Revolutionary Antibody Therapy eliminates Need for Chemotherapy and Radiation Before Stem Cell Transplants
Table of Contents
A groundbreaking new antibody therapy developed at Stanford Medicine offers a perhaps life-saving option to traditional, toxic conditioning treatments-chemotherapy and radiation-required before stem cell transplants, according to results from a phase 1 clinical trial.
A new era in stem cell transplantation is dawning, offering hope to patients with debilitating genetic disorders like Fanconi anemia and potentially expanding access to this critical treatment for a wider range of conditions. researchers have demonstrated that an antibody, known as briquilimab, can safely prepare patients for stem cell transplants without the devastating side effects associated with conventional methods.
A lifeline for Fanconi Anemia Patients
The study, published in Nature medicine, focused on individuals with Fanconi anemia, a rare inherited disorder that compromises the body’s ability to repair DNA, leading to bone marrow failure and increased risk of cancer. Traditional stem cell transplants, while potentially curative, are exceptionally dangerous for these patients due to their heightened sensitivity to the damaging effects of chemotherapy and radiation.
“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,” explained Agnieszka Czechowicz, MD, PhD, assistant professor of pediatrics and co-senior author of the study. “Specifically, we were able to deplete their bodies of potentially dangerous beta T-cells, which can cause graft-versus-host disease, a potentially fatal complication.This method, pioneered by Alice Bertaina, MD, PhD, enables safe transplants from half-matched donors, including parents.”
“we are expanding the donors for stem cell transplantation in a major way, so every patient who needs a transplant can get one,” stated Rajni Agarwal, MD, professor of pediatric stem cell transplantation and co-first author.
Ryder’s Story: A Beacon of Hope
Eleven-year-old Ryder Baker of seguin, Texas, was the first patient to receive the innovative treatment, undergoing a transplant at Lucile Packard Children’s Hospital Stanford in early 2022. Today, Ryder is thriving.
“He was so tired,he didn’t have stamina.It’s fully different now,” saeid his mother, andrea Reiley.”His Fanconi anemia doesn’t slow him down like it used to.” Ryder has since finished fifth grade, participates in sports, and was recently recognized with an “Up and Coming Player” award from his school soccer team.
Promising Results and Future Directions
The phase 1 trial involved three children with Fanconi anemia,all of whom have been followed for two years and are doing well. Researchers are optimistic that this approach will substantially reduce the risk of secondary cancers, which affect nearly all Fanconi anemia patients by age 40.
“If they don’t get a transplant in time, Fanconi anemia patients’ bodies eventually will not make blood, so they die of bleeding or infections,” Agarwal explained. “The reason I am so excited about this trial is that it is indeed a novel approach to help these patients, who are very vulnerable.”
the team is now leading a phase 2 clinical trial involving more children with Fanconi anemia and exploring the potential of this antibody-based approach for other rare bone marrow failure disorders, such as Diamond-Blackfan anemia. Researchers are also investigating whether the antibody coudl benefit elderly cancer patients who are unable to tolerate traditional conditioning regimens.
“That population is often at a disadvantage,” Agarwal said. “It may provide us with a way to treat them with less intensity so it’s possible for them to get a transplant.”
The research was supported by funding from anonymous donors, the California institute of Regenerative Medicine, and the Fanconi Cancer Foundation. Jasper Therapeutics provided the antibody briquilimab, and the Stanford Clinical Trial Program supported the study’s implementation.
