Gene Replacement Therapy
To avoid the serious toxicities that are still possible with transplantation of bone marrow or stem cells, a new alternative known as Gene Replacement Therapy has been developed.
In this therapy, some of the FA patient’s own marrow or stem cells are collected, a normal (non-mutated) FA gene is transferred into these cells. The “gene-corrected” cells are then transplanted back to the FA patient. Because these cells come from the patient, the risk of an immune reaction, like GVHD or graft rejection, is lower. Also, the corrected cells have a natural advantage over non-corrected cells to survive once the transplant is complete. The hope is that this survival advantage will allow corrected cells to continue supporting the bone marrow and blood system of the patient and decrease the risk of aplastic anemia relapse.
In late 2011, Seattle Cancer Care Alliance initiated one of the first gene therapy clinical trials for FA patients who meet certain qualifications. Patients wanting more details about the study can learn more in Fanconi Anemia: Our Clinical Studies.