Advisory Board.

Abstract: Potential Mechanism of Action of Concentrated Bone Marrow Aspirate in Regenerative Medicine

Concentrated bone marrow aspirate (cBMA) has been explored as potential therapies in preclinical models and clinical trials in a variety of therapeutic indications. Characterization of cBMA could inform how it might address various disease conditions. The first proposed mechanism of action for cBMA was attributed to “stem cells.” However, cBMA contains a low number of stem cells in comparison to culture-expanded stem cell approaches. An emerging understanding of cBMA is that it is a white blood cell, progenitor cell, and platelet-rich product which enables it to contain a high concentration of anti-inflammatory cytokines and anabolic growth factors that can act both in anti-inflammatory and angiogenic fashion.


 
 
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Lawrence S.B. Goldstein, Ph.D. |

Lawrence S.B. Goldstein is a Cell Biologist, Geneticist and Neuroscientist recognized for his work on molecular motors and the role of molecular transport pathways in neurodegenerative disease. Goldstein was born in Buffalo, New York and grew up in Thousand Oaks, California. He graduated from UC San Diego with a degree in Biology in 1976 and from the University of Washington with a Ph. D. in Genetics in 1980. He was a postdoctoral fellow in Cell Biology at University of Colorado Boulder and MIT. He joined the faculty in Cell and Developmental Biology at Harvard University in 1984 where he was promoted to Full Professor with tenure in 1990. He returned to UC San Diego and the Howard Hughes Medical Institute in 1993. He is currently a Professor of Cellular and Molecular Medicine and of Neurosciences at UC San Diego. In collaboration with faculty and administrative colleagues he launched the UC San Diego Stem Cell program, the Sanford Consortium for Regenerative Medicine and the Sanford Stem Cell Clinical Center. He has received the Public Service Award from the American Society for Cell Biology and has had a Public Policy Fellowship named for him by the International Society for Stem Cell Research. He is a member of the American Academy of Arts and Sciences and the National Academy of Sciences.

Abstract: Insights from stem cell derived models of Alzheimer’s Disease

Although animal models of Alzheimer's disease have been very useful, they have not yet led to an effective treatment or a complete understanding of what goes wrong in the human brain during AD. Human in vitro models derived from human induced pluripotent stem cells (hIPSC) may prove to be of substantial utility given that they are euploid and can differentiate to bona fide cell types. In addition, purified cells with controlled genetic background and content can be cultured and differentiated together to develop functional cell structures composed of different cell types and genomes. In my talk, I will discuss our recent progress including a description of our drug screening efforts with FAD APP duplication neurons, which have turned up some very interesting potential leads. I will also summarize our efforts studying and comparing early phenotypes of FAD mutations in isogenic backgrounds.


 
 
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Anthony Ting, Ph.D. |

Dr. Anthony Ting is the Vice President of Regenerative Medicine and Head of Cardiopulmonary Programs at Athersys. He has over 30 years of experience in cell and stem cell biology, with expertise in translational clinical studies using adult stem cell therapies, and has managed the entire process in the development of MultiStem®. In addition, Dr. Ting serves on various regenerative medicine society committees, such as the Alliance for Regenerative Medicine as well as the International Society for Cell Therapy. Prior to Arthersys, Dr. Ting worked as a Principal Investigator and Head of Screening for Novel Inhibitors group at the Institute of Molecular and Cell Biology (IMCB) at the National University of Singapore. Preceding IMCB, Dr. Ting was a postdoctoral fellow with Dr. Richard Scheller at the Department of Molecular and Cellular Physiology at Stanford University. He received his Ph.D in Cell Biology from Johns Hopkins University and B.A. in Biology from Amherst College.

Abstract: Development of Cell Therapy for Acute Respiratory Distress Syndrome

The development of a cell therapy product requires the integrations of multiple partners.  Here we will discuss the requirements for developing an adherent adult bone marrow-derived cell product, MultiStem, for a clinical trial in patients with acute respiratory distress syndrome (ARDS). Cell characterization and identification of mechanisms of action will be reviewed including results from in vivo models of ARDS. The design and top-level results from a phase 1/2 clinical study, MUST-ARDS will be presented.


