The ASPHO 2024 Distinguished Career Award goes to Dr. David A. Williams

Dr. David A. Williams Dr. David A. Williams has been awarded the 2024 American Society of Pediatric Hematology Oncology Distinguished Career Award. Throughout his career, David has had a major impact in multiple areas of pediatric hematology/oncology, with a remarkable range of expertise and accomplishments. He is an outstanding basic researcher, innova tive clinical trialist, dedicated clinician, enthusiastic teacher/mentor, tireless advocate, and visionary administrator. His major research interests are in the study of blood stem cell biology and the treatment of genetic blood diseases using gene therapy. Here are a few highlights of his many contributions to the field. David received his undergraduate degree from Indiana State University, where he met and married his wife of 50 years, Dr. Lucinda (Cindy) Williams, who is an accomplished PhD nurse practitioner. He received his MD degree from Indiana University where he worked with Dr. Robert Baehner and Dr. Laurence Boxer to study macrophages. During medical school, he published his first paper (as first author) in Nature, a journal which he now jokingly admits he had never heard of before. He presented this work at his first scientific meeting, the 1976 American Society of Hematology where he met Dr. David G. Nathan, who would become his life-long mentor. Following this auspicious entry into the field of hematology, he completed his residency in pediatrics at Cincinnati Children's Hospital Medical Center, and then moved to Harvard for his fellowship in pediatric hematology oncology where he trained "in reverse" by first setting up his lab at Boston Children's Hospital prior to completion of his clinical training. He worked with Dr. Richard Mulligan at MIT and the Whitehead Institute, and was the lead author on a highly cited paper pioneering the transduction of human hematopoietic stem cells using retroviral vectors (more on that below). He was appointed assistant professor at Harvard Medical School and was a Howard Hughes investigator for 16 years. David was recruited back to leadership positions at Indiana University, Cincinnati Children's Hospital, and then back to Harvard where he has held numerous titles including Chief of the Division of Pediatric Hematology, the Leland Fikes Professor of Pediatrics, Director of Translational Research, Chairman of the Clinical and Translational Research Executive Committee, President of Dana-Farber/Boston Children's Cancer Care, Executive Vice President and Chief Science Officer of Boston Children's Hospital. He has held multiple national and international leadership positions and received many prestigious awards too numerous to list here. David's research spans basic to clinical studies, integrating fundamental scientific insights from his laboratory with clinical translation. His lab has been continuously NIH-funded since 1986. He has published over 424 research articles, reviews, and book chapters, many in the highest impact basic and clinical journals. He holds 60 patents with additional patents under review. His laboratory co-discovered and cloned interleukin-11 (Neumega), and his subsequent studies contributed to FDA approval of this drug, the first approved drug to treat thrombocytopenia. David's basic research on molecular and biochemical studies of hematopoietic stem cells within the bone marrow supporting environment defined Rac GTPases (Rac1 and Rac2 in particular) as key regulatory switches that control stem cell adhesion, migration, and survival/proliferation. David has been a thought-leader in the field of gene therapy throughout his career. His laboratory developed the use of fibronectin in retrovirus-mediated gene transfer, now widely used (as Retronectin) in clinical gene therapy trials and basic research utilizing gene transfer. His seminal work on gene transfer has led to clinical gene therapy trials in Fanconi anemia, chemoresistance of hematopoietic stem cells, and the use of chemoresistance to facilitate dose intensification in high-risk pediatric and adult brain tumors. He has served as sponsor/investigator of multiple gene therapy trials in severe combined immunodeficiency (SCID-X1), Wiskott Aldrich syndrome, childhood cerebral adrenoleukodystrophy (CCALD), sickle cell disease, and chronic granulomatous disease. He was also the coordinating investigator for the pivotal gene therapy trial of Lenti-D modified autologous stem cells (Lenti-D Drug Product) for the treatment of subjects with CCALD leading to FDA approval of SKYSONA (elivaldogene autotemcel), the first stem cell gene therapy approved in the United States for a metabolic disease. Following the identification of BCL11A as a regulator of fetal hemoglobin by Vijay Sankaran in Dr. Stuart Orkin's lab, David developed and licensed an shRNA embedded in microRNA-targeting BCL11A (a "shmiR") for modulation of fetal hemoglobin in sickle cell disease and β-thalassemia. The first in human clinical trials led by David with Erica Esrick validated BCL11A as a molecular target in humans, and have led to a pivotal, multicenter national study for the treatment of sickle cell disease, a ground-breaking milestone for this disease. David is a tireless advocate for pediatric research. He was the Chair/co-organizer of a working group on Pediatric Centers of Research Excellence, which led to national legislation passed in Congress and signed into law for the National Pediatric Research Network Act. To address the need for a national clinical research consortium to study pediatric bone marrow failure, David and I worked together to establish the North American Pediatric Aplastic Anemia Consortium (NAPAAC), which currently includes over 50 pediatric hematology centers. NAPAAC has published numerous research studies and conducted clinical trials for pediatric bone marrow failure. He also co-founded the Transatlantic Gene Therapy Consortium. David is committed to fostering the careers of trainees and faculty at all levels, not only when things are going well but, more importantly, also when they are not. He has appointed numerous accomplished women within our faculty to lead divisional programs and has supported their academic promotion to Professor at Harvard Medical School. His trainees are now leaders in basic science and clinical hematology internationally, and they continue to maintain close ties to David. David's wife Cindy recently retired from her position as the Director of Research Nursing at Boston Children's Hospital. David is the proud father of Dr. Emily Williams, who is a pediatric cardiologist and his son-in-law Dr. Brad Leshnower, section chief of cardiovascular surgery, both at Emory University. He is the doting grandfather of two lively grandsons, Max and Jake. Harking back to his Indiana roots, David maintains a farm replete with horses, donkeys, chickens, and bees, and is an avid equestrian. He regularly brings in delicious fresh eggs and honey from his farm to share with the division. He works tirelessly in the community to maintain trails for bikers, hikers, and horseback riders. The ASPHO Distinguished Career Award honors the tremendous impact of David's contributions to basic research, clinical trials, mentorship, and administrative leadership in pediatric hematology/oncology. It is a pleasure and great privilege to work with David. Please join us in celebrating David's inspiring achievements at the 2024 ASPHO meeting. The authors declare no conflicts of interest.