SPRING 2017 ’ 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 with. They all chose to come to Stanford University and Lucile Packard Michael T. Longaker, MD 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. Leonard says. “What’s remarkable is that has advanced to a point where we are within expectant mothers and children. Government funding for medical research has today, the gene that causes his disease has been reach of fulfilling Lucile’s dream, in very Leonard’s focus is on building the infrastruc- been declining for decades, and it does not cover identified, and a cure is available. Given how tangible ways that were unimaginable just a ture and capacity needed to translate research the costs of the basic infrastructure needed for devastating this disease used to be, it seems decade ago. New techniques like stem cell and into cures. It may not be the headline-making studies to be completed. In the United States, almost miraculous that we now have a cure.” gene therapy are moving forward with unprec- stuff of science-fiction-come-true, but she is philanthropy is now the single most important She is determined to do the same for the edented momentum, bringing the potential to diligently working behind the scenes to mentor source of funding for fundamental, early-stage many children who still suffer enormous chal- cure hundreds of diseases affecting millions of and equip a generation of disease-fighting research and clinical trials. Without it, research lenges. Today, we can still do almost nothing children worldwide. researchers. will sputter along, and few if any true cures will for children with certain deadly brain tumors. “We can now imagine a future,” says Leonard, You know that feeling when you’re driving a ever reach the children who need them. Prematurity remains the number one cause of “when we can predict and prevent devastating great car? You get in, and everything just works. “We are on the cusp of major breakthroughs death in children under age 5 worldwide. And birth defects and childhood illnesses before they It gets you from Point A to Point B so seamlessly in many life-threatening diseases in children,” many mystery diseases remain incurable. occur. And when illnesses strike, we will not just that you don’t even have to think about what’s she says. “Lack of funds slows down the engines In her own research in bone health, Leonard treat them, but cure them definitively—keeping happening under the hood. that move these discoveries into clinical care. has seen young adults who survived previously children and mothers healthy not just for today, That’s what Leonard aims to build at “But with philanthropic support,” Leonard incurable diseases, but who now have osteopo- but for their entire life.” Stanford for child health and maternal research: adds, “we can get there—faster.” rosis that normally would only occur in elderly It’s a worthy goal, to be sure, but what will it a better engine for discovery that hums like a

6 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 7 Bubble Boy 2.0 BY TOM AHERN Maria Grazia Roncarolo, MD, has already cured “Bubble Boy” disease. What incurable disease will she cure next?

Fixing a single gene gave a child back the immune system he’d lacked at birth, allowing him to function normally in a dirty world within a year of treatment. That mission accomplished, Maria Grazia Roncarolo, MD, left Italy’s top medical research job and came to Stanford University in 2014. Here she’s leading efforts to fast-track stem cell and gene therapy out of the laboratory and into clinical trials. This year, she Why did you go into medicine Were you “destined” for a medical career? launched Stanford’s ambitious new Center for Definitive and Curative in the first place? I come from a family of business people. My My husband always says, “Maria Grazia went father had a completely different agenda for me. Medicine. around the world to pursue her dream, which He expected me to head the research depart- In this Q&A, Roncarolo, professor of pediatrics and of medicine, was to cure patients with incurable diseases. ment at the family-owned company [industrial And in the journey she encountered her husband paints for cars]. Unfortunately the problem was talks about her Center’s urgent near-term goal: to fully cure another and produced two beautiful children.” That’s solved because he passed away when I was in my how he always introduces me. And it’s true! last year of high school. 50 diseases ASAP, beginning with sickle cell anemia. [She laughs, then turns serious.] My mother and my elder sister who took over Philanthropists, start your engines. It won’t be cheap. From when I was a medical student, I was the company said, “The worst thing you can do exposed to the situation where many of these to yourself is to go into a job where you don’t children, we just had to say to the parents, have the passion.” “Sorry. We cannot do anything for your child.” So they gave me a green light. Especially my And that was incredibly frustrating. elder sister, who’d just gotten her MBA. I am It was in my mind when I was even a teen- the only one in my family who’s in the medical MARIA GRAZIA RONCAROLO, MD ager: to really make a difference for people who field. And this was something unexpected for & GROEBEN— DER VON NORBERT MEDICINE OF SCHOOL STANFORD are sick. And the idea was always for children. my family. Q8 supportLPCH.orgA PACKARD CHILDREN’S NEWS | SPRING 2017 9 “The potential at Stanford is unique. And when

