The Precision Revolution

Dr. Bertaina pioneered alpha/beta T-cell depletion — a technique that removes the specific immune cells responsible for graft-versus-host disease, making it possible to transplant stem cells from a partially matched donor, usually a parent. That foundational approach has opened two distinct frontiers: a dual immune/solid organ transplant (DISOT) in which a stem cell transplant from a parent is followed by a kidney transplant from that same donor, eliminating the need for lifelong immunosuppression — a procedure performed at only one hospital in the world, Lucile Packard Children’s Hospital Stanford — and a clinical trial combining her transplant technique with T-allo10, a cell therapy designed to strengthen immune recovery post-transplant, in which patients have achieved 100% two-year leukemia-free survival. She is now working to extend her immune tolerance approach to heart transplants. 

Dr. Aghaeepour is harnessing AI to predict risks, prevent complications, and individualize care for mothers and babies from the very start of life. His AI platform—trained on nearly 80,000 nutrition prescriptions—is tackling what he calls the single largest source of medical error in neonatal care worldwide, while separate work has identified biomarkers that predict pre-eclampsia months before symptoms emerge. 

More than 90% of children diagnosed with acute lymphoblastic leukemia are cured today—but for the 10% whose disease returns, roughly half do not survive five years past relapse. Using single-cell analysis, Dr. Davis is identifying the precise cellular triggers behind treatment resistance, with the goal of predicting which children are at risk before relapse occurs. 

Dr. Mackall’s answer to childhood cancer, built over four decades, is to turn the body’s own immune system into a targeted weapon. Her CAR T-cell therapies have pushed remission rates for relapsed pediatric leukemia from 50% to over 70% and produced outcomes once unimaginable in diffuse intrinsic pontine glioma (DIPG). 

One in 100 children is born with congenital heart disease, and existing surgical techniques are reaching their limits. Dr. Ma is partnering with Stanford bioengineers to bring computational modeling to the operating room — designing the optimal repair for each child’s unique heart digitally before making a single incision — work that has already saved lives. 

Dr. Monje’s pioneering research revealed that gliomas hijack the brain’s own electrical circuitry to fuel their growth—helping to open an entirely new field, cancer neuroscience. In partnership with Dr. Mackall, she developed a CAR T-cell therapy for DIPG now in clinical trials, with one patient already achieving a complete response lasting four years. This is a remarkable milestone for a cancer once considered universally fatal. For this body of work, she was recently awarded the Brain Prize, widely regarded as one of the most prestigious honors in neuroscience.

Dr. Porteus has spent his career building the science behind gene editing for children’s blood diseases—work that culminated in December 2023 when the FDA approved Casgevy, the first CRISPR-based therapy in history. He is now advancing a next-generation approach that precisely corrects the sickle cell mutation itself, with early clinical results that are promising.

Modern medicine can now save babies born as early as 22 weeks—but up to 90% of the youngest survivors face significant neurodevelopmental impairment. Using brain organoids, Dr. Anca Pasca is testing compounds that may prevent brain damage in premature babies before it begins and establishing the world’s first bank of fetal-type red blood cells from donated cord blood. 

Dr. Sergiu Pasca developed brain assembloids that replicate living brain tissue with remarkable fidelity, allowing researchers to watch circuits form and malfunction and to test therapies on patient-specific tissue. A first-ever clinical trial for Timothy syndrome, a rare genetic disorder causing seizures and heart arrhythmia, is now emerging directly from his lab. 

Dr. Skylar-Scott is working to fully 3D-printed human heart, built from a patient’s own stem cells, to eliminate the need for a donor heart entirely. In a significant 2025 development, his lab developed tools to print the intricate vascular networks that keep organs alive—up to 200 times faster than previous methods—and his team is now racing toward a first-in-animal heart valve implant.