Scientific Report 2018 Scientific Report 2018 www.stjude.org/scientificreport 262 Danny Thomas Place Memphis, TN 38105 Physician Referral Service 866.278.5833 Translating Science General Information 901.595.3300 into Survival Behind the Cover The scientific image on the cover is a fluorescence image of pyramidal neurons in the auditory cortex. The brain can modify the structure and function of neuronal connections in response to sensory experiences. This ability is known as neuroplasticity. Stanislav S. Zakharenko, MD, PhD (Developmental Neurobiology), and his Faculty Editorial Board colleagues are investigating the role of neuroplasticity in the Terrence L. Geiger, MD, PhD Nickhill Bhakta, MD, MPH auditory cortex during learning and how dysfunction in that part of Michael A. Dyer, PhD the brain can cause catastrophic neurologic or psychiatric diseases. Alberto S. Pappo, MD Leslie L. Robison, PhD Carlos Rodriguez-Galindo, MD Stephen W. White, DPhil Stanislav S. Zakharenko, MD, PhD Editoral Direction Angela J. McArthur, PhD, ELS Creative Direction Jerry L. Harris Photography Peter Barta Seth Dixon Ann-Margaret Hedges Jere Parobek Prepared by Departments of Scientific Editing and Biomedical Communications St. Jude Children’s Research Hospital and ALSAC are registered trademarks. ST. JUDE INVESTIGATORS, BACKED BY EXTRAORDINARY RESOURCES AND SUPPORT TEAMS, HAVE THE FREEDOM TO FOCUS ON MAKING BIG DISCOVERIES. OUR CULTURE AND CAMPUS FOSTER THE FREE EXCHANGE OF IDEAS AMONG SCIENTISTS AND CLINICIANS TO PROMOTE CREATIVE, COLLABORATIVE SCIENCE. Privileged communication. Copyright © 2018 St. Jude Children’s Research Hospital. No part of this communication may be cited, reproduced, stored in a retrieval system, or transmitted by electronic or other means without prior written permission of the President and CEO and the appropriate investigator. This report reflects the activities of St. Jude Children’s Research Hospital during 2017. TRANSLATING SCIENCE INTO SURVIVAL CEO’S STATEMENT 4 USHERING IN A NEW ERA OF 6 EDUCATION AT ST. JUDE ST. JUDE CREATES AN INTERNATIONAL 20 RESOURCE FOR PEDIATRIC SOLID TUMOR RESEARCH AND TREATMENT A NEW APPROACH TO CALCULATING 36 THE COST OF SURVIVING CHILDHOOD CANCER DECIPHERING THE MECHANISMS OF 50 NEUROPLASTICITY IN LEARNING AND ANIMAL MODELS OF PSYCHIATRIC DISEASES LEADERSHIP PROFILES 66 SCIENTIFIC HIGHLIGHTS 72 PROGRAMS 84 ACADEMIC DEPARTMENTS 88 BOARDS & EXECUTIVE STAFF 98 OPERATIONS & STATISTICS 101 2018 Scientific Report | 2 3 | 2018 Scientific Report In the early 1960s, the first medical director of research, gene therapy, and cellular therapy St. Jude Children’s Research Hospital employed programs. The hospital also welcomed two new a unique sales pitch to lure talent to the fledgling department chairs: Stephen Gottschalk, MD, Bone hospital. “Here, you have the opportunity to make Marrow Transplantation & Cellular Therapy; and your own center. It’s unplowed ground. If you come Charalampos “Babis” Kalodimos, PhD, Structural here, you’re an innovator,” Donald Pinkel, MD, Biology. These scientists are leading exciting promised recruits. Within a decade, those words initiatives in their respective areas and helping proved true as the hospital showed the world that us to better understand life and disease. childhood cancer—once deemed incurable—could, Through the St. Jude Research Collaborative, in fact, be successfully treated. In the years that the institution is funding two new projects— followed, survival rates for childhood cancer have exploring gene therapy and gene editing to risen to more than 80%. St. Jude also has made a cure sickle cell disease and understanding the lifesaving difference for young people with other epigenetic regulation of pediatric cancer. These catastrophic illnesses, such as sickle cell disease or collaborations involve investigators at St. Jude, bleeding disorders. Harvard Medical School, Dana-Farber Cancer In this Scientific Report, we detail some of our Institute, and Rockefeller University. latest innovations. In the first feature, we celebrate Last year, St. Jude also expanded its footprint on the opening of the St. Jude Children’s Research campus and around the globe. We opened a new Hospital Graduate School of Biomedical Sciences, data center to consolidate the hospital’s advanced LLC. This historic event marks a new era of higher computing infrastructure and support resources, education at St. Jude. The second article describes began planning a 625,000–square foot advanced the recent creation of the Childhood Solid Tumor research center, and took steps to broaden the Network, a resource that freely provides well- institution’s reach for children with cancer in low- characterized solid tumor samples to researchers and middle-income countries. worldwide. This