The Roncarolo I say unique, I mean unique in the world.” team aims to move stem cell and gene — MARIA GRAZIA RONCAROLO, MD therapy to the clinic quickly and to translate basic science discoveries into treatments Medicine and in other schools, it’s unique. see if the same approach can take on cancer, for patients with Yet when you look at how much of this heart disease, and other common illnesses.” genetic diseases science is translated into novel therapies for and cancer. children, it’s not proportional. We have to ask: Do you have hobbies? So I felt that if I could really close this gap— OK, now I go full disclosure: my job is my passion. what scientists call the Valley of Death, between And I think that I’ve been incredibly lucky. In March 2017, news from Paris reported disease that affects millions of people world- the fundamental discovery and the cure—we My children know about my job. They know the first patient cured of sickle cell anemia wide, including certain populations of people could make a difference for many children with about the science and the patients I treated. through an experimental gene therapy, [in India and Africa] who do not have access incurable diseases. They were engaged in that all the way. in which bone marrow stem cells were to medical services. This could make a big We have in the pipeline about 15 clinical My husband is also a scientist. He under- extracted from the boy, altered so that difference. trials that we can start in the next three to five stands what it takes. And he could tolerate a wife they would make normal hemoglobin, and Currently, sickle cell can be treated with stem years in cancer, genetic diseases of the blood who was always very busy. And when she was then put back into the boy’s body. Was that cell transplantation, but this therapy comes at a and immune system, autoimmune diseases such not busy, she was exhausted. [Laughs again.] the same technique you pioneered in Italy? high cost for the patient’s quality of life. For at as diabetes or chronic inflammatory diseases, I’m a woman. I have two children; a husband least six months, he needs to be protected. He skin defects such as epidermolysis bullosa, and who has been with me 30 years. And a job that is It’s a similar approach. cannot go to school. He cannot play with other metabolic diseases. Each of these clinical trials a passion. There was no space for anything else. But I can tell you that what we’re doing here children. He cannot live in a normal household. would cost at least $10 million to show safety People say to me, “Oh, this was a big sacrifice now at Stanford is much more advanced. And it comes with a risk of mortality. and efficacy—to close the Valley of Death. for you.” Don’t get me wrong: the old approach works! If we can come in with an approach that is Not really. It was not a sacrifice. It was But it needs to be rendered more precise to be curative, without the toxicity and risk of the What’s your dream? a choice. broadly useful. The major problem with the old stem cell transplantation that we do today, it To cure what is curable, and to make curable I lived in the Bay Area from ’89 to ’97. That is approach? We never know where this healthy would be a major benefit. what is not. not a short period of time. gene lands in the genome of the cells. The precision medicine approach here at You know, if we think to the future and we People say to me, “Oh, you went to Yosemite.” It’s like, you have a genetic dictionary with Stanford is really the future of gene therapy. We dream a little bit, I always say the ideal situation I say, “No, we didn’t.” many words. And there is one word that is are working very, very intensively to bring sickle is to perform a newborn screening for a genetic “Oh, you went to Big Sur.” I say, “No, we misspelled and you put the correct word back in cell treatment to the clinical stage next year. disease, and then to cure it in the newborn. And didn’t.” SCIENCE randomly all over the dictionary, without alpha- if you want to go one step further, maybe 10 or 15 “Oh, you went to Las Vegas.” “No, we didn’t.” FICTION? betical order. That’s the best example I can give. During the clinical stage, a potential cure years from now, we will diagnose and repair the They would go on and on and on. “You went So what Dr. Matt Porteus does here at is tested on humans to prove its worth and defect in utero. to Cabo San Lucas.” “No, we didn’t.” Explore the Stanford with genome editing is very different. uncover any side effects. Clinical trials are a Ultimately, you want to have a patient that “What did you do?” potential of Because what we do is remove the misspelled prerequisite for approval by federal regulators. never becomes a patient. I worked. And I produced two children. stem cell and word from the dictionary and replace it with the [Laughing.] gene therapy correct gene, the correct word, exactly in the Where are we going to be in five years? In 2017, the MIT Technology Review My sister says, “Maria Grazia, how do you CLICK TO READ same position as it should be. The potential at Stanford is unique. And when I featured Dr. Maria Grazia Roncarolo’s live?” I live as I choose to live. I have two say unique, I mean unique in the world. work in its annual review of “breakthrough beautiful children who are amazingly balanced In 2018, there will start to be clinical trials? Because when you look at the incredible science technologies.” In an article titled Gene Therapy despite this mom who was always busy, busy, Correct. You know, severe combined immuno- that is done in this building [the Lorry I. Lokey 2.0, the review wrote: “Scientists have solved busy. And I have the incredible luck to have a job deficiency, what we call “bubble boy” disease, Stem Cell Research Building], and not only in fundamental problems that were holding back that is a passion.

is very rare. But sickle cell anemia is a nasty this building, but throughout the School of DOUGLAS PECK cures for rare hereditary disorders. Next we’ll

10 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 11 Unleashing Cancer Immunotherapy Against a Diabolical Disease

BY JULIE GREICIUS There is no “better battle to wage in this It was in 6th grade that Crystal Mackall, MD, associate director of the Stanford Cancer Institute and professor of pediatrics world than to (hematology/oncology) and of medicine at the Stanford University fight cancer School of Medicine, first knew she wanted to become a doctor. “I in kids.” had a female science teacher who was very inspiring to me,” says Mackall. “We did a lot of work on anatomy and physiology, and I just thought it was the coolest stuff in the world.”

