The David Koch Jr. Foundation recently gifted Stanford Medicine $15 million to launch the Kidney Health Innovation Program. Three champions of pediatric kidney disease healthcare sat down to discuss the advances in the field made possible by the donation. 

Alice Bertaina, M.D., Ph.D., initially caught the interest of the David Koch Jr. Foundation as an investor in Stanford Medicine’s pediatric kidney disease research. In 2017, Bertaina left a successful career as the Head of the Stem Cell Transplant Unit in the Department of Hematology and Oncology at the Bambino Gesù Children’s Hospital in Rome to join Stanford Medicine. 

“I wanted to be surrounded by people who could take my research to the next level,” Bertaina said.

At Bambino Gesù Children’s Hospital in Rome, Bertaina was pioneering a groundbreaking method where a 50% — as opposed to the previously required 100% — genetic match could serve as a stem-cell donor. Bertaina’s approach was about to revolutionize pediatric kidney disease and organ transplantation.

Her first case at Stanford involved a young brother and sister, who had come to Lucile Packard Children’s Hospital Stanford from Alabama seeking treatment for a rare genetic disease called Schimke immune-osseous dysplasia (SIOD), which impacts the kidneys. It was impossible to transplant their kidneys because the siblings’ immune systems were so weak, and it was dangerous to do a stem-cell transplant because they would have to remain on dialysis that could endanger their already weak kidneys. 

Bertaina proposed what the New England Journal of Medicine referred to as the “holy grail” of treatments for patients like this. She hypothesized that sequentially transplanting stem cells and a kidney from the same donor could address both medical issues. Many were skeptical of her method, now termed the dual-immune solid organ transplant (DISOT) approach, but Bertaina proved them wrong. Today, both children are several years out from their DISOT and doing well.

Mary Leonard, M.D., MSCE, the Chair of the Department of Pediatrics at the School of Medicine, described the impact of Bertaina’s treatment. 

“What Alice did for these kids has been nothing short of a miracle,” Leonard said. 

She went on to explain why the David Koch Jr. Foundation’s gift had come at an impactful time in pediatric kidney research. Studies indicate that nearly 1% of all dialysis patients in the U.S. are children, leading drug developers to focus on adult diseases with the hope that medical advancements will eventually trickle down to pediatrics. 

“Kids, though, are not tiny adults,” Leonard said, a motto that inspires her and her colleagues in their push to discover new treatments for pediatric kidney diseases. 

The gift will fund research grants for pediatric kidney disease, including glomerulonephritis, which inflames the filtering units of the kidneys. By expanding research across fields including genetics and biostatics, Leonard hopes that researchers at Stanford can advance new kidney disease treatments, similar to how they developed DISOT. One doctor is trying to make sure that is possible.

Sophia Giang, MD, has been fascinated with immunology and autoimmune disease for a long time. When a family member was diagnosed with lupus nephritis, Giang wanted to do something. 

“[Lupus nephritis] was such a black box to me then, which made me curious, and led me to other pediatric kidney diseases like glomerulonephritis,” she said. 

When Giang arrived at Stanford in 2024, the field of pediatric kidney disease was undergoing a renaissance due to the work of researchers like Bertaina. Giang noticed that glomerulonephritis was an untouched area in pediatrics and wanted to further develop research on the disease. 

“No one had made a mark in the space yet,” Giang said. “I thought I could fill that void.” 

With the David Koch Jr. Foundation’s gift, and Giang’s dedication to the disease, the void will be filled. 

Bertaina, Leonard and Giang aim to continue their work and save many more lifetimes by implementing lab-based methodology into clinical trials. Eventually, they hope to improve the health of kids like these and alleviate the effects of pediatric disease. 

“In pediatrics, you can save a lifetime,” Leonard said.