Neurology Stewardship Report by The Children's Hospital of Philadelphia

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A Report on Your Investment in Hope

Hope Cause for

NEUROSCIENCES

Contents 3 Welcome Letter 4 News Briefs 6 Pediatric Stroke Research 8 Neuroprotective Efforts 10 Epilepsy Research 12 Ketogenic Kitchen 14 Developmental Disabilities Fellowship 16 Behavioral Sciences Family Fund 18 Child and Adolescent Mood Program 20 Special Events 22 Center for Autism Research 25 Navigating Autism 26 Understanding Autism 28 Building Hope

Left: Sultan, 4, traveled to Philadelphia from the United Arab Emirates for brain surgery.

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A message from

Joseph W. St. Geme, III, M.D. Dear Friends, Imagine for a moment that you have been noticing that your 2-year-old daughter can no longer say words she was able to say two months ago. Or consider how it would feel to watch your 10-year-old son struggle with uncontrolled seizures, despite trying medication after medication. Now think about the enormous relief and hope you would experience in knowing that experts at The Children’s Hospital of Philadelphia are here to help you and the child you love. At CHOP, we have three major groups of specialists who work tirelessly to provide comprehensive services and to perform cuttingedge research to assist patients with disorders of the brain. Our Division of Neurology is among the largest and most broad-reaching child neurology programs in the nation and is a world leader in diagnosing and treating a range of pediatric neurological problems such as headaches, stroke, epilepsy and neuromuscular diseases, among others. Our Division of Developmental and Behavioral Pediatrics provides care for conditions that cause enormous challenges for patients and families and that affect up to onefifth of the population, including attention deficit hyperactivity disorder (ADHD), developmental delay and autism. Our Department of Child and Adolescent Psychiatry treats children who are gripped by mental illness and who experience a variety of behavioral abnormalities. Your support of CHOP has made it possible for our physicians and staff to create innovative and compassionate programs for children with disorders of the brain, providing vital resources and hope for these patients and their families.

In the following pages, you will see examples of lifesaving and life-changing care that is happening here every day. You will also read about the tireless efforts of scientists at CHOP to make discoveries that will ensure a better future for children. Investing in the education of the next generation of pediatricians and other pediatric providers is another major priority of CHOP. By providing a world-class learning environment for medical students, pediatric residents and subspecialty fellows, The Children’s Hospital of Philadelphia ensures excellence in pediatric care — now and in the future. Our comprehensive approach to providing and improving care for pediatric patients in the Philadelphia region and around the world is possible because of the extraordinary support we receive from our donors. The power of your giving fuels our ongoing patient care, research and education programs. Together we can make the lives of patients and their families healthier and brighter. Thank you for all you do to improve children’s health. Sincerely,

Joseph W. St. Geme, III, M.D. Physician-in-Chief Leonard and Madlyn Abramson Endowed Chair in Pediatrics

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News Briefs Brenda L. Banwell, M.D., Joins CHOP as Chief of Neurology Brenda L. Banwell, M.D. (pictured left), joined Children’s Hospital as chief of the Division of Neurology in July 2012 and was named the Grace R. Loeb Endowed Chair in Neurosciences. She had previously been at The Hospital for Sick Children in Toronto. “My decision to come to Children’s Hospital was inspired by the institution’s clear commitment to clinical care and research for children living with neurological disorders,” says Banwell. “In addition to the resources provided by CHOP for the Division of Neurology, the Grace R. Loeb Endowed Chair provides key support for my research in pediatric multiple sclerosis.” Banwell remains the lead investigator of the Canadian Pediatric Demyelinating Disease Program (with five principal investigators, 47 collaborating researchers and 23 site investigators), which has now partnered with the Pediatric Demyelinating Disease Program at CHOP. It provides innovative research into the clinical features, MRI findings, immune cell behavior and cognitive impact of multiple sclerosis and related disorders in children. Banwell is a member of the National Institutes of Health Committee on Common Data Elements and the International Committee on Multiple Sclerosis Diagnosis. She is a member

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of the editorial board of Neurology, and is a founder and chief editor of Multiple Sclerosis and Related Disorders.