Mackall, who is also medical co-director of no better battle to wage in this world than to the Stanford Laboratory for Cell and Gene fight cancer in kids.” Medicine, stayed true to her dream, even Since then, Mackall’s own work has as she refined her goal during training. “I helped define the battle lines. In 1984, she thought I wanted to be a heart surgeon,” read an early article about cancer immuno- Mackall says, “but it turned out I really therapy—an approach that uses the body’s wasn’t that good with my hands, and my own immune system against cancer cells. nose always itched when I had to go in the “Nobody ever thought it would really work,” operating room.” says Mackall, who still vividly recalls the Soon enough, she found the perfect fit: early days when “we were kind of a fringe pediatric cancer medicine. “I thought, this element in oncology. People thought we were is where I want to put my energy. There is a little bit ‘out there.’” CRYSTAL MACKALL, MD ELENA ZHUKOVA ELENA

12 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 13 “Cancer is diabolical. It’s adaptive, and keeps finding ways to come back. abnormality or mutation, because there aren’t new therapies, and getting them into the clinic, We have to outsmart it.” enough of them. “We have to actually direct the but also to learn about the toxicity and making cells to the tumor.” the treatment safer.” — CRYSTAL MACKALL, MD T cells—the “bloodhounds”—are removed T Cell: the from the patient, engineered with “the scent” Accelerating Research “bloodhound” in Mackall’s analogy, which is the chimeric Two organizations that began supporting of the immune system antigen receptor (CAR), and then returned to the children’s cancer immunotherapy research early patient’s bloodstream to begin their targeted and with sizable investment were Stand Up 2 Bold Mentoring still badly needed. With treatments like chemo- immunotherapy response. The CAR points the Cancer and St. Baldrick’s Foundation, which By 1989, she was studying the immune system in therapy, radiation, and stem cell transplantation, T cell to a specific tumor cell, such asCD 19 in in 2013 began funding the Pediatric Cancer cancer at the National Institutes of Health under five-year survival for children with cancer now acute lymphoblastic leukemia (ALL). When Dream Team, a group of eight institutions the direction of Philip Pizzo, MD, who was chief reaches above 85 percent, where it was once 10 treated with CD19-CAR immunotherapy in the co-led by Mackall. Lucile Packard Children’s of the National Cancer Institute’s Pediatric percent. Yet survival rates are much lower for active dose range, children in clinical trials Hospital Stanford became part of the Pediatric Department and who would later go on to serve rare cancers, and standard treatments can be with ALL—all of whom had prior relapses or no Cancer Dream Team in January 2016, when as dean of the Stanford University School of lengthy, sometimes taking years, and with brutal other treatment options left—are already seeing Mackall joined the Stanford University School of Medicine from 2001 to 2012. side effects.(See “Even Wondergirl Needs Heroes,” response rates of 80 percent. It’s also a shorter Medicine faculty. The new Parker Institute for “From early on, Crystal was committed to page 18.) And there are children for whom the course of treatment than standard approaches, Cancer Immunotherapy, led by philanthropist immunology and the potential for immuno- existing treatments do not work, or who suffer and one she anticipates will have fewer long- and entrepreneur Sean Parker, also provides therapy,” Pizzo recalls. “I was really struck by multiple recurrences, with the disease becoming term side effects. Mackall expects FDA approval support for infrastructure and research. that, and gratified to watch her evolve at the less responsive to treatment. of the CD19-CAR in the near future. Even with this funding, children’s cancers National Cancer Institute to pursue her vision “Cancer is diabolical,” says Mackall. “It’s Building on strong results with CD19-CARs, still present vast opportunities for support. and eventually obtain the leadership position adaptive, and keeps finding ways to come back. Mackall and her team have investigated a “Cancer is not one disease,” says Mackall. “It’s that I once held.” We have to outsmart it.” second target: CD22. Clinical trials for CD22 a bunch of different diseases. Each of them has “Phil Pizzo taught me to be bold in terms of began in 2015, and Mackall will soon publish a different set of targets that are going to need clinical investigation in children with dread A Targeted Approach the results. Having a second target in hand now a different group of investigators that have to disease, and in creating new therapeutics for One key to her progress in outsmarting cancer provides the opportunity for physicians to do develop it, because you can’t target a disease kids,” says Mackall. lies in the difference between pediatric and more than try each target in succession, but to unless you understand the disease. That’s the That boldness fueled Mackall’s persistence adult tumors. Immune cells seek out abnor- hit the two targets simultaneously. “Doing it beauty of this new CAR approach: It looks like for more than two decades, as she and her malities, which are easy to find in adult cancers one step at a time seems to give the tumor too it could be amenable to the treatment of solid colleagues gradually unlocked the secrets of because they have so many mutations. Because much time to adapt,” says Mackall. “So we are cancers or blood cancers, and we could give it in LISTEN immunotherapy. Initial approaches began to pediatric tumors have fewer abnormalities, now coming in with the first double-CAR.” The combination with other treatments.” show promise. “Cancers that were resistant to “they look more like an immature cell, so the first human testing of the double-CAR, target- “There are a whole lot of opportunities Crystal Mackall every other type of therapy were being able to be immune system can’t see it,” says Mackall. It’s ing CD19 and CD22, will begin clinical trials at out there for what we could tackle next,” discusses the controlled and put into remission with immuno- counterintuitive, but the relative flawlessness Stanford and the National Cancer Institute in a Mackall adds. biggest thing to hit therapy,” Mackall says. of pediatric tumors is also what makes them few months. “But right now, we have to choose based cancer in decades Then “everything changed in 2010,” when harder to fight. In addition to building on the progress of on resources. More money would allow us to CLICK TO LISTEN the National Cancer Institute, under Mackall’s That’s why Mackall has focused her efforts immunotherapy, Mackall is also determined to address more cancers simultaneously.” leadership, reported one of the first clinical on the promise of an approach that targets the continue research on its side effects, which can With Mackall at the helm of this research, successes with a targeted approach to activating cells identified in a specific type of tumor, like include, in very rare instances, neurotoxicity. Stanford will be helping to lead the field of the body’s own immune response. “The whole leukemia, for example. Using what she calls “When you turn on the immune system, you’ve pediatric oncology into a new realm of discovery Big news in the last two thing just exploded wide open,” Mackall adds. synthetic biology, “we create something new. got to turn it on really strong to fight cancer, and and treatment, redefining not only how we years—cancer “Now it’s the biggest thing in cancer medicine.” We hijack the immune system,” she says. “It’s that sometimes can cause bad side effects, which understand pediatric cancer, but how we work immunotherapy Despite many advances in childhood cancer sort of like a bloodhound. You give it a scent can sometimes be life-threatening,” Mackall to eradicate it. It’s inspiring work from a treatment and survival over the past four and say, ‘Go get this.’” In pediatric cancers, it’s says. “We’re working diligently not only to former 6th-grade girl who was just enjoying her

