Shahin Rafii, M.D.
Scientific Founder
Dr. Rafii is the Arthur B. Belfer Professor of Genetic Medicine at Weill Cornell Medicine. Dr. Rafii, a board certified hematologist-oncologist, is engaged in basic and translational research as well as patient care. He is the director of the Ansary Stem Cell Institute at Weill Cornell and is a Howard Hughes investigator.
Dr. Rafii was the first to develop the innovative E4-ORF1 technology to generate abundant stable and functional endothelial cells. He received his M.D. degree from Albert Einstein College of Medicine and completed his internship and residency in Internal Medicine followed by a fellowship in Hematology-Oncology at Weill Cornell.
He carried out his post-doctoral training in vascular and hematopoietic stem cell biology under the supervision of Dr. Ralph Nachman and Malcolm Moore at Weill Cornell. He is the principal inventor in numerous patents on therapeutic uses of vascular cells for stem cell self-renewal and organ regeneration as well as tumor targeting.
Jason M. Butler, Ph.D.
Dr. Butler is the Director of Stem Cell Therapeutics and Professor of Medical Sciences at the Center for Discovery and Innovation at Hackensack Meridian Health, as well as an Associate Professor at Georgetown University. Dr. Butler obtained his B.S. in Zoology from the University of Florida in 2001 and continued his graduate education at UF where he completed his Ph.D. in stem cell biology in 2005. Dr. Butler completed his postdoctoral fellowship with Dr. Shahin Rafii
in stem cell and vascular biology at Weill Cornell Medical College in 2011.
Dr. Butler’s laboratory is dedicated to understanding the role of bone marrow endothelial cells (BMECs) in establishing unique instructive vascular niche cells that produce the correct milieu and stoichiometry of growth factors to support hematopoiesis during physiological aging and hematopoietic regeneration following hematopoietic insults, such as chemotherapy and inflammation. Research from Dr. Butler’s laboratory has shown that BMECs are indispensable for supporting hematopoietic stem cell (HSC) self-renewal and differentiation into lineage-committed progeny. Notably, they have recently demonstrated that aged BMECs can instruct young HSCs to function as aged HSCs, whereas young BMECs can rejuvenate aged HSCs. Building upon these findings, the Butler laboratory has identified the critical signaling pathways altered within the aged endothelial niche that impair their instructive capacity to support HSCs and hematopoiesis. They have also demonstrated that as little as one dose of sublethal
chemotherapy leads to significant damage to the BM vascular niche and that regeneration of the vascular system is crucial for the recovery of the hematopoietic compartment. Dr. Butler has published data demonstrating that infusion of BMECs can restore and protect an aged BM microenvironment and improve the functional output of aged HSCs, as well as provide widespread vascular protection, following myelosuppressive injury. Indeed, these foundational pre-clinical studies led to Angiocrine Bioscience’s Phase 1b/2 clinical trial wherein endothelium are being infused during HSC transplants to accelerate regeneration of vascular niches following myeloablation.
The overarching goal of Dr. Butler’s research program is to gain fundamental insights into how aging interferes with proper blood vessel function within the bone marrow microenvironment, which in turn impairs the vascular system’s ability to function as a supportive niche that regulates hematopoiesis. Dr. Butler’s laboratory is aiming to deconstruct the mechanisms guiding the interactions between the blood stem cell and the bone marrow vascular niche to reveal key molecular and cellular mechanisms that negatively impact the hematopoietic and cardiovascular systems. The Butler laboratory is currently developing protocols to modulate novel anti-aging targets for therapeutic rejuvenation of hematopoietic and vascular systems to enhance longevity and health-span.
David Cheresh, Ph.D.
Dr. Cheresh is Distinguished Professor and Vice Chair of Pathology at the Moores Cancer Center in the University of California, San Diego. He is also Director for Translational Research at the Moores UCSD Cancer Center. Prior to relocating his laboratory in 2005, Dr. Cheresh was a professor in the Departments of Immunology and Vascular Biology at The Scripps Research Institute, focusing on the role of adhesion receptors and growth factors in the angiogenesis of tumors. Dr. Cheresh received his doctorate in Immunology from the University of Miami in Florida. In 1982, he joined The Scripps Research Institute as a postdoctoral fellow in the Department of Immunology and moved up through the ranks where he became Professor in 1996. Dr. Cheresh is the recipient of various awards including the 15th Hans Linder Memorial Lecture from the Weizmann Institute of Science in Rehovot, Israel, the XXIII Annual Myron Karon Memorial Lectureship from the University of Southern California, the Robert Flynn Professorship Award from Tufts University School of Medicine, the Judah Folkman lectureship, the Paget-Ewing award from the Metastasis Research Society/AACR and was a recipient of The American Cancer Society Faculty Research Award and a Merit Award from the National Cancer Institute.
Dr. Cheresh studies the mechanism of action of signaling networks that regulate angiogenesis, tumor growth, stemness, drug resistance and metastasis. He discovered that integrin ανβ3 is a functional marker of angiogenic blood vessels. His work is both basic and translational focusing on new strategies for biologically-based drug development including the development of several drugs now in various stages of clinical development. Dr. Cheresh’s research has been widely cited with seven of his peer-reviewed publications being cited over 1,000 times. Dr. Cheresh was the scientific founder of TargeGen, a San Diego based biotechnology company, which developed a number of small molecules based in part on discoveries made in the Cheresh laboratory. TargeGen’s JAK2 inhibitor has shown clinical activity in patients with myeloproliferative disease and has met its primary endpoint in a Phase III registration trial. Most recently, Dr. Cheresh and his colleagues have developed a novel scaffold-based chemistry approach to stabilize kinases in their inactive state. These studies have lead to the discovery of a first in class RAF inhibitor that has distinct advantages relative to ATP mimetics of RAF.
