Gene Therapy Reverses Progression of Bone Marrow Failure in Small Fanconi Anemia Trial
December 10, 2024
Treatment with autologous gene-corrected hematopoietic stem cells led to sustained engraftment and reversed progression of bone marrow failure (BMF) without long-term side effects in patients with Fanconi anemia. Investigators published the findings from the small, phase 1/2 trial Dec. 3 in
The Lancet.
Investigators mobilized peripheral blood CD34-positive cells from nine patients with Fanconi anemia-A in the early stages of BMF. The cells were transduced with a therapeutic FANCA-encoding lentiviral vector and re-infused without any cytotoxic conditioning treatment.
Among eight evaluable patients, five achieved sustained engraftment of gene-corrected cells without requiring genotoxic conditioning. The therapy was well-tolerated, with most adverse events being non-serious and unrelated to treatment. Serious adverse events were rare and resolved without long-term effects.
Fanconi anemia is a rare genetic disorder that impairs the bone marrow's ability to produce blood cells, often resulting in severe anemia and bone marrow failure. Many patients rely on frequent blood transfusions to address critically low levels of red blood cells and platelets.
