Supported Research

Phase 1: Pilot Study of Metformin for Patients with Fanconi Anemia

2018 | Dana-Farber/Boston Children’s Cancer and Blood Disorders Center | Clinical Trial

Amount Funded: $686,848

Low blood counts due to bone marrow failure are a common complication of Fanconi anemia (FA) which affect health and quality of life. Metformin improves blood counts in mice with FA and may protect against DNA damage. Laboratory studies suggest that Metformin may be protective against aldehydes, which are toxic to FA mice and to FA patients. Metformin is an FDA-approved oral medication used for many decades to treat diabetes and insulin resistance. Metformin is currently being studied in other clinical trials as an anti-aging and anti-cancer drug, but it is not known how Metformin will affect people with FA. In this study which is opening at Boston Children’s Hospital, we want to learn whether Metformin improves blood counts in people with FA. You may be eligible for this study if you have FA and low blood counts, are between the ages of 6-35 years and have not had a hematopoietic stem cell transplant (bone marrow transplant). As a participant in this study, you will be provided with Metformin for 6 months. It is not known whether Metformin improves blood counts in patients with Fanconi anemia. Your blood counts, other laboratory tests, and clinical symptoms will be monitored while you are on the study. If you would like to learn more about this study, please call or email co-investigator Dr. Elissa Furutani at (617)-632-1978 or

Researchers: Akiko Shimamura