decades, more work in cancer immunotherapy is not enough to direct the immune system to an BLAUSEN MEDICAL discover better ways of treating cancer, making science class.

14 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 15 YOU CAN FUND A REAL CURE

TAKE THE YOU TOUR ARE See for yourself how your philanthropy HERE at Packard Children’s can change the future for hopelessly sick kids.

Once upon a time, philanthropy for medical research was “nice” to have. These days, it is absolutely essential.

With your support, researchers like Drs. Leonard, Roncarolo, and Mackall can make serious progress in children’s medicine. INCURABLE With your help, we’ll move new discoveries from the lab bench to the bedside—and cure sick children, faster.

FIND OUT MORE. Thanks to donors like you, Call to schedule your tour. Hyrum received Contact Sophie Emmerson at (650) 498-6168. the lifesaving care he needed.

16 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 17 WHO YOUR SUPPORT HELPS

BY GISELLE CONTRERAS, AGE 5, Even Wondergirl AND HER MOM, GABRIELLA MEDRANO-CONTRERAS Needs Heroes GABRIELLA: The day before Giselle’s 3rd birthday, we came to Packard Children’s. She had been sick for two weeks, and we wanted more extensive testing done. On the way home, I received a phone Giselle, aka Wondergirl call from the hospital asking where we were. I told them I was driving (right) and her sister, Siena (left), won’t stop back to San Jose. She told me to get off the highway, turn off the car. fighting until there’s a cure for all kids When she asked me to return to the hospital, I knew immediately. with cancer. Cancer—leukemia—had been a possibility, and I just knew.

We celebrated Giselle’s 3rd birthday in the hospital … with a bone marrow aspiration and her first chemo.

She became very ill during her first week of treatment and spent two months in the Pediatric Intensive Care Unit at Packard Children’s. Once she was released from the hospital, our family knew we had a long fight ahead.

When she has a fever, because of these kids’ compromised immune systems, the doctor always says to drive to the nearest hospital. I’ve

learned that as soon as she gets a fever, I start driving to Packard In spite of how sick she was, Giselle Children’s. By the time the on-call oncologist calls us back to tell me to never lost her go to the nearest hospital, I’m already at Packard! When you have a child bright smile. with cancer, there’s just no other place I would go.

I still remember when the nurses gave Giselle a toy that someone had donated. It meant the world to us because we had been stuck in a hospital room for weeks. It made us feel that we hadn’t been forgotten.

18 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 19 November 16, 2016, was a momentous day. Giselle and her family delivered hundreds of toys to Packard Children’s for patients. With their friends from Team G Childhood Cancer Foundation, they also presented a $4,885 check to Dr. Crystal Mackall for cancer immunotherapy research. (Dr. Mackall was not JOIN THE involved in Giselle’s care, but Giselle’s family was interested in the potential FIGHT for her research to provide better options to Giselle’s “cancer friends.”) In Give Giselle return, Giselle’s nurses in the Bass Cancer Center surprised her with a cake to and her friends Wondergirl is calling on heroes like medicine that celebrate finally being cancer-free. you to support pediatric research. doesn’t hurt CLICK TO GIVE Why Wondergirl still needs heroes like you

GABRIELLA: Packard Children’s was there when our family needed them. We are so grateful for the many donors who gave to this hospital so that Medicine that works, but hurts she could fight and beat her cancer. However, this isn’t enough for Giselle. As you’d expect from someone named Wondergirl, she won’t stop fighting GABRIELLA: Giselle lost her ability to walk due to the chemo. She ended until there is a cure for all kids with cancer. up in leg braces for some time. Her kidneys started to produce stones, she became septic several times, she had skin issues, her immune system Giselle does not like to see others suffer. She’s having a very hard time constantly needed a boost, the list goes on and on. understanding that the medicine she raised money for won’t come today. She’s heartbroken. I’ve done my best explaining how research works, but She saw it happen to other children in the hospital too. She would ask me, Giselle and her dad she’s not convinced. at the 2016 Summer why is he not playing, or why does she have a tube in her nose? She asked Scamper benefiting our hospital me why, if the medicine (chemo) was so bad, did they keep giving it to her? She wants the medicine for her cancer friends now! I explained that we had nothing else but this medicine.

