Arthur Nienhuis, MD, at the ASH Annual Meeting in 2009

Arthur Nienhuis, MD, at the ASH Annual Meeting in 2009

Dr. Arthur Nienhuis, physician-scientist, past National Institutes of Health (NIH) branch chief, and director of St. Jude Children’s Research Hospital, as well as editor-in-chief of Blood and past president of ASH (1994), died February 3, 2021. Throughout his remarkable career, Art received numerous professional honors, including induction into the National Academy of Medicine. Art’s legacy is unparalleled. His career exemplified the molecular revolution in understanding and treating blood diseases. He was dedicated to promoting hematology as a discipline, and he was an extraordinary mentor to generations of hematologists.

Art was born in Michigan into a family of educators. He attended Cornell College before moving to Los Angeles to receive his MD from the University of California, Los Angeles. He completed an internship and a year of residency in internal medicine at Massachusetts General Hospital in Boston before arriving at NIH in 1970 and immersing himself in the application of groundbreaking molecular tools to understand globin gene expression and hemoglobin switching. Art completed fellowship training in pediatric hematology at Boston Children’s Hospital and then returned to NIH to set up his own research program within the National Heart, Lung, and Blood Institute, where he soon became chief of a newly created Clinical Hematology Branch.

Over the next 16 years, he developed an extraordinarily productive bench-to-bedside research program, making many seminal discoveries and beginning his legacy of mentoring young hematologists. Art was among the first to recognize that reactivation of fetal hemoglobin expression could potentially be used to treat sickle cell disease (SCD) and thalassemia. Art, together with collaborators including Paul Heller, Joe DeSimone, Alan Schechter, and George Stamatoyannopoulos, and then trainees Tim Ley and Griffin Rodgers, led a series of clinical trials at NIH demonstrating that fetal hemoglobin expression could be reactivated in patients with thalassemia and SCD, first with 5-azacytidine and subsequently with hydroxyurea, thus taking the first steps toward the effective pharmacologic treatment of SCD. Additionally, Art made significant contributions to iron chelation therapies for the hemoglobinopathies, greatly extending the lifespan of patients with transfusion-dependent anemias. Toward the end of the 1980s, Art began to focus on gene therapy as a curative approach to blood diseases, carrying out impactful basic and early clinical studies using engineered viral vectors to modify hematopoietic stem cells for the treatment of blood disorders.

In 1993, Art left NIH to become the director and chief executive officer of St. Jude Children’s Research Hospital in Memphis. He was a visionary and effective leader of this world-renowned pediatric hospital and research institution, while continuing his own investigations, expanding his focus to include gene therapy for hemophilia. Art stepped down from the directorship of St. Jude in 2004, and until his retirement in 2016 he focused on research, leading the first successful trial of gene therapy for a bleeding disorder, specifically hemophilia B.

Art believed strongly in all aspects of ASH’s mission — supporting research, clinical care, training, and advocacy focused on blood diseases. Art was a strong and thoughtful leader during a period of incredible growth for both ASH and Blood. As editor-in-chief of Blood from 1987 to 1992, he spearheaded the transition of the journal to ASH ownership, accelerating the journal’s impact and centrality to ASH. He served as ASH President in 1994, overseeing substantial growth of the ASH annual meeting. He was a founder and president of the American Society of Gene and Cell Therapy (ASGCT) and modeled its structure on his experiences leading ASH. For these efforts and many more, Art was awarded the Stratton Medal by ASH in 1998.

Equally significant were Art’s contributions to hematology as a mentor. He welcomed clinicians with little scientific background into his laboratory, molding them into physician-scientists proud to carry on his legacy. His list of trainees is extraordinary, including two ASH and ASGCT Presidents, editors-in-chief of Blood and Seminars in Hematology, the director of the National Institute of Diabetes and Digestive and Kidney Diseases, heads of at least four major research institutions or hospitals, more than 13 cancer center directors or department chairs, and many others in key leadership positions around the world. An early trainee, 2000 ASH President Dr. Ed Benz recalls that, “Art’s influence on my life and the lives of so many colleagues went beyond his brilliance as a scientist, incredible compassion as a physician, and skills as a teacher. He was a fabulous and caring mentor who was in the trenches with us at every moment, sharing our excitement about the projects, our struggles in the lab, and our glee when good results rolled in.” 1993 ASH President Dr. Frank Bunn who spent a sabbatical in Art’s laboratory learning molecular biology said, “He was remarkably generous with his time and gave me one-on-one tutoring, details of which I remember to this day.” The authors of this obituary can testify that Art truly served as a “sponsor,” giving us visibility in the hematology community and unwavering advice and support, even after completion of training. Current NIH Director Dr. Francis Collins said, “He inspired many scientists who were just starting out. Forty years ago, I was one of them.” Honoring these efforts and many more, Art received the ASH Mentor Award in 2009.

Finally, Art was a wonderful colleague and friend. Many stressed how rare his loyalty and basic “niceness” were, given his accomplishments and ambitions. 1986 ASH President David Nathan, who supervised Art at Boston Children’s and remained a collaborator, competitor, and colleague for the rest of their careers, summed it up perfectly saying, “Arthur Nienhuis was a superb doctor, a brilliant and far-sighted scientist, a total gentleman, and a great leader both at NIH and at St. Jude. I dearly loved him and am intensely proud that I had a small role in his training.”

Art is survived by his wife, Corinne; his children, Carol, Craig, Kevin, and Heather; multiple grandchildren; and siblings Marilyn, Wilma, Robert, and Jan. He was predeceased by his wife Sheryl and his sister Dorothy. Speaking for Art’s trainees, collaborators, and colleagues, we can attest that Art Nienhuis will be deeply missed.