Research & Innovation
At Dana-Farber/Children's Hospital Cancer Center, our care is informed by our research. We have a long history of research and innovation in conditions associated with bone marrow failure, including Fanconi anemia in children.
- David Williams, MD, chief of the Division of Hematology/Oncology, is studying the communication between hematopoietic stem cells and the bone marrow in which they sit, as well as developing a new gene therapy to treat FA.
- Williams is also overseeing a stem cell transplant clinical trial for FA patients that avoids exposure to radiation during induction therapy, hopefully reducing the risk of secondary cancers in FA patients.
- Colin Sieff, MB BCh, is evaluating whether an androgen called danazol is safe treatment for Fanconi anemia. This clinical trial is open to new patients.
- Researchers at DF/CHCC are also developing a new diagnostic blood test that compliments the current DEB test.
For more information about current research, visit our Fanconi Anemia & Bone Marrow Failure Multidisciplinary Clinic.
Stem cells teach lessons about blood diseases and more
Researchers George Daley, MD, PhD, and David Williams, MD, are using embryonic stem cells (ES cells) to model Fanconi anemia. Children with FA often don’t show low blood counts until about age 7. However, ES cell studies have revealed that the disease may originate before birth, during the beginnings of blood formation.
In addition, these researchers are using new methods to create blood stem cells from skin fibroblasts obtained from FA patients. These “reprogrammed” cells can be genetically corrected. They hope this approach will lead to new treatments for the aplastic anemia that occurs with FA.
This research suggests that children with FA are born with only a small number of blood stem cells. These can sustain the blood for some time, but eventually the inability to repair damaged chromosomes (a hallmark of FA) kills them off. Researchers hope that ES cells can be used to test possible drugs that might prevent marrow loss.