Fellowship is First to Combine Study of Autism and Epilepsy In July 2013, CHOP welcomed Alyssa Rosen, M.D., as its first fellow ever to combine the study of autism and epilepsy. For the twoyear training opportunity, Rosen will pursue research at the Center for Autism Research, clinical learning with colleagues in Psychiatry, Psychology and Developmental Pediatrics, and clinical practice in the Division of Neurology. “I’m very excited to be able to design a training program around my clinical and research interests in the overlap between epilepsy and autism,” Rosen says. “Despite a wellknown relationship between epilepsy and autism, researchers don’t fully understand the connection. I believe that a better understanding of this relationship may lead to insights into the underlying cause of autism in some children and open up new avenues for treatment.” Rosen received her bachelor of science in biopsychology and cognitive science from the University of Michigan and her medical degree from Harvard Medical School. She first came to CHOP in 2010 for residency in General Pediatrics and Child Neurology. At the completion of her residency in June 2013, Rosen was given the Meg Olivia Barkman Award, an annual award that recognizes Neurology residents who demonstrate clinical excellence and a compassionate approach to care. The award was created by a generous gift from

Meg Barkman’s parents, Ronald and Kristie, to memorialize the life of their daughter, a former CHOP patient. “The human connections in medicine are what I value most about my work,” says Rosen, who completed a five-month Kenneth B. Schwartz Fellowship in Pastoral Care while studying at Harvard. “This is the most meaningful award I could receive, and I am honored to have been chosen for it.”

Evenings with the Experts Advances in Pediatric Medicine A DISTINGUISHED SPEAKER SERIES

Donors’ Grants Will Fund Pilot Research for Inflammatory Brain Disorders In the spirit of collaboration, mentorship and program development, Meg and Richard Hayne, longtime supporters of various programs at Children’s Hospital, have generously funded three $20,000 grants for pilot research in inflammatory brain disorders. “Advancement in medicine begins with a novel idea; however, it is difficult to obtain financial support for this research,” says neurologist Amy Waldman, M.D., M.S.C.E. (pictured above), who is overseeing the grant process. “These donors recognize this and want to provide investigators with pilot funding to work on such projects.” Grant applications are currently under review by a committee of clinical and basic science experts in central nervous system inflammation. Proposed research projects must focus on clinical, immunologic or radiographic aspects of central nervous system inflammation or demyelination. The pilot grants will support research that is novel, explore scientifically highrisk concepts, provide critical data for a larger grant, or allow for a new collaboration.

May 6 — Look Inside Brain Power A presentation this spring by Brenda L. Banwell, M.D., chief of the Division of Neurology, will offer a glimpse into insights CHOP experts have found regarding migraines, headaches, epilepsy and other neurologic conditions. She will highlight the breakthrough work that allows CHOP to care for kids and families dealing with neurological disorders at more than 17,000 outpatient visits each year. Banwell’s presentation — which will take place at the Union League of Philadelphia on May 6 — is part of CHOP’s Advances in Pediatric Medicine speaker series, an annual series that offers the community a rare opportunity to engage with the Hospital’s medical leaders and experts about CHOP’s groundbreaking work. In addition to the brain, other topics in this year’s series include nutrition, concussions, eating disorders and bullying. For more information or to attend one of these sessions, please contact Lori Busch at busch@email.chop.edu or 267-426-6465.

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Uniting to Fight an Unexpected Foe Connecticut family, community support pediatric stroke research at CHOP

n 11 years as an emergency medical technician on the ambulance crew in Darien, Conn., Claudia Newton had administered a simple test for stroke many times. But always to older people. Now she stood in her own living room. She asked her 11-year-old son Alex to close his eyes and hold his arms straight out, palms up. His left arm drifted down. She had him do it again. His left arm drifted down.

It was a snow day. Claudia believes that may have saved Alex, because he told his mom when his left leg was tingling in the morning and a few hours later that the strange feeling was now in his arm. “Then he said, ‘My face is tingling,’ and that’s when the hair on the back of my neck stood up,” she says. She ran the stroke test. Then she put Alex in the SUV and drove through the snow to the emergency room. An MRI confirmed the fears of Claudia and her husband, Bob: Alex had suffered a stroke. They were dumbfounded. Stroke is caused by a disruption of blood supply to the brain, most often due to a blood clot or narrowing of a vein or artery. Without enough oxygen, the brain quickly suffers irreparable damage. Stroke is the sixth leading cause of death in children. Yet pediatric stroke is often unrecognized, even by first responders and emergency room staff whose decisions can reduce the time to diagnosis — and the damage.

