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Tanja Gruber, MD, PhD, knows firsthand the power that philanthropy can have in saving lives. She is an expert in leading clinical trials for infants with acute lymphoblastic leukemia (ALL), a disease that is fatal in two-thirds of children under age 1. Using the science that emerged from her lab, Gruber and her fellow researchers identified a promising treatment approach that is now being evaluated in a clinical trial. Early data emerging from the study demonstrates a significant boost in the cure rate for newly diagnosed infants with ALL.
"All of this was possible because of philanthropy. Without investment, we know what's possible, but we can't do it—and that's heartbreaking," she says.
Gruber plans to continue infant ALL research that she started at St. Jude's Children's Hospital at Lucile Packard Children's Hospital and internationally, with the goal of raising the cure rate to 80%. She was recruited to Stanford in 2020 as the new divison chief of Hematology, Oncology, and Stem Cell Transplantation and Regenerative Medicine in the Department of Pediatrics at the School of Medicine, and director of the Bass Center for Childhood Cancer and Blood Diseases at Packard Children's Hospital.
"Our program is about translating science into cures," says Gruber. "We still don't understand what drives cancers, so the research coming out of our laboratories aims to uncover the underlying biology to inform which treatments will improve outcomes. They will need to make the leap from the laboratory to the clinic and bring patients who have poor prognoses to Stanford to receive those new, innovative treatments. It's not enough for us to deliver the standard of care. We need to set the new standards of care. That's really my dream."
Philanthropic giving has fueled Packard Children's Hospital since its inception. A visionary gift of $70 million by David and Lucile Packard in 1986 launched construction of the new hospital, the culmination of Lucile's lifelong dedication to children's health.
Our hospital continues to rely on philanthropic support to recruit specialists; fund research; expand and equip buildings; and, of course, provide care for thousands of children with serious health conditions, including cancer, heart defects, autoimmune diseases, and more.
The key to solving difficult medical problems—whether in children or adults—lies with research, and research is expensive. However, the primary public funder of medical research, the National Institutes of Health, reserves disproportionally less funding for research focused on children. For instance, only 4% of governement funding for cancer research is directed toward pediatrics, according to the Children's Cancer Research Fund.
In addition, the pharmaceutical industry is reluctant to invest in early-stage, high-risk research. Gruber says this has left pediatric oncologists like herself with only a handful of drugs approved for children and many young cancer patients without alternatives if those therapies fail.
Over the years, Packard Children's Hospital and the School of Medicine have recruited some of the leading scientists in the word to find new solutions to the most pressing childhood illnesses. Fortified by a collaborative culture and shared commitment to driving bold ideas, these innovators are making incredible progress. Yet breakthroughs are possible only because of the heroes behind the scenes: donors like you.
Philanthropy fuels cutting-edge patient care in two significant ways:
One of the most powerful tools to recruit prominent faculty is endowed professorships, which provide a permanent source of funding for pediatric leaders who want to pursue high-risk, high-reward research ideas that could lead to life-saving treatments. Professorships reduce faculty members' dependence on clinical revenue and grant funding and free them to spend more time focusing on their research, as well as on training the next generation of physician-scientists.
Unfortunately, cost often stands in the way of progress. Expenses include purchasing drugs, hiring staff, and conducting the trials. "That's where philanthropy can help us bridge the gap and make a life-saving difference," Gruber says.
These are two areas of philanthropy—research and talent—are intertwined. When the hospital has funding to support exciting and promising research trials, innovative scientists want to take part. And when those scientists come, research thrives.
Elaine and John Chambers have made many significant contributions toward talent and research over their 16-year relationship with our hospital. They funded the endowed professorship that helped bring Gruber to Stanford. They also responded to a matching gift opportunity by Jeff Chambers, chair of the hospital board of directors, and his wife, Andi Okamura, to establish an endowed professorship in cancer biology, held by Juilen Sage, PhD. Like Gruber, Sage is focused on developing novel treatments for children with some of the most lethal cancers. His work centers on discovering what supresses tumor growth and what allows cells to proliferate at the molecular level.
John, CEO of J2C Ventures and former CEO of Cisco Systems, explains that he preferes to invest in the "right people and right culture" as a way to solve difficult challenges.
"That's one reason we like Tanja so well. She plays at a different level. She is so humble but wicked-smart," he says. "And what's unique about Packard Children's Hospital is they take on the impossible."
This fall, the Chambers family made another significant gift to support Gruber and her bold vision: to speed the development of new therapies by increasing the number of clinical trials at Packard Children's Hospital and doubling the number of patients enrolled.
"Dr. Gruber let us know clinical trials are the critical part of moving forward. How can you improve a child's health until we have better, safer medicines?" says Elaine, who previously served as board chair of the Lucile Packard Foundation for Children's Health. "As a society, we want the best and latest innovations, but there's no way to have that unless you invest in it."
Clinical trials are essential to bring newer, more effective and safer treatments to children and save lives. To achieve her vision, Gruber plans to build a "dream team" by recruiting several leading clincians and scientists to Stanford. But it will require substantial philanthropic support over the next few years.
"The future is ours," says Gruber, compelled by a sense of urgency. "With the breadth of expertise across Stanford, leading-edge laboratories and care facilities, and key investments by the hospital and School of Medicine, we have the foundation to advance new therapies and bring hope to kids with cancer who have run out of options. Now we need philanthropy to unlock our potential. Honestly, with the support of the community, I feel there are no boundaries. We're unbeatable if we all work together."
The Chambers have generously committed to matching all gifts in support of cancer clinical trials up to $1.25 million. If you'd like to make a gift, contact Dawn Mitchell at (650) 724-5765 or Dawn.Mitchell@LPFCH.org.
This article originally appeared in the Fall 2021 edition of Packard Children's News.