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.

“Specifically, we could eliminate the use of radiation and genotoxic chemotherapy called busulfan, with exceptional outcomes.”

In the trial, published in Nature Medicine, the antibody used in combination with other drugs enabled transplants for three children with Fanconi anemia. All three have completed two years of follow-up and are doing well.