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Claudia Newton, for example, had never been trained to consider stroke for ambulance calls involving children. But her instincts as a mother and EMT helped Alex get help quickly on that day. It was the beginning of a very difficult road. “It’s a journey you don’t want to go on,” Claudia says. After his first stroke, Alex was put on daily aspirin to thin his blood. Ten months later, he had a larger stroke. While he was in intensive care at a hospital in New York City, a doctor there told the family to call the leading expert on pediatric stroke: Rebecca Ichord, M.D., at The Children’s Hospital of Philadelphia. Since then, the Newton family has made the 3½-hour drive to Philadelphia many times to visit Ichord and the Pediatric Stroke Program team, including Julie Neitzke, R.N., B.S.N., the nurse who listened to Claudia cry during her first call. “I sobbed on the phone, and Julie’s compassion was what I needed,” Claudia says. “To be at CHOP is a gift. Dr. Ichord makes you feel like you’re the only patient and the only family she has because of the time she gives and her commitment.” During one of their appointments, Claudia was struck by the number of children she saw who were being treated after strokes, many in wheelchairs, and the relatively small amount of support for research into pediatric stroke. “It has been shocking to learn about pediatric stroke and how many children don’t have the kind of outcome that Alex has had,” Claudia says.

She gathered a group of friends into a committee, and they organized a “Night of Hope” dinner and silent auction in Darien, raising $58,000 for Ichord’s research at Children’s Hospital. Alex’s older brother Jack and his college fraternity have gotten involved, too, recently raising $5,000. Ichord is part of an international consortium investigating possible causes and new treatments for pediatric stroke, including the use of thrombolytics, often called “clot busters,” in children. Thanks to the Newtons, Ichord has been able to hire a full-time research coordinator, which increased what she and her team could accomplish in their studies. “There’s a huge, growing undercurrent of advances in laboratory research in the neurosciences, and we need to be able to find ways to translate those findings safely to patients,” Ichord says. “There are just amazing opportunities right now, but there’s a huge gap between the work that needs to be done and the dollars to do it.” The “Night of Hope” was Claudia’s first foray into fundraising. She intends to repeat the event next

Rebecca Ichord, M.D., testing Janae, 2, for left side dexterity 15 days following her stroke.

September. “It was as simple as asking. That was the hardest part, just asking,” she says. “Once you ask, people will bend over backward to help. It’s not as daunting as it looks, and the reward is so widespread and meaningful.” Though he has now had three strokes and suffers migraines, Alex’s cognitive impairment has been minimal, and he maintains good grades. Now 13, he is on two medicines and sees a hematologist at CHOP who is investigating rare blood disorders as possible causes of his strokes. “Alex is a curious boy who is full of joy,” Claudia says. “He teaches me and often consoles me and tells me it’s going to be OK. Because of the latest stroke, all of the fear is back. But we have a tremendous amount of hope. In his lifetime, our wish is that research will get to a point that these children don’t have to suffer.”

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CHOP neurologist Daniel Licht, M.D., with a patient and her family.

Hearts and Minds CHOP researchers advance neuroprotective efforts for children with congenital heart disease

orty years ago, most children with complex congenital heart disease (CHD) died before reaching adulthood. Now, the vast majority survive. But half of all children who have heart surgery as infants experience significant neurodevelopmental problems — including behavioral issues and learning difficulties — later on. CHOP neurologists Daniel Licht, M.D., and Robert Clancy, M.D., who care for children with CHD as part of the Hospital’s NeuroCardiac Care Program, are each leading research efforts to determine why these problems are so prevalent in children with CHD and how they can be prevented.

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An ‘A-ha’ Moment MRI scans performed in infants who have undergone early heart surgery for CHD show that more than 50 percent have an injury to the white matter of their brains. But it was another finding — that 20 percent of these babies show evidence of white matter injury prior to surgery — that was the “a-ha” moment for Licht. It was clear that for some babies, the risk of neurologic injury was highest not during or after surgery, as many believed, but before it. But what could be done to mitigate that risk? Licht and his team looked at MRIs taken before and after surgery, as well as measurements of cerebral blood flow and brain oxygen

saturations taken after surgery, and found that the lower the oxygen content of a baby’s blood before surgery, and the older he was at the time of surgery, the higher his risk of white matter injury. “It’s the condition in which they go into surgery that makes the difference,” says Licht. “You can’t do surgery fast enough for these kids.” Licht’s work, which was made possible by generous support from Stephen and June Wolfson, could lead to changes in surgical practice and has also led Licht to a new question: “If the most important thing is the health of the brain before surgery, what does that say about fetal life?” Licht is working with cardiothoracic surgeon J. William Gaynor, M.D., and Jack Rychik, M.D., director of the Fetal Heart Program, to identify drugs that can be given to a woman who is expecting a baby with CHD to protect the brain of her developing fetus. “That really is the next frontier for this research,” he says. “Instead of focusing on protecting the brain during and after surgery, we want to find ways to protect the fetus’ brain before birth.”

