New Hope for Treating a Rare Genetic Disease Before Birth

Stanford Medicine scientists are opening a clinical trial for prenatal treatment of a rare blood disorder called Fanconi anemia. The study will test stem cell transplants performed before birth, using healthy blood-forming stem cells donated by the mother.

Evidence from preclinical studies in animals suggests that these transplants may be able to treat Fanconi anemia without any of the chemotherapy, radiation or immune suppression that are needed for typical stem cell transplants.

“The research gives us real reason to believe prenatal transplants would stabilize the bone marrow in these patients and prevent the need for subsequent therapies,” said Stanford Medicine pediatric hematologist Agnieszka Czechowicz, MD, PhD, who is leading the new study.

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