No adult atem cell treatment? Are you sure?
“FANCONIS ANEMIA
Bitan M et al., Fludarabine-based reduced intensity conditioning for stem cell transplantation of fanconi
anemia patients from fully matched related and unrelated donors, Biol Blood Marrow Transplant.
12, 712-718, July 2006
Tan PL et al., Successful engraftment without radiation after fludarabine-based regimen in Fanconi anemia
patients undergoing genotypically identical donor hematopoietic cell transplantation, Pediatr Blood
Cancer, 46, 630-636, May 1, 2006
Kohli-Kumar M et al., Haemopoietic stem/progenitor cell transplant in Fanconi anaemia using HLAmatched
sibling umbilical cord blood cells, British Journal of Haematology 85, 419-422, October
1993”
Indeed, never say never in medicine. However, those citations highlight why adult stem cell or induced pluripotent stem cell therapies are problematic with genetic disorders, and why adult stem cell or induced pluripotent stem cell therapies are so advantageous, i.e. they usually avoid all of the headaches of transplantation medicine which their titles state. You don’t have to worry about using an immunosuppressant like Fludarabine or HLA matching to avoid graft versus host disease or host versus graft disease. Genetic diseases are a different kettle of fish.