The objective of this proposal is to conduct a pilot study of metformin to treat FA.
This pilot study will explore whether Metformin is safe and efficacious in improving hematopoiesis in patients with Fanconi Anemia. We will focus on pediatric and young adult patients with FA to determine safety, tolerability, and preliminary efficacy of Metformin in Fanconi Anemia patients. This study will also integrate biological correlative studies to glean mechanistic insights which may inform future studies. The anticipate enrolling 20 patients, with a study duration of 2 years.
Researchers: Akiko Shimamura