My hope is that immunotherapy will become frontline therapy for My baby was so sick from chemo. But she smiled and stayed positive children with leukemia. I want it to solve the horrible issues and the life- through it all. So we started calling our brave and strong superhero threatening side effects chemo has on children. If the cancer doesn’t kill “Wondergirl.” our children, the chemo will. That is NOT okay. We need to allow research hospitals like Lucile Packard Children’s Hospital Stanford to unlock the For her 5th birthday, Giselle decided she didn’t want toys or gifts. Instead mystery of less invasive methods with fewer side effects. she wanted her friends and family to donate to the hospital. I asked her what we should do with the money raised. My hope is also that Giselle will live a full and normal life. That she draws Read more about strength from this experience to work harder in life to reach her goals. Dr. Mackall’s cancer GISELLE: I want to raise money for my cancer friends, for medicine that immunotherapy research on doesn’t hurt. page 12. GISELLE: When I grow up? I want to be a singer and a doctor to help kids with cancer.

20 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 21 We served We welcomed patients from

509,852 states & 4,585 2016 clinic visits 50 babies into the world REPORT ON GIVING 40 countries In 2016, 14,430 donors like you gave a total of $207.4 million to Lucile Nearly Packard Children’s Hospital Stanford and the child health and chaplains5 provided community 400 members obstetric programs at Stanford spiritual care to patients attended our first and families, 24 hours a University School of Medicine. Adolescent Mental day, seven days a week Wellness Conference Your generous support made all this and much more possible for patients and families over the last year. children50 ages 8 to 18 In the first program of cancer patient1 received years 25of innovation, enjoyed just being kids its kind in the country tickets and a limo ride to excellence, and care for Thank You! at the 22nd annual Solid Super Bowl 50 children and expectant Organ Transplant mothers GIFTS MADE BETWEEN SEPTEMBER 1, 2015, AND AUGUST 31, 2016 Summer Camp trained15 parent mentors offered care management strategies, as well as a shoulder to cry on, to parents of newly diagnosed patients

patients5,057 were seen in our Brain & Behavior Center, a national leader in care and research for epilepsy, Ranked in brain tumors, concussions, autism, free2,715 visits to our Teen % all and more. of patients40 benefited pediatric specialties10 on Health Van by at-risk youth. from financial US News and World Report’s Each $1 spent now for care assistance 2016–17 and prevention = $10 saved Best Children’s Hospitals in future health care costs.

Thank You NOTES

Stanford Dance Marathon $10 Million Gift from Julia Raises $110,000 for and David Koch Establishes Bass Cancer Center Clinical Research Unit for MORE THAN 1,000 STUDENTS Allergy and Asthma Hearts of Harvest Every day, donors like you make gifts of danced their way to a high-kicking A NEW CLINICAL RESEARCH UNIT Supports Heart Families $110,000 at the annual Stanford will be established thanks to Julia IN NOVEMBER, the Hearts of all sizes to build a healthier future for children Dance Marathon. Over 24 hours and David Koch’s visionary $10 Harvest Foundation gave a generous on February 18–19, students, million gift to Packard Children’s. $22,500 gift to support patients and and expectant mothers. Your support makes faculty, and staff dined and danced The unit will operate within the families receiving care at our in honor of children affected by Sean N. Parker Center for Allergy Children’s Heart Center. Hearts of our hospital a special place for our patients and pediatric cancers. Event proceeds and Asthma Research at Stanford, Harvest’s mission is to provide supported uncompensated care home to the groundbreaking allergy financial assistance and support to families, and we are tremendously grateful. at the Bass Center for Childhood and asthma clinical trials led by Kari local families and children who

