Pioneering Hematologist to Receive 2018 Nemmers Prize in Medical Science

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Stuart H. Orkin, MD, an investigator of the Howard Hughes Medical Institute at Boston Children’s Hospital and Dana-Farber Cancer Institute and professor at Harvard Medical School known for his landmark discoveries into blood cell development and the genetic basis of blood disorders, is the recipient of the 2018 Mechthild Esser Nemmers Prize in Medical Science at Northwestern University.

The Mechthild Esser Nemmers Prize in Medical Science, which carries a $200,000 stipend, was made possible by a generous gift to Northwestern by the late Erwin Esser Nemmers and the late Frederic Esser Nemmers. One of five Nemmers Prizes awarded by the University, it is given to a physician-scientist whose body of research exhibits outstanding achievement in their discipline as demonstrated by works of lasting significance. A jury of distinguished scientists from around the country made the final selection.

The disciplinary-specific Nemmers Prizes are given every other year, and carry some of the largest monetary stipends in their fields. Prizes also are awarded in earth sciences, economics, mathematics and music composition. Combined, the five Nemmers Prizes winners receive $900,000.

Orkin, the David G. Nathan Distinguished Professor of Pediatrics at Harvard Medical School, has devoted his career to illuminating the development and function of the blood system, the control of stem cells and the molecular basis of inherited blood disorders.

“The Nemmers Prize in Medical Science affirms my conviction that rigorous science fuels discovery and ultimately leads to transformative advances in our understanding and treatment of devastating diseases,” Orkin said. “I am very gratified, thrilled, honored and humbled by this special recognition. My research is not mine alone, but rather a group endeavor that has benefited from the dedication of many extraordinary trainees and colleagues and from unwavering institutional support.”

In connection with this award, Orkin will deliver a public lecture and participate in other scholarly activities at Feinberg in the coming year.

“Stuart is a pioneering physician-scientist whose discoveries have fundamentally transformed our understanding of hematology and blood diseases,” said Eric G. Neilson, MD, vice president for Medical Affairs and Lewis Landsberg Dean. “We are honored to award him the Nemmers Prize in Medical Science, which rightly recognizes his deep and lasting contributions to improving the health of humankind.”

Orkin’s Accomplishments

Orkin’s research has led to a number of breakthroughs that have significantly advanced the field of hematology. Early in his career, Orkin discovered the mutations responsible for beta-thalassemia, an inherited blood disorder that reduces the production of hemoglobin. The research, published in Nature, provided a foundation for better diagnosis and treatment of the disease. In addition, Orkin and his collaborators were the first to use positional cloning to identify a gene for a human disease, in this case, X-linked chronic granulomatous disease (X-CGD).

Orkin has also had a transformative impact on the understanding of hematopoiesis, or normal blood cell development. He identified the master transcriptional regulator of the process, called GATA-1, as well as many other transcription factors critical for blood cell development.

More recently, in research published in Science and Nature, Orkin’s laboratory characterized the molecular switch from fetal to adult hemoglobin, solving a long-held problem in the field. The team identified the BCL11A gene as a major regulator of fetal hemoglobin levels, and demonstrated the potential of targeting the gene for the treatment for sickle cell anemia and beta-thalassemia.

In total, Orkin has authored more than 450 peer-reviewed publications spanning the fields of hematology, human genetics and stem cell biology.

Orkin is a member of the National Academy of Medicine (formerly the Institute of Medicine), the National Academy of Sciences (NAS), the American Academy of Arts & Sciences, and the American Philosophical Society, among numerous other professional organizations and committees. His many awards and honors include the Jessie Stevenson Kovalenko Medal from the NAS; the William Dameshek Prize from the American Society of Hematology; the Lifetime Impact Award from Boston Children’s Hospital; the William Allan Award, the highest honor given by the American Society for Human Genetics; and the George M. Kober Medal of the Association of American Physicians.

Orkin received his medical degree from Harvard Medical School in 1972, after earning a bachelor of science at Massachusetts Institute of Technology. He completed clinical training in pediatrics and hematology/oncology at Boston Children’s Hospital and the Dana-Farber Cancer Institute, as well as postdoctoral research at the National Institutes of Health.

Previously, Orkin served as chairman of the Department of Pediatric Oncology at the Dana-Farber Cancer Institute and associate chief of the Division of Hematology/Oncology at Boston Children’s Hospital.

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