Dr. Leonard Zon, a distinguished hematologist and stem cell researcher, has been awarded the 2026 March of Dimes Richard B Johnston Jr, MD Prize for his groundbreaking contributions to developmental biology, particularly in understanding blood development during embryogenesis. His innovative research using the zebrafish model has unraveled critical pathways of blood formation, advancing knowledge of rare hematologic disorders with direct implications for maternal and child health. Dr. Zon’s work has illuminated complexities of genetic blood diseases and has paved the way for novel therapeutic strategies benefiting infants and children worldwide.
At the helm of the Stem Cell Research Program at Boston Children’s Hospital and serving as the Grousbeck Professor of Pediatric Medicine at Harvard Medical School, Dr. Zon has been a pioneer in deploying the zebrafish as a powerful model organism. This diminutive tropical fish, measuring just 1.5 inches and characterized by transparent embryos and prolific reproduction, has enabled detailed observation of vertebrate developmental processes in vivo. These attributes make zebrafish an exceptional system for dissecting genetic and molecular mechanisms underlying human blood disorders and cancers affecting pediatrics.
Dr. Zon’s initial turn toward zebrafish research in the early 1990s was motivated by the urgent need to clarify the embryonic origins of blood cells and discover interventions for genetic blood diseases devastating to neonates. His lab exploited the advantages of zebrafish embryos—which allow direct imaging of developmental processes—to trace hematopoietic pathways, identify novel genes, and elucidate their functions. This work has yielded insight into the molecular orchestration of blood cell formation, critical not only for fundamental biology but also for clinical applications in pregnancy and childhood illnesses.
A seminal achievement of Dr. Zon’s research has been the identification of ferroportin, a gene encoding an iron transporter essential for delivering iron from mother to fetus via the placenta. Iron is indispensable for hemoglobin synthesis, and the discovery of ferroportin clarified mechanisms underlying iron homeostasis in early development. This breakthrough transformed understanding of iron deficiency and anemia in pregnancy—conditions globally recognized for their impact on fetal growth and birth outcomes—thus opening new therapeutic possibilities to prevent preterm birth and low birth weight.
Further advancing the field, Dr. Zona nd his team discovered a growth factor capable of exponentially increasing the population of hematopoietic stem cells, a discovery that has had profound clinical implications. By enhancing stem cell proliferation, the research has directly informed the development of gene therapies for inherited blood diseases such as sickle cell anemia and thalassemia, conditions that afflict thousands of children worldwide. These therapies now leverage expanded stem cell populations harvested from umbilical cord blood, dramatically improving transplant outcomes.
Dr. Zon has not only expanded the breadth of developmental hematology through these discoveries but also through establishing the International Society for Stem Cell Research in 2002, fostering collaborative efforts in regenerative medicine globally. His lab’s revelation in 2007 that prostaglandin E2 stimulates hematopoietic stem cell growth in zebrafish led to translational human trials. These demonstrated a remarkable 400% increase in stem cell engraftment success in leukemia patients undergoing umbilical cord blood transplants, firmly bridging bench science with clinical innovation.
The influence of prostaglandin E2 in stem cell expansion has since become a cornerstone in gene therapy protocols worldwide, particularly for treating congenital blood disorders and primary immunodeficiencies in pediatric populations. Dr. Zon’s zebrafish model continues to serve as an indispensable platform for screening and validating potential treatments before clinical application, enabling more precise and safe therapeutic designs.
Ongoing research in Dr. Zon’s laboratory is pushing boundaries beyond hematology. One promising avenue involves novel therapeutic approaches for Diamond-Blackfan anemia, a rare congenital bone marrow failure syndrome. Zebrafish studies have identified drug candidates now nearing human clinical trial phases, bringing hope for effective treatments to patients suffering from this challenging condition. Additionally, Dr. Zon’s entrepreneurial ventures have spearheaded the development of novel interventions for spinal muscular atrophy, a severe neurodegenerative disease in children, with therapies under active review by regulatory authorities.
Dr. Zon’s legacy exemplifies the power of model organism research to unlock complexities of human biology and translate those insights into tangible medical advancements. The zebrafish’s unique attributes have allowed decades of research revealing fundamental tenets of blood development and disease, from gene discovery to innovative therapeutic methods. His work demonstrates how developmental biology interweaves with genetics, hematology, oncology, and regenerative medicine to address some of the most pressing health challenges affecting vulnerable populations.
Balancing a demanding clinical schedule with prolific research productivity, Dr. Zon embodies the physician-scientist ideal, driving discoveries from the bench to the bedside. His dedication to pediatric hematology and stem cell science continues to inspire a generation of scientists and clinicians, underscoring the symbiotic relationship between foundational research and translational medicine. The March of Dimes Prize rightly honors these monumental achievements, celebrating scientific breakthroughs that profoundly improve the lives of mothers and children worldwide.
The recognition of Dr. Leonard Zon with the 2026 March of Dimes Richard B Johnston Jr, MD Prize highlights the transformative impact of developmental biology on understanding human health. His pioneering zebrafish research has not only elucidated critical molecular pathways of blood formation during embryonic life but has also led to groundbreaking therapies for devastating genetic blood diseases and complicated pregnancy conditions. This accolade underscores the vital role of basic science in driving clinical innovation and offers a beacon of hope for continued advances in maternal and infant health.
As Dr. Zon remarked, his early experiences with leukemia patients fueled a lifelong quest to decode the biology of blood and conquer inherited disorders. This journey, powered by the humble zebrafish, has yielded priceless insights and therapeutic breakthroughs that continue to save lives and enhance quality of life for the smallest and most vulnerable patients. The honor bestowed upon him serves not only as recognition of past accomplishments but also as motivation to further explore the mysteries of developmental biology and their applications to human medicine.
Subject of Research: Developmental biology of blood formation using zebrafish as a model; genetic blood diseases; stem cell therapies for pediatric hematologic conditions.
Article Title: Renowned Developmental Biologist Dr. Leonard Zon Wins 2026 March of Dimes Prize for Pioneering Work in Blood Development and Therapy
News Publication Date: 2026
Web References:
https://www.marchofdimes.org/about/news/renowned-developmental-biologist-wins-march-dimes-prize
Keywords: Developmental biology, zebrafish, stem cell research, leukemia, blood diseases, hematopoiesis, iron transporter gene, ferroportin, prostaglandin E2, gene therapy, sickle cell anemia, Diamond-Blackfan anemia, spinal muscular atrophy