 
 
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Ya-Huei Kuo, Ph.D. |

As a key researcher in the Department of Hematologic Malignancies Translational Science within Beckman Research Institute of City of Hope, Ya-Huei Kuo runs a laboratory focused on understanding the molecular anatomy of leukemia stem cells, and developing novel methods for targeting and attacking them. She has published dozens of scholarly papers and received the 2012 American Cancer Society Research Scholar Award. Dr. Kuo joined City of Hope in 2008 after a post-doctoral fellowship in Genetics, Hematology and Cancer Biology at University of Massachusetts Medical School. She received her Ph.D. in Cell Biology from the University of Connecticut and B.S. in Zoology from the National Taiwan University.

Abstract: Targeting Leukemia Stem Cells - Hitting a Moving Target

Acute myeloid leukemia (AML) is the most common type of leukemia in adults and chemotherapy remains the current standard of care. The overall five-year relative survival rate for AML patients is only 28%, mainly due to therapy resistance and relapse. Leukemia stem cells (LSCs) represent a reservoir of self-renewing malignant cells that is believed to drive therapy resistance and relapse. New therapies effective in eradicating LSCs are needed to improve the outcomes of AML patients. Studies have shown that relapse relevant LSCs can arise from multiple sources, including the rare primitive malignant stem/progenitor cells, committed myeloid cells with self-renewing properties, and continuing clonal evolution of pre-malignant hematopoietic stem cells (HSCs). The complex evolution of pre-leukemic HSC and LSC compartments presents a daunting challenge to selectively target functional leukemia-initiating cells. We will discuss our efforts in understanding the molecular program(s) supporting LSC evolution and highlight the role of histone deacetylase 8 (HDAC8) and microRNA (miR)-126 in promoting LSC activity and maintenance. Our studies indicate that HDAC8 inhibitors and miRisten, an anti-miR-126 therapeutic, show promising efficacies in targeting AML LSCs.


 
 
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Christopher J Centeno, M.D. |

Christopher J. Centeno, M.D. is an international expert and specialist in regenerative medicine and the clinical use of mesenchymal stem cells in orthopedics. He is board certified in physical medicine as well as rehabilitation and in pain management through The American Board of Physical Medicine and Rehabilitation.

Dr. Centeno is one of the few physicians in the world with extensive experience in the culture expansion of and clinical use of adult stem cells to treat orthopedic injuries. His clinic incorporates a variety of revolutionary pain management techniques to bring its broad patient base relief and results. Dr. Centeno treats patients from all over the US who travel to Colorado to under-go innovative, non-surgical treatments. Dr. Centeno has chaired multiple international research-based conferences. He also maintains an active research-based practice, with multiple publications listed in the US National Library of Medicine. Dr. Centeno has also served as editor-in-chief of a medical research journal dedicated to traumatic injury. Dr. Centeno trained at the Baylor College of Medicine, Texas Medical Center and the Institute for Rehabilitation Research. He hails from both Florida and New York and currently resides in Boulder, Colorado with his wife and three children.

Abstract: Can Interventional Orthopedics Procedures Using Simple Regenerative Medicine Products Reduce the Need for Invasive Orthopedic Surgery?

Medicine has often moved from more invasive to minimally invasive procedures. For example, much of what was cardiothoracic surgery in the 1990s has now been replaced by interventional cardiology. The same trend is unfolding in orthopedic care, where interventional procedures focused on using simple regenerative medicine products are replacing the need for invasive surgery. RCT data on several interventional orthopedic procedures using platelet-rich plasma and bone marrow concentrate will be discussed. In addition, the cost savings from using these procedures will be reviewed in addition to how large corporations are adding these procedures to insurance plans.