Fanconi Anemia and Bone Marrow Failure Multidisciplinary Clinic

Our innovative approach

The Dana-Farber/Children's Hospital Cancer Center's Fanconi Anemia and Bone Marrow Failure Multidisciplinary Clinic is focused on understanding why bone marrow failure happens and constantly developing better treatments.

Our researchers include scientists from Children’s Hospital Boston, Dana-Farber Cancer Institute, the Massachusetts Institute of Technology and Brigham and Women's Hospital, and our program is one of the nation's top ranked pediatric research programs for cancers related to bone marrow failure syndromes.

Research & innovations

Dana-Farber/Children's Hospital Cancer Center has a long history of research and innovation in conditions associated with bone marrow failure. In fact, Schwachman-Diamond syndrome and Diamond-Blackfan anemia were first identified here.

Today, our researchers are conducting some of the world's most advanced studies of bone marrow failure syndromes. We believe research into these conditions offers significant opportunities for improving treatment therapies. Studies are currently underway in the following areas:

• In the lab of Alan D'Andrea, MD, researchers are investigating the molecular basis of Fanconi anemia as well as other chromosomal breakage syndromes. They are also exploring targeted therapies to treat Fanconi anemia.
• Studies by David Williams, MD, examine the communication between the bone marrow environment and blood-forming stem cells. In addition, his laboratory has developed new therapies for Fanconi anemia, including the use of gene therapy.
• In the lab of Benjamin Ebert, MD, researchers study both congenital and neoplastic causes of bone marrow failure, including myelodysplastic syndrome and Diamond-Blackfan Anemia.
• The lab of Harvey Lodish, MD, is a leading research center for the study of hematopoiesis, cell signaling, and adipocyte biology.
• Colin Sieff, MB, BCh, studies the families of many Diamond-Blackfan anemia (DBA) patients to uncover new genes that cause the disease.
• Studying blood cell formation in zebra fish, researchers in the lab of Leonard Zon, MD, have gained important insight into the biology of blood-forming stem cells and the genetic factors regulating blood cell development.