Hans-Peter Kiem, M.D., Ph.D.
Dr. Hans-Peter Kiem is a world-renowned pioneer in stem-cell and gene therapy and in the development of new gene-editing technologies. His focus has been the development of improved treatment and curative approaches for patients with genetic and infectious diseases or cancer. For gene editing, his lab works on the design and selection of enzymes, known as nucleases, which include CRISPR/Cas. These enzymes function as molecular scissors that are capable of accurately disabling defective genes. By combining gene therapy’s ability to repair problem-causing genes and stem cells’ regenerative capabilities, he hopes to achieve cures of diseases as diverse as HIV, leukemia and brain cancer. With preclinical models of HIV, Dr. Kiem and his colleagues have demonstrated that they can modify a key viral entry gene and prevent it from working in transplanted blood stem cells. He also hopes to apply these technologies to cure genetic blood disorders such as Fanconi anemia and sickle cell disease. He is also pioneering in vivo gene therapy approaches to make gene therapy and gene editing more broadly available and accessible to patients and those living with HIV, especially in resource-limited settings.
Lee M. Ellis, M.D.
Dr. Ellis graduated from the University of Virginia School of Medicine in 1983, and completed his residency in surgery at the University of Florida in 1990. Dr. Ellis went on to complete a surgical oncology fellowship at the M. D. Anderson Cancer Center (MDACC), where he has been on the faculty since 1993. Dr. Ellis has a clinical practice in Surgical Oncology, focused on patients with colorectal cancer and liver metastases. Academically, Dr. Ellis has established a reputation for expertise in the area of angiogenesis and growth factor receptors in gastrointestinal malignancies and is funded by several grants for research in this area. He has served on numerous NIH study sections and is a consultant to the National Cancer Institute, where he currently serves on the NCI GI Cancers Steering Committee. In 2000, Dr. Ellis was awarded the Faculty Scholar Award from the MDACC, and he was also the inaugural recipient of a grant from the George and Barbara Bush Endowment for Innovative Cancer Research. In 2007, he was awarded the William C. Liedtke, Jr., Chair in Cancer Research.
Dr. Ellis serves on 8 editorial boards, including the Journal of Clinical Oncology, Cancer Research, and Clinical Cancer Research (Senior Editor). Dr. Ellis has also authored over 220 peer-reviewed publications, 90 invited reviews and editorials, three books, and 30 book chapters. In 2013, he published a seminal paper in Cancer Cellon Angiocrine signaling. In addition, he has published important editorials in journals such as the NEJM, Nature, Cell, Cancer Cell, Nature Medicine, JNCI, JCO, and The Lancet. Dr. Ellis has been a plenary discussant at ASCO in 2005 and 2009. He is co-chair of the largest clinical angiogenesis meeting in the world held annually in February. Dr. Ellis serves in a leadership role in the major cancer societies such as the American Society for Clinical Oncology (immediate past chair of the Cancer Research Committee, and a member of the Nominating Committee), and the American Association for Cancer Research (Chair of CME Committee). Dr. Ellis currently serves on the ECOG DMB. He was Chair of the 2007 GI Cancers Symposium and was Co-Chair of the 2009 and 2011 AACR annual meetings. Dr. Ellis chaired a Keystone Symposium on Angiogenesis in January 2009. In June 2008, he was appointed Chair, Ad Interim, Department of Cancer Biology at MDACC and served in that position until June 2012. He was Director of the Metastasis Research Center from 2010-2012. Dr. Ellis served as Co-Director for the ASCO/AACR Workshop on Methods in Clinical Cancer Research from 2010-2012. In May, 2013, Dr. Ellis assumed leadership roles in SWOG including Vice Chair for Translational Medicine, and serving as a member of the Executive Committee.
Robert S. Kerbel, Ph.D.
After graduating from the University of Toronto, Dr. Kerbel commenced graduate studies receiving a Ph.D. in 1972; he then undertook postdoctoral training in London after which he started his independent research program in 1975 at Queen’s University in Kingston, in the Cancer Research Laboratories, becoming its Director in 1981. In 1985 he was recruited to develop and direct a cancer biology research division at Mt. Sinai Hospital Research Institute in Toronto; he then moved to Sunnybrook Health Sciences Centre (where he is presently located) to do the same from 1991 until 2001. He currently holds a Canada Research Chair in Tumor Biology, Angiogenesis, and Antiangiogenic Therapy and is a professor in the Dept. of Medical Biophysics at the University of Toronto. Throughout his career, Dr. Kerbel’s main interest has been to devise new cancer treatment strategies having both improved efficacy and reduced toxicity. This culminated in his pioneering studies of lower dose frequently administered ‘metronomic’ chemotherapy and antiangiogenic drugs. A number of his findings are now being evaluated in Phase II and Phase III clinical trials, particularly in breast, ovarian, colorectal, and various pediatric cancers. His other contributions include developing improved preclinical models involving early stage or advanced metastatic disease for experimental therapeutic investigations, linking the fields of angiogenesis and oncogenes, uncovering mechanisms by which antiangiogenic drugs increase the efficacy of chemotherapy, or alter malignant tumor progression, especially in the adjuvant treatment setting, and elucidating mechanisms of resistance to antiangiogenic drugs.
Dr. Kerbel’s work has been supported by grants from four Canadian funding agencies and the US National Institutes of Health. He has published 372 papers, given over 750 invited lectures including numerous keynote and plenary presentations. Among the recent honors and awards he has received include the Canadian Cancer Society Robert Noble Award for Excellence in Cancer Research, the Breast Cancer Research Award from the European Institute of Oncology, and a Man of Distinction Honor by the Israel Cancer Research Fund.