An antibody treatment developed at Stanford Medicine successfully prepared patients for stem cell transplants without toxic busulfan chemotherapy or radiation, a Phase I clinical trial has shown.

While the researchers tested the protocol on patients with Fanconi anemia, a genetic disease that makes standard stem cell transplant extremely risky, they expect it may also work for patients with other genetic diseases that require stem cell transplants.

“We were able to treat these really fragile patients with a new, innovative regimen that allowed us to reduce the toxicity of the stem cell transplant protocol,” said the study’s co-senior author, Agnieszka Czechowicz, MD, Ph.D., assistant professor of pediatrics.