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2000 Distinguished Clinical Scientist Award

Awarded: Aug 07, 2000

$10.5 million over 5 to 7 years

In 2000, seven outstanding physician-scientists at the mid-career level each received grants of $1.5 million to be used over 5 to 7 years.

Alan D. D'Andrea, M.D.
Professor of Pediatrics
Dana-Farber Cancer Institute 


Alan D. D'Andrea, M.D., is a Professor of Pediatrics at Harvard Medical School and the Ted Williams Senior Investigator at the Dana-Farber Cancer Institute in Boston. Dr. D'Andrea obtained his M.D. from Harvard Medical School in 1983 and completed a Pediatric Residency training at the Children's Hospital of Philadelphia in 1986. He performed his post-doctoral research training at the Whitehead Institute for Biomedical Research in Cambridge, MA. At that time he and his collaborators cloned the gene for the Erythropoietin Receptor, a major regulator of normal human blood cell formation. After completing his research training, Dr. D'Andrea joined the Harvard Medical School Faculty in 1990. Dr. D'Andrea is internationally known for his research in the fields of human blood cell formation and cancer susceptibility. He directs a laboratory at the Dana-Farber Cancer Institute and is the Co-Director of the Clinical Gene Therapy Center at Children's Hospital, Boston. Dr. D'Andrea is the recipient of numerous awards, including the Lucille P. Markey Scholar Award (1990), the American Academy of Pediatrics Award for Excellence in Research (1997), and the Stohlman Scholar Award from the Leukemia and Lymphoma Society (2000). Dr. D'Andrea's basic research on Fanconi Anemia and his contributions in the field of gene therapy were recently featured on CBS Sunday Morning.


Establishment of a Diagnostic & Treatment Center for Fanconi Anemia

Dr. D'Andrea's research focuses on Fanconi Anemia (FA), a hereditary anemia and cancer susceptibility disorder. Children born with FA have birth defects and often develop bone marrow failure (anemia) by their fifth birthday. Many of these children go on to develop leukemia or other cancers. The treatment of choice for FA patients is a bone marrow transplant from a histocompatible sibling donor. Dr. D'Andrea's laboratory focuses on the molecular basis of this disease. His laboratory has determined that three of the FA gene products (proteins) normally associate to activate a fourth FA protein. The function of this fourth FA protein is to accumulate in regions of DNA damage on chromosomes, presumably to aid repair, thereby preventing further genetic mutations and the progression to cancer. Understanding the molecular basis of this disease is likley to lead to a more rational approach to the diagnosis and treatment of FA patients. While FA is a rare disease, acquired mutations in the FA genes may also contribute to the formation of bone marrow failure and cancers in the general (non-FA) patient population. In the course of his research, Dr. D'Andrea has established a Comprehensive Fanconi Anemia Center at Harvard Medical School which is the focus of his Doris Duke Distinguished Clinical Scientist Award. This Center offers a wide range of services to patients and families with Fanconi Anemia.