A Different Approach Clancy is also exploring neuroprotective options for babies with CHD. He believes that a drug called topiramate, which is already FDA-approved to treat epilepsy and prevent migraines, could block a chemical process in the brain that leads to white matter injury — and thus may have neuroprotective qualities if given to a baby before, during and after CHD surgery. In preclinical studies made possible by generous donations from Anne and Michael DeLuca, Lizabeth and Stephen Raynes, and Kathleen and David Pratt, Clancy’s team

“The interest has started to shift from ‘Can they survive?’ to ‘What do we know about the causes of these neurologic deficits?’ After a certain period in these kids’ lives, their CHD is the least of their worries.” — Robert Clancy, M.D.

determined that the doses of topiramate they believe are necessary to provide neuroprotection in children with CHD are the same as the doses already widely used to treat epilepsy in children. The finding gave the team confidence that their approach is likely to prove safe and feasible, and in July, after years of work by Clancy and James Cloyd, a pharmacologist at the University of Minnesota, the FDA granted approval to develop an IV form of the drug — another critical step in the work. Clancy’s team is currently in the process of obtaining the approvals needed to begin a clinical trial. CHOP is uniquely equipped to handle such studies, which must enroll a large number of patients to be considered reliable. In addition to the NeuroCardiac Care Program, which provides ongoing care and follow-up for scores of CHD patients, “we have a receptive Cardiac Center with lots of patients, we have research pharmacology, we have cardiac research nurses,” Clancy says. “Everything is right here.”

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Pushing Progress, Honoring Children Philanthropy crucial to epilepsy research and care at CHOP

recent Institute of Medicine report estimated that one in 26 people in the United States will suffer from epilepsy at some point in their lives, with the disease particularly common among children. Up to 40 percent of patients suffer intractable epilepsy, or lifelong seizures. There are currently no treatments to prevent the development of epilepsy in patients who are at risk, such as after a brain injury or viruses that cause brain infection. While research in the neurosciences is burgeoning, funds have become more and more scant. Philanthropy is crucial to fuel the very best in innovative care and to find causes, better treatments and ways to prevent this terrible disease.

One of the most effective ways for donors to benefit an area of research and care at Children’s Hospital is to create an endowed chair. The endowments provide guaranteed annual support for work important to the donor and give the Hospital a tool to recruit and retain the most talented physician-scientists in their fields. In 2009, Hospital donor Catherine D. Brown endowed a chair in epilepsy research that supports a promising fellow in neurology. “Her support has been a hugely important part of the growth of the epilepsy program here at CHOP,” says Dennis Dlugos, M.D., M.S.C.E., a neurologist who is director of the Pediatric Regional Epilepsy Program at CHOP. The Pediatric Regional Epilepsy Program has also benefited from the generosity of the family of Jason

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Banner, who had surgery at CHOP for intractable epilepsy. Their support is making it possible for Dr. Dlugos to start the “Living with Epilepsy” program. This program, which includes a dedicated psychologist, will focus on management of epilepsy beyond seizures, including associated cognitive, behavioral and social manifestations of epilepsy. Shelby and Tim Myers, through Clayton’s Hope Organization, the foundation they named for their son, have contributed to epilepsy care and research at CHOP for the past six years, focusing more recently on the science that will help physicians understand and therefore better treat epilepsy. “We don’t want doctors to find another Band-Aid,” Shelby says. “We want them to find a cause. And that’s the road they’re on at CHOP. The research is innovative, cutting-edge.” Ethan Goldberg, M.D., Ph.D., a neurologist and researcher at CHOP whose work is supported by Clayton’s Hope, seeks to learn more about the basic mechanisms of epilepsy to inform the development of novel treatments. “For those patients who continue to have seizures despite the best efforts of biomedical science, we need to be thinking about wholly new treatment strategies that target the underlying mechanisms of epilepsy,” he says. Goldberg studies a type of brain cell called inhibitory interneurons. “Seizures arise from dysfunctional brain circuits, which are the complex collections of interconnected neurons that process particular types of information, such as a memory or visual perception,” he says. Interneurons make