Cancer and Blood Diseases. Nadeau, MD, PhD. ELGORT ARTHUR are in a health crisis, with priority Dancers were inspired by cancer Currently, Nadeau’s lab research allergies, as well as the life-changing given to children with heart condi- patients like 7-year-old Ellie from is on the Stanford campus, while effects of a clinical trial to safely tions. Its founder, Becki B. Brown, Redwood City, who was diagnosed her clinical research is at a Packard desensitize the allergies. “We made was inspired to give to our hospital with acute lymphoblastic leukemia Children’s licensed unit within El this gift with the goal of bringing because of her family’s experience $50 Million Gift from at age 2 and spent 852 days bravely Camino Hospital in Mountain View. better treatments to more children receiving care in our Heart Center Gordon and Betty battling cancer. Today, thanks to The Koch gift will enable Nadeau and adults suffering from dangerous and neonatal intensive care unit Moore Transforms the donor-supported care received at and her team to expand their clini- allergies,” said Mrs. Koch. Nadeau more than 20 years ago. Since 2012, Children’s Heart Center Packard Children’s, Ellie is three cal research to a redesigned unit at notes this “investment will have a Hearts of Harvest Foundation has In March, Gordon and Betty years cancer-free. Packard Children’s in 2018. tremendous impact on the care and contributed a total of $82,500, Moore made a $50 million Stanford Dance Marathon, Severe food allergies are a grow- treatment we provide. Together, we which has enabled our hospital to gift, the largest from an indi- founded in 2005, is the Bay Area’s ing epidemic, with rates having will make a difference for all those provide critical services to more vidual to Packard Children’s largest student-run philanthropic doubled in the last decade. The Koch who may benefit from research than 360 heart patients and their since the hospital’s founding event. On their Facebook page after family has experienced firsthand toward better, safe, and lasting families. Thank you from the gift from David and Lucile the event, organizers mirrored the anxiety of living with food therapies.” bottom of our hearts! Packard. In honor of this the sentiments of so many of our gift, Packard Children’s patients: “Thanks to everyone who internationally renowned has given us unyielding support … Children’s Heart Center will this journey was long, but it is far Inaugural Shop for Packard be named the Betty Irene from over. The strides we made Raises $50,000 for SUSANNA FROHMAN, SAN JOSE MERCURY NEWS Moore Children’s Heart the past few months are just the Packard Children’s Center. The gift provides funding for Moore Children’s Heart Center. beginning!” IN MARCH, 39 local retailers, restau- clinical and research facilities, an Stephen Roth, MD, MPH, chief of rants, and fitness studios hosted endowment for the Center’s highest pediatric cardiology and director of in-store shopping days that benefited strategic priorities, and endowed the Betty Irene Moore Children’s the Children’s Fund at our hospital. positions for faculty to lead special- Heart Center, also extended his The shopping festivities kicked off ized care and research. gratitude to the Moore family, noting with a party hosted by Shreve & Co. “Dr. and Mrs. Moore’s gift comes their partnership enables an unprec- and LumillaMingus, and continued at a critical juncture—enabling us edented opportunity for the Center over several weeks, with participating to advance beyond surgical repair to expand its state-of-the-art clinical retailers donating a portion of sales to to the discovery of transformational and research facilities, train future Packard Children’s. In total, the inau- treatments and interventions and, leaders of cardiovascular medicine gural event has raised over $50,000 to ultimately, to true cures,” said Frank and surgery, and improve the fields date. We are grateful to the wonderful Hanley, MD, the Lawrence Crowley, of pediatric and adult cardiology community of retailers and shoppers MD, Professor in Child Health and and cardiovascular surgery through that partnered with us to support Ellie, now three years cancer-free, at executive director of the Betty Irene innovative research. Stanford Dance Marathon Packard Children’s.

24 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 25 Thank You NOTES

Long-Time Partner Supports The Dinner Raises Awareness Dr. Greg Enns’ Research and Support for Mental Health HELP MITO KIDS is dedicated to ON MAY 5, we kicked off the first of spreading awareness about mito- many celebrations in our new hospital chondrial disease and helping expansion at our biennial gala, The families impacted by the life- Dinner. Held in the gardens and threatening disease. Mitochondria lobby of the new hospital building, are energy factories found in every the sold-out dinner and live auction cell of the human body. In mito- featured Rob Lowe and Jerry Seinfeld, chondrial disease, which primarily and raised over $3.2 million for the affects children, cells lose their “There’s No Such Thing as Too Much!” child and adolescent mental health The Ambassadors for Lucile Packard Children’s Hospital 2016–2017 Board of Directors. (Below) Co-president Margaret Munzig, Dr. Lucy Kalanithi, and co-president Kristen Wilsey. ability to produce energy and EIGHT-YEAR-OLD MAISY has a zest for fashion and philanthropy that has programs at Packard Children’s and sustain growth, and entire organs resulted in an amazing venture supporting kids with cancer. Maisy began by Stanford School of Medicine. Special can be damaged or shut down. making hats out of paper for kids who had lost their hair from chemotherapy thanks to co-chairs Gioia Arrillaga, Ambassadors for Lucile Packard Children’s Hospital Raise Currently, there is no cure for this treatments. “But then I thought headbands were more fun,” she says, so she Susan Ford Dorsey, Elizabeth Dunlevie, More Than $164,000 at the 10th Annual Lunch and Learn disease. moved to making headbands with colored puffs and even some bling. and Stacey Siebel, and the event’s ON MARCH 14, the Ambassadors and Learn, the Fund-A-Need goal Since 2011, Help Mito Kids has Flash forward a year and a half, Maisy Puffs have raised an amaz- steering and honorary committees, for for Lucile Packard Children’s was met and surpassed. They have donated more than $60,000 to ing $4,000 for childhood cancer research! In addition to providing cute their tireless work on this signature Hospital celebrated 10 years of raised more than $275,000 this support Dr. Greg Enns’ research headbands for kids with cancer, Maisy Puffs also provide our patients and event. On behalf of the children and giving, learning, and volunteering year, including $165,000 through to identify new ways to diagnose families something even more important—symbols of hope. families that will benefit from the together at their annual Lunch and Lunch and Learn, which will fund and treat mitochondrial disorders, Thank you, Maisy! event proceeds, thank you to everyone Learn at Sharon Heights Country a bereavement therapist for two including the development of a panel who made The Dinner a success! Club. Guests heard from Dr. Lucy years; a palliative care physician of sensitive blood biomarkers so that Become a Champion like Maisy at championsLPCH.org Kalanithi, the widow of Dr. Paul specializing in pain management patients with primary or secondary Kalanithi, a Stanford neurosurgeon for two years; and one year of the mitochondrial dysfunction can be and author of the New York Times Annual Day of Remembrance for monitored non-invasively. Thank Happy Year of the Rooster! bestselling memoir, When Breath families who have lost a child. These you, Help Mito Kids, for making THANK YOU to C.M. Capital Becomes Air, for which she wrote programs rely solely on philan- these advances possible! Foundation for bringing its annual the epilogue. During a fireside chat thropy, and we are deeply grateful Chinese New Year celebration to our with NBC contributor Liza Meak, to the Ambassadors for their tireless hospital for the fourth year in a row. Dr. Kalanithi shared her experience work supporting our hospital and its Our patients and families were treated caring for a terminally ill spouse patients and families. to calligraphy demonstrations, craft and a newborn. Guests were also projects, and delicious food. Lion honored by fellow Ambassadors dancers were the highlight of the member Esther Levy, who shared celebration, delighting everyone who her family’s experience with our gathered to ring in the Year of the hospital’s palliative care program. Rooster. The Ambassadors 2016–2017 Since 2014, C.M. Capital Foundation Fund-A-Need supported our has committed $150,000 to our hospital, hospital’s Bereavement & Family and is returning again as a Spotlight Guidance and Palliative Care Sponsor for the 7th annual Summer

