Advancing Childhood Cancer Treatment: A Precision Approach from UCSF
A new approach to chemotherapy conditioning before stem cell transplant is dramatically improving survival rates and reducing long-term side effects for children with blood cancers, according to research led by UCSF. Published in Blood Advances and funded by the National Institutes of health (NIH) through the National Cancer Institute (NCI), the advancements mean more children are surviving with minimal lasting impact from their cancer treatment.
For decades, chemotherapy conditioning guidelines used for children were based on adult protocols, relying solely on height and weight. Recognizing the need for improvement, a team led by Dr. charles dvorak developed a personalized conditioning treatment that minimizes chemotherapy dosage and eliminates the need for radiation.
“Optimal chemotherapy conditioning accounts for not only children’s height and weight but also many other personalized factors like immune cell count and kidney function that helps us calculate a precise amount of chemotherapy and eliminate the need for radiation,” explained Dr. Dvorak, lead study author and chief of the UCSF Allergy, Immunology and Bone Marrow Transplantation Division in Pediatrics.
Historically, chemotherapy conditioning for stem cell transplant lacked a strong scientific basis.”It is indeed sad to say, but we were basically guessing at how best to treat kids, while giving them far too much chemo and radiation,” stated Dr. Janel Long-Boyle, UCSF professor of Clinical Pharmacy and co-author of the paper. Driven by her own sister’s experience with leukemia, Dr. Long-Boyle created an algorithm to calculate precise chemotherapy doses, factoring in genetics, sex, race, immune cell count, kidney function, and other individual characteristics alongside height and weight. This precision approach, coupled with the elimination of radiation, helps prevent long-term side effects such as infertility and secondary cancers.
Dr.Long-Boyle’s algorithm is the first globally to demonstrate the benefits of this new conditioning method. “This is a direct example of how research funding goes from bench to bedside, developing treatments in the lab that reach patients. Now the real challenge is shifting clinicians’ mentalities to be open to the new approach,” she added.
UCSF is actively expanding its research partnerships to extend the reach of this innovation. Collaborations are underway with institutions including the university of Utah, children’s Hospital Los Angeles, Children’s National Hospital, and Children’s Wisconsin.
“UCSF’s work created the foundation for clinicians and researchers throughout the country, like me, to deliver highly specialized care to treat children in our communities who have severe blood cancers,” said Dr. Soohee Cho, a pediatric hematologist-oncologist at the University of Utah and a former UCSF Fellow.
Dvorak and Long-Boyle also freely share their clinical expertise with colleagues across the nation, supporting physicians and families in states like Florida, New York, Oklahoma, and Tennessee.
This article is based on a piece originally published September 2, 2025, by the University of California, San Francisco News Center and is republished with permission.
