[Agnieszka Czechowicz] is now an assistant professor of pediatrics at the Stanford University School of Medicine. In her lab research, her medical practice, and her work as an entrepreneur, she focuses on people who suffer from severe diseases like bone marrow failure or acute myeloid leukemia, who can’t produce blood or immune cells the way they are supposed to. The treatment approach she’s been working on for most of her career could represent a new way to help them.
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Could you erase the defective cells using an antibody, instead of chemo? An antibody might be able to target a patient’s stem cells more specifically, with less toxicity for the whole body.
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They found the antibody they were looking for. It attaches to a receptor called CD117 on the surface of blood-forming stem cells. Normally, a different molecule binds to this receptor and tells the stem cells to stay alive, [pathologist and developmental biologist Irving] Weissman explains. But when the antibody binds to the receptor instead, blocking the “stay alive!” signal, the stem cells die.
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Earlier this year, Czechowicz and colleagues got FDA approval to begin a clinical trial of the anti-CD117 antibody in people with Fanconi anemia.
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