Programs. Through the generosity Help Mito Kids supports patients Scamper. We could not be more grateful of members and guests at Lunch like Morgan, 12, and Taylor, 6. for their partnership. Xiè xie!

26 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 27 In the NEWS

In case you missed it … Back on the Court After Complex Surgery TWO YEARS AGO, Chris Formaker chose a procedure called rota- tionplasty to treat his bone cancer. Orthopedic surgeon Larry Rinsky, MD, removed the tumor in Chris’ femur and tibia, turned his foot and ankle 180 degrees, and reattached them with skin and muscle to Chris’ upper thigh. Once healed, a prosthetic leg was fitted with the backward-facing foot serving as Chris’ new knee joint. Says Rinsky, “there are advantages, particularly for kids like Chris who want to maintain mobility and continue playing sports. It is truly a long-term solution to a difficult problem.” Now 10 years old, Chris is cancer-free and doing what he loves most: playing basketball and cheering for the Warriors.

Adolescent Bariatric Surgery Program Receives Landmark Accreditation THE ADOLESCENT BARIATRIC Surgery Program at Packard Children’s recently received accreditation from the American College of Surgeons Metabolic and Bariatric Surgery Accreditation and Quality Improvement Program, making it the first and only adolescent bariatric program on the West Coast and one of three programs nationally to receive such a distinction. Led by surgical director, Matias Hospital Recognized with Tall Tree Award Bruzoni, MD, and medical director, Lawrence Hammer, MD, THE PALO ALTO CHAMBER of hospital was lauded for its work its support of uncompensated and the program offers behavioral, educational, and medical Commerce and the Palo Alto Weekly with East Palo Alto’s Ravenswood undercompensated care and its services to overweight children and adolescents. recently awarded Lucile Packard Family Health Center and Palo Alto’s dedication to community health Micaela Chapa, who underwent laparoscopic sleeve Children’s Hospital Stanford its Tall Mayview Clinic, both of which offer services, including adolescent mental gastrectomy four years ago when she was a senior in high Tree: Outstanding Nonprofit award services to low-income individuals health, child safety programs, and school, says, “I feel like the whole process with Packard for its longstanding contributions and their families. Packard community health education. Children’s was transformative... I really felt like a changed and service to the community. Our Children’s was also recognized for person with a very powerful tool on my side.”

Leslee Subak Appointed New Chair of Obstetrics and Gynecology Even Legalized, Marijuana Is Not Safe for Teens LESLEE SUBAK, MD, has been appointed the chair of the Department of A REPORT, co-authored by Seth Ammerman, care to adolescents and talking about drug Obstetrics and Gynecology at Stanford University School of Medicine, MD, professor of pediatrics (adolescent use are plentiful. He cautions that adolescents effective May 1. She succeeds Jonathan Berek, MD, the Laurie Kraus medicine), and published in the March are more vulnerable to the adverse effects Lacob Professor, who served as chair of the department since 2005. issue of Pediatrics, summarizes scientific of marijuana use than older users. He notes Subak is an expert in urogynecology, and her research focuses on evidence for the negative effects of mari- that teens who use marijuana regularly may multidisciplinary approaches to treating urinary incontinence in women. juana use on the developing brain and gives develop serious mental health disorders “I love building and participating in collaborations—across departments, physicians information to navigate conver- including addiction and depression, and have schools, and continents and across clinical, training, and research sations with parents and teens who believe more problems succeeding in school and missions,” she says. Subak is also dedicated to training the next genera- using marijuana is harmless. work. He also notes that long-term users tion of leaders in women’s health. She is a 1991 graduate of Stanford Through his work with the Teen Health of marijuana may develop unrecoverable School of Medicine. Van, Ammerman’s experiences providing cognitive deficits, even if drug use stops.

