Antibody-Based Protocol Enables Safer Stem Cell Transplants Without Chemo or Radiation

Published on Stanford Medicine News

Stanford researchers have successfully used an antibody, briquilimab, to prepare children with Fanconi anemia for stem cell transplants without chemotherapy or radiation. In a phase 1 trial, three patients received donor cells following CD117-targeted antibody conditioning, avoiding the genotoxic toxicity typically seen with traditional regimens. Two years later, all patients show full donor cell engraftment and restored blood production. The approach may expand transplant access to children with fragile immune systems and is now being tested in other bone marrow failure syndromes.

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👉 Stem cell transplant without toxic preparation successfully treats genetic disease

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