Fanconi anemia future or investigational therapies
Therapies under development
● Gene therapy – Gene therapy has the potential to improve bone marrow function in individuals with FA since the origin of bone marrow failure is deficiency of an FA gene function. Gene-corrected CD34+ stem cells from FA patients have been engrafted in immune-deficient mice, but successful clinical applications of gene therapy for FA have not yet been demonstrated.[1]
● Metformin – In a mouse model of FA (FANCD2 gene knockout), metformin produced modest increases in white blood cell (WBC) counts, hemoglobin levels, and platelet counts.[2] There was also reduced p53-dependent tumor formation and a suggestion of decreased susceptibility to DNA damage. Metformin has not been evaluated in patients with FA.
References
- ↑ Río P, Navarro S, Guenechea G, Sánchez-Domínguez R, Lamana ML, Yañez R; et al. (2017). "Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients". Blood. 130 (13): 1535–1542. doi:10.1182/blood-2017-03-774174. PMID 28801449.
- ↑ Zhang QS, Tang W, Deater M, Phan N, Marcogliese AN, Li H; et al. (2016). "Metformin improves defective hematopoiesis and delays tumor formation in Fanconi anemia mice". Blood. 128 (24): 2774–2784. doi:10.1182/blood-2015-11-683490. PMC 5159699. PMID 27756748.