28 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 29 In the NEWS

Adolescent Mental Health seen at Packard Children’s emergency Inpatient Unit Opens at department and in other clinics can Mills-Peninsula Hospital be transferred to Mills-Peninsula. A follow-up on our Fall 2016 issue focused Antonio Hardan, MD, chief of child on teen mental health and adolescent psychiatry at Packard Children’s, and Daniel Becker, MD, STANFORD CHILDREN’S HEALTH and medical director of behavioral health Lucile Packard Children’s Hospital at Mills-Peninsula, believe this team Stanford have formalized a partner- approach supports a larger number 1 2 ship with Mills-Peninsula Hospital in of patients while providing trainees Burlingame to offer new youth mental hands-on experiences in an inpa- health programs and services for teens tient setting. The collaboration also in San Mateo and Santa Clara counties. increases access to Stanford doctors No Longer Conjoined, A team that includes a child-adolescent while enhancing continuity of care Sandoval Twins Return Home psychiatrist and Stanford doctors is and lowering societal stigma for ON MARCH 29, Eva and Erika now staffing up to eight beds in an inpa- patients and families seeking psychi- Sandoval, formerly conjoined twins tient setting at Mills-Peninsula. Patients atric treatment. who were surgically separated at our hospital in December, returned to their home in Antelope, California. The 2-year-old sisters left Packard Children’s in early March and spent 3 two weeks at UC Davis Children’s Hospital in Sacramento, where they Virtual Heart Revolutionizes Education and received physical and occupational Care of Congenital Heart Defects therapy to improve their mobility and “The heart is a complicated three-dimensional organ, and it’s really learned to eat independently. “The hard to describe what’s going on inside of it—especially when girls have just blossomed in terms of something is going wrong,” says David Axelrod, MD, clinical personality,” says lead surgeon Gary assistant professor of pediatric cardiology at Stanford University Hartman, MD. “They’re very engaging School of Medicine and co-designer of the Stanford Virtual Heart and chatty.” Program. “Virtual reality eliminates a lot of that complexity by Their mother, Aida, notes, “Now letting people go inside the heart and see what’s happening them- that we’re here, it’s amazing. We’ve selves—it’s worth way more than a thousand words.” been waiting two years for this.” Eva The Children’s Heart Center, Oculus, and virtual reality expert 4 5 and Erika will continue to receive David Sarno from Lighthaus, Inc., partnered to develop the regular checkups with Dr. Hartman Stanford Virtual Heart. The immersive technology allows users to and other caregivers at Packard rotate, open, inspect, and even teleport into a virtual heart. This Children’s. To date, more than 100 interaction, when guided by a cardiologist, gives patients, families, hospital staff across many depart- and trainees a deeper understanding of the problem and treatment ments participated in their care. options for a heart defect. 1. The twins say goodbye to their family Two additional virtual reality studies are under way at before being taken to the OR for separation. Packard Children’s: Project Brave Heart helps patients cope with 2. Surgery begins. 3. The twins’ grandmother, their anxiety by using cognitive behavioral therapy techniques, Isabel Pineda, embraced her grandson, the twins’ older brother, Emilio, after hearing that including relaxation and exposure; and True3D virtual imag- the girls had been successfully separated. ing technology inside the operating room helps surgeons during 4. Eva and Erika at their princess-themed procedures. Though adoption is in its early stages, the team is farewell party. 5. The girls enjoy wagon rides around the hospital, visiting with doctors and already seeking ways for virtual reality to be expanded across the others they’ve met since they were born at hospital in the future. Packard Children’s in August 2014. 6. Dozens from the twins’ care team gathered to wish 6 them farewell.

30 supportLPCH.org PACKARD CHILDREN’S NEWS | SPRING 2017 31 In the NEWS

HUMANS OF PACKARD CHILDREN'S Humans of Packard Children’s

HumansOfPackardChildrens.org

“When he was little, they told us he might not walk, he might not talk. I celebrate every little milestone, like the first time he talked back to me. It was really exciting to see. He’s like any other kid, pushing his limits! Despite his laundry list of health problems, he is happy 99 percent of the time.”

— TYLER’S MOM, JENNIE

Tyler, heart and epilepsy patient, with his mom, Jennie, and dad, David DOUGLAS PECK DOUGLAS Nonprofit Organization U.S. Postage 400 HAMILTON AVENUE, SUITE 340 P A I D PALO ALTO, CA 94301 Palo Alto, CA Permit No. 1 (650) 497-8489 supportLPCH.org

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