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Groundbreaking Researchers Help Rewrite the Hematology Script

December 9, 2024

Drs. Douglas Cines and Katherine High Receive 2024 Henry M. Stratton Medal 

Named after the late cofounder of the medical publishing house that first published ASH’s flagship journal, Blood, the Henry M. Stratton Medal honors senior investigators whose contributions to basic and clinical/translational hematology research are well-recognized and have taken place over a period of several years. 

Douglas Cines, MD, Stratton Medal for Basic Science 

“I greatly appreciate that my work has been recognized as deserving by colleagues in a field with so many accomplished scientists. Of course, none of my work was done alone. I needed the support of talented colleagues, technologists, and willing patients — and the support and encouragement of my family.” 

Dr. Cines, professor of pathology and laboratory medicine at the University of Pennsylvania Perelman School of Medicine, is being honored for more than 40 years of research discoveries that have led to significant increases in the understanding and treatment of thrombocytopenic disorders, including immune thrombocytopenia, heparin-induced thrombocytopenia (HIT), and thrombocytopenia in pregnancy. Dr. Cines’ main research interest is immunothrombosis, or how the immune system and coagulation interact. He was instrumental in identifying the role of endothelial cells as targets of immune injury in lupus, HIT, and antiphospholipid syndrome (APS), thereby providing a rationale for the connection of these disorders to thrombosis. 

A graduate of the New York University School of Medicine, Dr. Cines completed his internship and residency at N.C. Memorial Hospital in Chapel Hill and his fellowship in hematology-oncology at the Hospital of the University of Pennsylvania.  

“From a research point of view, during my internship and residency, I found myself intrigued with the question of why some otherwise healthy individuals suddenly develop antibodies to platelets but only their platelets, while others develop antibodies only to red blood cells or only to ADAMTS13, the enzyme that’s targeted in thrombotic thrombocytopenic purpura,” Dr. Cines recalled. “From a clinical point of view, I found the breadth of disorders challenging. New treatments for leukemia and lymphomas and bone marrow transplantation were being introduced that were offering hope to our patients.” 

Over the years, Dr. Cines has held many faculty positions at the University of Pennsylvania, including director of the Hematology Laboratory and the Coagulation Laboratory, co-vice chair of the Department of Pathology’s Division of Laboratory Medicine, and both vice chair for academics and director of faculty development in the Department of Pathology and Laboratory Medicine. 

In reflecting upon what he considers his most notable research accomplishment, Dr. Cines notes that the starting point was his interest in why some clinical disorders associated with platelet antibodies lead to thrombocytopenia and bleeding, while others are associated with thrombosis.  

“I wanted to investigate the hypothesis that one difference might reside in the latter set of disorders being caused by the antibodies or antigen-antibody complexes that damage the endothelium,” he explained. “Techniques to culture human endothelium from umbilical cords had recently been developed, allowing this hypothesis to be addressed. We identified endothelial-cell reactive antibodies in lupus, HIT, and APS that promote clotting and inhibit clot lysis and extended the question to whether antibodies to trophoblasts that comprise much of the placenta might also be targets of antibody injury and contribute to pregnancy loss.” 

In addition to his celebrated research accomplishments, Dr. Cines is an esteemed mentor and trainer of clinical and basic investigators, who themselves have gone on to become leaders in the fields of hemostasis, platelet biology, immunohematology, immunology, transfusion, and maternal-fetal medicine.  

“The fact that blood flows throughout the body gives you the opportunity to work with patients with disorders affecting multiple organ systems and their health care providers,” he said. “Knowledge about hematologic conditions is expanding so rapidly that it is a welcome challenge to keep up to date. New diagnostic platforms, tests, and therapies are being introduced that change your practice over and over again.” 

Fun Fact: “I’m a diligent — and unaccomplished — student of classical guitar and the mandolin. I only play in my soundproof basement.” 

Katherine High, MD, Stratton Medal for Translational/Clinical Science 

“During my 40 years of practice, it's been extraordinary to see how many things in the hematology textbook have been completely rewritten in terms of improved understanding of molecular genetics and — for many diseases — much better treatment.” 

Dr. High, emerita professor of hematology at the University of Pennsylvania Perelman School of Medicine and a visiting professor at Rockefeller University, is being recognized for spearheading the development of a gene therapy for hemophilia B, approved earlier this year by the U.S. Food and Drug Administration (FDA), the European Medicines Agency, and Health Canada. This success follows more than two decades of research during which she contributed to the understanding of DNA defects in hemophilia B, factor VII, and factor X, as well as performed several first-of-their-kind clinical trials in gene therapy.  

After graduating with a bachelor's degree in chemistry from Harvard, Dr. High attended medical school at the University of North Carolina. Although briefly leaving the program to pursue a master’s in chemistry, she ultimately returned to medical school and discovered the roots of her lifelong passion.  

“I did an internal medicine residency, and partway through I had to decide on a subspecialty,” she recounted. “I wanted something that would make use of my background in chemistry. So, I thought about which patients had been the most interesting, and it was definitely the hematology patients.” 

As a hematology fellow at Yale University School of Medicine, Dr. High was dismayed to witness the intense suffering experienced by individuals with sickle cell disease, and how little could be done at the time to help them. Since then, she has been awed by the strides in treatment. 

“I had the privilege of talking with one of the men who enrolled on the trial for gene editing for sickle cell disease,” she said. “And it was amazing to see, if you look at the data, how all of these painful crises that characterize the disease just go away.” 

Dr. High spent seven years as a professor in UNC’s Departments of Medicine and Pathology, while also serving as director of the university’s Clinical Coagulation Laboratory. She subsequently relocated to the University of Pennsylvania, where she served as William H. Bennett Professor of Pediatrics for more than a decade. During this time, she also held several key positions at the Children’s Hospital of Philadelphia, including director of the Hematology and Coagulation Laboratories, director of research in the Hematology Division, founding director of the Center for Cellular and Molecular Therapeutics and Investigator, Howard Hughes Medical Institute. 

In 2014, Dr. High co-founded Spark Therapeutics, serving as the company’s president and chief scientific officer through 2020. She has been elected to the National Academy of Sciences, the National Academy of Medicine, and the American Academy of Arts and Sciences. Earlier this year, she received the American Society for Gene and Cell Therapy’s Founders Award for her “extraordinary contributions to the field.” 

Dr. High’s research interests also extend to inherited blindness. She played a key role in developing a first-ever gene therapy to restore vision loss in patients with inherited blindness, which was approved by the FDA in 2017. 

“The more you work on something that you are passionate about, the more it becomes a part of who you are,” said Dr. High. “Even if you feel you don’t have the background for a particular problem, that’s not important. Figure it out.” 

Fun Fact: “I have three children, and my second daughter, Sarah Steele, is a successful actor on both stage and screen. She costarred in the film Spanglish with Adam Sandler and was a regular on the TV series The Good Wife.” 

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