network holds the promise of expanding knowledge about these rare diseases For its efforts, St. Jude was ranked the No. 1 and expediting the development of more effective pediatric cancer hospital in the nation by U.S. treatments and cures. News & World Report. In addition, the hospital received several top workplace recognitions, The third story presents a new approach to including Fortune magazine’s “100 Best Companies calculating the cumulative burden of surviving to Work For,” Glassdoor’s “Best Places to Work,” childhood cancer. Using new statistical methods, and People magazine’s “50 Companies That Care.” epidemiology researchers are assessing the Faculty and staff were recognized with scores of chronic medical conditions that arise in this accolades, including the National Cancer Institute vulnerable population. They are also applying Outstanding Investigator Award and the Society of this approach globally to determine the true Memorial Sloan Kettering Prize. Fittingly, Dr. Pinkel cost of treating pediatric cancers in low- and was also inducted into the Tennessee Health Care middle-income countries, where officials often Hall of Fame. assume pediatric oncology care is economically out of reach. The fourth feature highlights Although we have made great strides, we still exciting discoveries in neuroplasticity and neural have much ground to cover. There is an urgency circuit research. Stanislav Zakharenko, MD, PhD, to our work, as we seek to cultivate untapped Developmental Neurobiology, and his colleagues potential and opportunities in science and medicine. are studying the mechanisms underlying age- We remain committed to pursuing the paths to related changes in auditory learning and severe discovery and innovation to ensure that children mental illness. This work is advancing our everywhere have the best hope for the future. knowledge and identifying novel therapeutic targets for patients with brain injury such as stroke or diseases like schizophrenia. Beyond the work outlined herein, St. Jude James R. Downing, MD established the Center for Advanced Genome President and Chief Executive Officer Engineering, a shared resource for basic 2018 Scientific ReportReport | | 4 4 55 | | 2018 2018 Scientific Scientific Report Report USHERING IN A NEW ERA OF EDUCATION AT ST. JUDE Since its founding, the mission of St. Jude Children’s Research Hospital has been to advance cures and means of prevention of pediatric catastrophic diseases. This cannot be achieved without the dedicated work of researchers who continuously push against the barriers of what we know and what we can do. St. Jude recognizes its obligation is not only to tackle these diseases now but also to train the next generation of scientists who will continue to fight to develop new cures for children with life-threatening illnesses. Alex Hughes 2018 Scientific ReportReport | | 6 6 77 | | 2018 2018 Scientific Scientific Report Report In 2015, the state of Tennessee granted St. Jude a charter to establish the St. Jude Children’s Research Hospital Graduate School of Biomedical Sciences, LLC, and become a PhD-granting academic institution. This created opportunities for some of the brightest and most motivated students in the country to come to St. Jude at the earliest stages of their scientific careers and join this fight. Here, we share the story of this historic achievement and what it means for the future of our institution. James I. Morgan, PhD; James R. Downing, MD; Stephen W. White, DPhil ST. JUDE BECOMES A DEGREE-GRANTING this concept. A graduate program would increase the INSTITUTION visibility of St. Jude’s research programs and draw to Since shortly after its inception, St. Jude began St. Jude laboratories top students from the U.S. and, accepting graduate students and fellows from other ultimately, around the world. Most importantly, a institutions to perform or supplement the research St. JudeB graduate program could imprint on students portion of their training in St. Jude laboratories. The at the earliest stage of their careers the integrated hospital quickly became a desired destination for approach to discovery and clinical application that many graduate students in the biological sciences. characterizes St. Jude science. Senior leaders recognized the unmet need These new scientists could carry St. Jude’s unique for graduate educational opportunities and emphasis on translating basic knowledge into cures contemplated expanding our academic programs. for childhood diseases to laboratories and institutions The group studied many options and concluded around the world. Once enrollment reaches its that St. Jude would have the greatest impact planned steady state, the graduate school will provide on developing scientists by establishing its