Stuart H. Orkin, MD, honored with Lifetime Impact Award at Boston Children's Hospital Innovation Summit

BOSTON (November 11, 2015)Stuart H. Orkin, MD, associate chief of hematology/oncology at Boston Children's Hospital and chair of pediatric oncology at Dana-Farber Cancer Institute, was recognized with Boston Children's Hospital's Lifetime Impact Award at the hospital's third annual Global Pediatric Innovation Summit, Taking on Tomorrow (#PedInno15).

The Lifetime Impact Award recognizes a clinician and/or researcher who has delivered or developed practice-changing innovations or discoveries that significantly impact pediatric care. It also provides national and international recognition to an individual who has made extraordinary and sustained leadership contributions throughout his/her career to improve health care in the field of pediatrics. The presentation of the Lifetime Impact Award and companion Rising Star Award in a ceremony at the Seaport World Trade Center on November 10 marked the culmination of a two-day summit that brought together innovators and thought leaders from across the globe to examine clinical, informatics and business opportunities in pediatric innovation.

"Dr. Orkin's contributions to the patients, families and staff from both our hospitals have been immeasurable," said Boston Children's Hospital President and CEO Sandra L. Fenwick. "For all of his dedication to research and care, he has never lost sight of teaching the next generation of researchers and caregivers, and we have all learned so much from him, particularly when it comes to commitment to excellence."

Orkin completed his medical degree at Harvard Medical School in 1972, followed by postdoctoral research at the National Institutes of Health and clinical training in pediatrics and hematology/oncology at Boston Children's Hospital and Dana-Farber Cancer Institute. Guided by his mentor, 2014 Lifetime Impact Award recipient David G. Nathan, MD — he set off on a career dedicated to understanding the molecular drivers of blood cell development (hematopoiesis). In the process, he has consistently led the field of hematology/oncology in bringing new technologies to bear on the study of cancer and disorders of the blood.

For instance, Orkin's was one of the first laboratories to apply molecular biology and DNA sequencing techniques to thalassemia, a blood disorder characterized by defects in genes that provide the instructions for producing hemoglobin (the oxygen-carrying protein in red blood cells). In addition, he has systematically dissected the hematopoietic process, identifying nearly every one of the master genes called transcription factors that regulate the development of every cell type found in the blood.

"Stu has always been in the vanguard when it comes to expanding our understanding of gene regulation, hematopoiesis and how they can go awry to cause blood disorders and leukemias," said David A. Williams, president of Dana-Farber/Boston Children's Cancer and Blood Disorders Center. "We will continue to see the impact of his work as a scientist, a leader and a mentor for years to come."

In recent years, Orkin's laboratory has probed deeply into roles of two molecular switches — a gene called BCL11A and an enhancer that controls its activity — in controlling production of the adult and fetal forms of hemoglobin. Fetal hemoglobin is produced in the womb and for a short period after birth, at which point blood stem cells switch to adult hemoglobin production. Sickle cell anemia (another blood disorder) and thalassemia are both caused by mutations in adult hemoglobin. Orkin and his collaborators are attempting to use gene editing technologies such as CRISPR to manipulate BCL11A's enhancer and force red blood cells to dial down adult hemoglobin production in favor of the fetal form, thereby providing a genetic cure for these blood disorders.

“It’s hard to imagine another person who matches Stu Orkin’s scholarship, innovation, mentorship and vision,” said Dana-Farber Cancer Institute president and CEO Edward J. Benz Jr., MD. “The field of pediatric hematology has evolved into what it is today in large part thanks to his efforts, insights and inspiration. This award honors not only Stu, but the generation of scientists trained under his direction and their efforts to bring their shared vision to clinical fruition.”

Orkin is a member of the National Academy of Sciences, the National Academy of Medicine and the American Academy of Arts and Sciences. He is a fellow of the American Association for the Advancement of Science, an investigator with the Howard Hughes Medical Institute, and the David G. Nathan Professor of Pediatrics at Harvard Medical School.

Irene Sege

About Taking on Tomorrow

Boston Children's Hospital's Global Pediatric Innovation Summit + Awards 2015, Taking on Tomorrow, took place November 9 and 10 at the Seaport World Trade Center in Boston. It provided an engaging forum to build partnerships, accelerate innovations and help reshape the future of pediatric medicine. The dynamic and comprehensive agenda gathered innovators and thought leaders from across the globe to examine clinical, informatics and business opportunities in pediatric innovation.

Taking on Tomorrow featured IBM as its premier sponsor. Additional corporate partners included Philips Healthcare, Genzyme, Claritas Genomics, Deloitte, Verizon, RCN, Alere Analytics, Siemens Healthcare, American Well, Cerner Corporation, Shire, Vertex Pharmaceuticals, Janssen Research & Development, LLC, Alexion, Cisco, Presidio, AstraZeneca and Pfizer's Centers for Therapeutic Innovation (CTI). Summit Association Partners include the Massachusetts Biotechnology Council, Healthcare Information and Management Systems Society (HIMSS), National Organization for Rare Disorders (NORD), Network for Excellence in Health Innovation (NEHI), The Institute for Pediatric Innovation, The Institute for Healthcare Improvement (IHI), The Massachusetts Medical Device Industry Council (MassMEDIC) and Mass Technology Leadership Council (MassTLC). Media partners featured Modern Healthcare, Boston Business Journal, WBUR-90.9 FM (Boston's NPR News Station), MIT Technology Review, Clinical Informatics News, and Bio-IT World.

Follow the Summit online @BCH_Innovation #PedInno15.

About Boston Children’s Hospital

Boston Children’s Hospital is home to the world’s largest research enterprise based at a pediatric medical center, where its discoveries have benefited both children and adults since 1869. More than 1,100 scientists, including seven members of the National Academy of Sciences, 11 members of the Institute of Medicine, and 10 members of the Howard Hughes Medical Institute comprise Boston Children’s research community. Founded as a 20-bed hospital for children, Boston Children’s today is a 404-bed comprehensive center for pediatric and adolescent health care. Boston Children’s is also the primary pediatric teaching affiliate of Harvard Medical School. For more, visit our Answers blog and follow us on our social media channels: @BostonChildrens, @BCH_Innovation, Facebook and YouTube.