Grace, 5, treated for epilepsy at CHOP

up only a small percentage of all neurons in a circuit, but have critical functions in circuit operations, maintaining a balance between excitation and inhibition, and functioning as a “brake” to stop seizures. “Seizures and epilepsy may be due in part to abnormal activity of these inhibitory interneurons,” Goldberg says. Goldberg’s research uses novel approaches to monitor and manipulate the activity of interneurons in experimental models. He uses imaging techniques to monitor brain cell activity at the level of circuits, while manipulating interneuron activity using optogenetics. In this

technique, scientists implant DNA from a lightsensitive organism (such as certain plants) into neurons. This causes the neurons to react to light in a certain way, such as turning on or off. The technique has helped scientists identify which neurons control different activities and might be a step toward turning neurons on and off in humans to modify dysfunctional circuits. “For a young physician-scientist investigator starting my research career, the gift from Clayton’s Hope has been critical to moving my work forward,” Goldberg says.

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CHOP Opens Nation’s First Keto Kitchen Teaching families recipes to help children with epilepsy

t the opening of the Keto Kitchen at The Children’s Hospital of Philadelphia last September, Hospital leaders, donors, and patients and their families chatted and admired the new facility while snacking on chocolate mousse, salmon paté and a Greek yogurt-based dip for chips. They were not low-fat hors d’oeuvres,

Korey (left) and Brendan, Neurology patients who now follow the ketogenic diet

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by any stretch. In fact, CHOP Executive Chef James Lenhardt had done his best to create foods nearly entirely based on fats. It’s harder and less fun than it sounds. The foods reflected the ketogenic diet that parents and other caregivers will learn about in the new space, a teaching kitchen created

through the determination and generosity of CHOP patients and donors. More than 300 people gave to support the kitchen, covering half of the cost of outfitting the space, which includes a refrigerator, range, counters, cabinets and oven in a room also equipped with a computer, projector and central table that can be used for food preparation, meals or meetings with families. The unique configuration provides all the tools — culinary and educational — for parents and other caregivers to learn how to prepare a unique diet.

Korey Walton is one of the many patients who has had a dramatic improvement in his condition thanks to the diet. He was admitted to CHOP for five days while Bergqvist, dietitian Cagla Fenton, R.D., C.D.N., and nurse Claire Chee, R.N., gradually shifted him onto the ketogenic diet. Everything Korey eats is measured. He gets exactly 2,088 calories a day and takes vitamins to round out his diet. Almost every meal involves drinking heavy cream (specially donated by Wawa to CHOP families) and often stars ingredients like mayonnaise, eggs, butter, nuts, soy and oil.

The ketogenic diet — similar to an extreme version of the Atkins diet — is exactly 90 percent fat, 7 percent protein and 3 percent carbohydrates, and it has proven surprisingly effective for children whose epilepsy does not respond to medication, about 30 percent of children with epilepsy.

“He’s much more alert, a whole different kid,” says his mother, Dawn. “We have our son back.”

The human body prefers to get energy from carbohydrates like those found in starchy, sugary foods, but if deprived of carbs, our bodies will burn fat for energy instead. The ketogenic diet essentially leaves the body in full-time fat-burning mode, and the body’s chemistry changes in complex ways that help inhibit seizures. Children who have suffered scores, even hundreds, of seizures a day can have far fewer after starting the diet, with 20 percent of children on the diet cured of their seizures after a threeyear course of treatment, and most of the rest taking fewer medications, showing improved cognition, and in much better physical health. “For some of these kids, the changes are enormous,” says Christina Bergqvist, M.D., director of the Dietary Treatment Programs for Epilepsy at CHOP, describing as an example how some patients who start the diet wheelchairbound are later able to walk. “The diet is truly miraculous when it works.”

To help families like the Waltons succeed on such a labor-intensive and unusual diet, the dietary program staff teaches a class and provides recipes for keto-friendly foods, such as waffles from ground pecans and oil. But until the Keto Kitchen opened, there was no hands-on component, which made it harder for many parents to learn before heading home. “When I walked out of the class, I thought, ‘I don’t know if I can do this. I don’t know if Korey can do it.’ I would have liked to have seen the recipes made in front of me, and that would have made it easier going home,” Dawn says. “It hasn’t been easy, but it is the best thing we have ever done for our son.” The Waltons were grateful for the care they received at CHOP and wanted to help the families who would come after them. They became some of the biggest supporters of the effort to build the Keto Kitchen, raising more than $33,000 from family and friends, and collaborating with other major donors and fundraisers, like William and Mary Becker and Janine Engelman-Ortega, to make possible the first ketogenic kitchen in the country. It will serve the 150 patients currently in the dietary treatment program, and the 50 patients added each year.

