Leonard I Zon, MD, an internationally recognized researcher in the Department of Stem Cell and Regenerative Biology at Harvard University (HSCRB), has been elected to the National Academy of Sciences for his pioneering work in stem cell biology and cancer genetics. Election to the academy, which was announced May 3, is considered one of the nation’s highest honors for achievements in science.

Dr. Zon is the Grousbeck Professor of Pediatric Medicine at Harvard Medical School, an Investigator with the Howard Hughes Medical Institute, Chair of the Harvard Stem Cell Institute’s Executive Committee, and Director of the Stem Cell Program at Boston Children’s Hospital which he co-founded in 2004.

“As a pioneer in developmental hematopoiesis, Len’s contributions to the field of stem cell biology have been foundational to our understanding of many blood diseases, ” says Paola Arlotta, PhD, HSCRB co-chair. Zon’s critical discoveries uncovering the mechanisms of blood cell production and melanoma emergence now guide the development of several promising therapies for blood disorders and cancer. Throughout his career, Zon’s research has focused on two critical avenues of investigation: identifying the genes that direct stem cell fates and functions, and understanding the mechanisms by which cancer starts.

Fundamental to his work is the use of novel chemical and genetic screens to gain mechanistic, as well as therapeutic, insights into disease. Additionally, Zon’s work was vital in establishing zebrafish as an exemplary genetic model for discovering key genetic regulators of blood cell formation and cancer development.

“Zon’s use of innovative chemogenetic screens, and particularly his application of these to living, developing zebrafish, have yielded otherwise unreachable insights into the biological processes that control stem cells and cancer cells inside the body and set a standard in the field for identifying relevant oncogenic genes and proteins and discovering new disease treatments that appropriately account for the complexity of the in vivo environment,” adds Amy Wagers, PhD, HSCRB co-chair.

Among his lab’s many firsts include the discovery of five new human blood diseases and the development of new compounds to treat them. He and his team also created a powerful technique for visualizing the first cell in an adult zebrafish that causes melanoma leading to a new way to find the cause of the cancer and hopefully stop it before it develops.

One of the leading insights of Zon’s career was the discovery that prostaglandin E2 (PGE2) stimulates the production of blood stem cells in the aorta. “We took this discovery to the clinic and over 100 patients have received this therapy,” says Zon. Today, PGE2 has become a standard method of enhancing engraftment and viral transduction in gene therapy and CRISPR trials.

“We are only at the dawn of stem cell therapy,” said Zon, who also founded the International Society for Stem Cell Research (ISSCR) 20 years ago to advance stem cell biology into clinical practice. “Back then, I predicted we would be treating patients with stem cell derived tissues, and we are.”

Zon received a B.S. in chemistry and natural sciences from Muhlenberg College and an M.D. from Jefferson Medical College. He subsequently did an internal medicine residency at New England Deaconess Hospital and a fellowship in medical oncology at Dana-Farber Cancer Institute. Dr. Zon is the former President of the ISSCR, President of the American Society for Clinical Investigation, and Head of the external investigators of the Zebrafish Genome Institution.

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Photo of Len Zon

Leonard Zon, M.D.

Professor of Stem Cell and Regenerative Biology