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SPRING 2017 SPRING ’ PackardThe magazine of the Lucile Packard Foundation for Children’s Children Health Newss CURING THE INCURABLE Translating discoveries into cures—faster inside: 2016 Report on Giving No Longer Conjoined, Sandoval Twins Return Home ’ Packard ChildrenNewss SPRING 2017 Volume 18, Number 1 EDITOR IN THIS ISSUE Jennifer Yuan ASSOCIATE EDITORS Megan Alpers-Raschefsky Lindsay Okamoto WEB EDITORS What childhood diseases can we Manya Khan Ali Koide cure next, with your support? WRITERS Tom Ahern Giselle Contreras and In my 35 years as a pediatrician, I’ve seen amazing improvement in the way Gabriella Medrano-Contreras Julie Greicius we take care of children. Many of the infectious diseases that caused kids to Jennifer Yuan be hospitalized during my residency are now almost medical history. Child- DESIGN 22 18 Andrea Hopkins Design hood cancer survival has jumped from 50 to over 80 percent. And today, there PHOTOGRAPHY are more surviving adults than children with congenital heart disease. Douglas Peck Norbert von der Groeben— What brought about all this change? Research. Decades of innovative and Stanford School of Medicine Elena Zhukova painstaking work by physicians and researchers, backed with the funding to LUCILE PACKARD FOUNDATION make change happen. FOR CHILDREN’S HEALTH Our work is not finished yet. When children are seriously ill, the two main BOARD OF DIRECTORS treatment options are still medication or surgery, sometimes accompanied by Elaine Chambers, Chair Manuel Henriquez, Vice Chair terrible side effects and a compromised quality of life. But through research, Mindy Rogers, Secretary C. Bryan Cameron, Treasurer new hope is emerging every day. Today, with stem cell therapy, gene therapy, David Alexander, MD, President and CEO Kirk Bostrom and immunotherapy, we are learning to use our patients’ own basic biology to Timothy Brackney fight back. 2 31 Jeff Chambers Christopher Dawes In this issue of Packard Children’s News, you’ll meet three incredible Ken Goldman Anna Henderson physician-scientists at the forefront of discovery. Individually, they are each Laurie Kraus Lacob 2 Curing the Incurable David Lee stars in their respective specialties. Together, they are a force to be reckoned 22 2016 Report on Giving Mary B. Leonard, MD Michael T. Longaker, MD with. They all chose to come to Stanford University and Lucile Packard 4 Translating Discoveries into Cures—Faster 24 Thank You Notes Lloyd Minor, MD Children’s Hospital because what we can achieve here for child health is Steve Mullaney Mary Leonard, MD, is building the engine David Orr unmatched anywhere else. for discovery. 28 In the News Susan Packard Orr Michelle Sandberg, MD From medicine to biosciences to engineering, the wealth of scientific Bill C. Sonneborn 8 Bubble Boy 2.0 32 Humans of Packard Children’s David K. Stevenson, MD knowledge at Stanford is staggering. But without sufficient funding, pediatric Maria Grazia Roncarolo, MD, already cured Matt Wilsey research will remain on the sidelines and move slowly. Your support enables “Bubble Boy” disease. What incurable disease FOUNDATION LEADERSHIP us to focus more attention on the health problems of pregnant women and David Alexander, MD, President and CEO will she cure next? Brian Perronne, Senior Vice President for children, and to leverage all that science toward finding new cures—faster. Development and Administration Donors like you give sick children hope for an even brighter future. What 12 Unleashing Cancer Immunotherapy Donna Richardson, Senior Vice President for ON THE COVER: Major and Principal Gifts childhood diseases can we cure next, with your support? Against a Diabolical Disease (From left to right) Drs. Maria Grazia Roncarolo, Eileen Walsh, Senior Editorial Director, Crystal Mackall, MD, brings new hope Programs and Partnerships Mary Leonard, and Crystal to the fight against cancer. Mackall at Stanford School of Medicine David Alexander, MD Packard Children’s News is published by the Lucile 18 Even Wondergirl Needs Heroes Packard Foundation for Children’s Health, an President and Chief Executive Officer independent public charity incorporated in 1996. Five-year-old Giselle Contreras (aka Wondergirl) For more information or to share Lucile Packard Foundation for Children’s Health still needs heroes like you. comments, please email [email protected] ii PACKARD CHILDREN’S NEWS | SPRING 2017 1 BEYOND THE LAB COAT CuringTHE INCURABLE These three brilliant and determined scientists all came to Stanford University to lead game-changing research in child health. Maria Grazia Roncarolo, MD (left), With your support, they Mary Leonard, MD (center), and Crystal Mackall, MD (right), are may change the world. leading efforts to translate new discoveries into better cures for children. ELENA ZHUKOVA ELENA 2 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 3 Translating “MY KIDS ARE 22 AND 24,” Leonard says. “But my role as a pediatrician has always been deeply influenced by my role as a parent.” Discoveries Last July, Leonard became the first woman to serve as the Adalyn Jay Physician-in-Chief at Lucile Packard Children’s Hospital Stanford and the Arline and Pete Harman Professor FASTER and Chair of Pediatrics at Stanford University into Cures – School of Medicine. She is now leading child BY JENNIFER YUAN and maternal health research into a truly exciting era. It’s fitting, given that Lucile Packard Children’s Hospital was also the creation of a visionary woman. In 1987, Lucile Salter Packard and her husband, David Packard, made the founding gift to build our hospital. Mary Leonard’s own children “My dream,” Mrs. Packard said then, “is that 50 years from now, the childhood diseases always know when she’s working in that cause so much anguish will have disappeared.” the hospital. They get unexpected In the 30 years since, we have made tremendous progress toward fulfilling that text messages from her, reminding dream. Children are surviving once deadly leukemias. Babies born with congenital heart them to look both ways before they defects and cystic fibrosis are living into adulthood. And diseases such as polio and cross the street. It’s usually on the tuberculosis, which were once the daily business of pediatricians, have largely days when she’s walked through the become history. pediatric intensive care unit and been acutely reminded of how devastating childhood injury and illness can be. MARY LEONARD, MD ELENA ZHUKOVA ELENA 4 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 5 “Now that I am physician-in-chief, the breadth of take to achieve it? All the scientific discovery in well-oiled machine, functioning in a systematic the world is of no use if there’s no way to trans- and efficient way. “What most excites me right late those insights from the lab into real-life now,” she says, “is seeing the spark of excite- death and suffering across our patients is much more treatments and cures for patients. That’s where ment for research in our junior trainees. I want READ Leonard, and your support, comes in. to do everything I can to support them to reach about Dr. Leonard’s on my mind. It motivates me every day to support our their full potential.” Her goal is to allow scien- research in: Building the Engine for Discovery tists to focus on the research rather than having At the Children’s Hospital of Philadelphia, to piece together a new engine every time. “Bad for the Bone: The Toll of passionate physician-scientists and clinicians, who are where she spent 25 years before arriving at That stuff under the hood, which will acceler- Childhood Chronic ate the discovery of new cures, includes support Stanford in 2014, Leonard directed the Office Disease” of Clinical and Translational Research, which for recruiting clinical trials participants who fit working to develop and deliver new cures as quickly as CLICK TO READ provided the resources, environment, opera- strict criteria for each protocol; budgeting and tions, and training to support and promote reporting to meet the requirements of funders — MARY LEONARD, MD possible.” the clinical and translational research of and regulatory agencies; biostatistics and data more than 300 principal investigators. Here management; oversight to enhance safety for Success is not inevitable. Government funding for medical research has been declining for decades, and it does not cover the costs of the basic infrastructure needed for studies to be completed. Leonard saw this progress up close in her adults. “Now that I am physician-in-chief, work as a pediatric nephrologist. Many years the breadth of death and suffering across our at Stanford University, where Leonard also patients; and training for the next generation of ago, she took care of a little boy—the same age patients is much more on my mind,” Leonard serves as director of the Child Health Research qualified investigators. as her own son—who had been born with a very says. “It motivates me every day to support our Institute, she aims to build similar economies of It’s a massive, admittedly unglamorous, and rare genetic kidney disease. The boy struggled passionate physician-scientists and clinicians, scale. Stanford’s faculty and schools are already absolutely essential foundation for research that constantly against life-threatening episodes who are working to develop and deliver new known for winning Nobel Prizes and topping will not only shape the future of pediatrics, but of high blood pressure, kidney failure, anemia, cures as quickly as possible.” best-of lists in medicine, biosciences, and potentially transform the lives of millions of heart failure, and other complications. Leonard is in the right place at the right engineering. The next step is to marshal all that children. “I admitted him to the hospital 14 times,” time. Today, for the first time ever, the science scientific genius to solve the health problems of Success is not inevitable, however.

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