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Care Through Medical Education Donor’s gift fills need for developmental-behavioral training

he field of developmental and behavioral pediatrics is a bit of a mystery, even to other physicians. Primary care doctors who easily identify and refer children to specialists for a chronic condition like asthma are far less certain about how to determine whether their patients might need developmental assessments and, perhaps just as crucially, about how to discuss that need with parents.

of qualified applicants. But Blum is confident that a generous 2013 gift will help change that.

“We need more people trained in the field,” says Nathan Blum, M.D., director of the Developmental-behavioral Pediatrics Fellowship Program at CHOP. “There only are about 700 board-certified developmental-behavioral pediatricians nationally. All of the training programs have very long waiting lists, ours included.”

“I just want to help others,” says Barbara Brodsky, fondly known by friends and family as Bobbi, when asked what drives her charitable contributions. “I just want to know that when I leave this place, I made a difference.”

Currently, the Developmental-behavioral Pediatrics Fellowship Program accepts only one or two fellows each year, despite receiving dozens

The new Barbara Brodsky Endowed Fellowship in Developmental Disabilities will provide fellows with opportunities to develop clinical, research, advocacy and leadership skills in developmentalbehavioral pediatrics, a holistic approach to the field that dovetails with the philosophy already in place with the existing fellowship program.

Her gift is already making an impact. Manuel Jimenez, M.D., the inaugural Barbara Brodsky Fellow in Developmental Disabilities, is working on a project to improve the number of children who actually get early intervention services after being referred for them.

Manny Jimenez, M.D., who worked as a public policy fellow on Capitol Hill, shares his insights with a small group during the Mid-Atlantic Regional LEND (Leadership Education in Neurodevelopmental and Related Disabilities) Advocacy Day in November 2013.

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Dr. Jimenez has spent the past six months developing an educational video to open a dialogue between parents and primary care physicians about developmental delay, early intervention and creating a plan to follow up on a child’s progress, with or without formal intervention. At right is a screenshot from the video.

An earlier CHOP study found that half of about 300 kids referred for early intervention did not go. Many of these children missed appointments because of simple logistical problems. However, Jimenez is implementing a comprehensive intervention to help address the other main reason families did not follow through with early intervention referrals: they were unsure whether their child required such care. “They felt they should decide whether their kid receives services,” Jimenez says. Rather than attempting to force parents to use intervention services, Jimenez has spent the past six months developing an educational video to open a dialogue between parents and primary care physicians about developmental delay, early intervention and creating a plan to follow up on a child’s progress, with or without formal intervention. “We want to respect parents’ autonomy and expertise, but help empower them to make informed, thoughtful decisions. For families who feel it is not the right time, we’re going to provide them with educational materials, things they can do with their child in the meantime, and follow up.”

The video was first used with the CHOP Care Network’s Cobbs Creek site in Philadelphia this past fall, and as it proves its effectiveness, it will be rolled out throughout the CHOP Care Network. Successful programs to increase followthrough on early intervention services have huge potential to improve the lives of children across the country. Jimenez has the opportunity to dedicate time to this project and many other responsibilities thanks to the new Brodsky Fellowship. In addition to the video, which is both a clinical innovation and a research project as he seeks to prove its effectiveness, Jimenez will be seeing patients, teaching in the Leadership Education in Neurodevelopmental and Related Disabilities research course for other fellows, and teaching residents clinical skills. He also works with CHOP’s PolicyLab, which brings together CHOP researchers and policy makers to maximize the impact of better care being developed at the Hospital.

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A Helping Hand Behavioral Sciences Family Fund is there to assist families with nonmedical expenses

he last seven years of 8-year-old Rheana Gravell’s life have been a cycle of frustration. Rheana had chronic ear infections and eventually developed permanent hearing loss. Her hearing loss contributed to behavioral problems. Her hearing issues couldn’t be addressed because standard ear infection treatments didn’t work. With ongoing hearing loss, Rheana has trouble communicating at home and in school, so she gets frustrated and her behavior gets worse. Luckily for Rheana, The Children’s Hospital of Philadelphia has the expertise to break the cycle and deliver the comprehensive treatment she needs to address all of her health issues, including her behavioral health needs. And her family is also getting the help they need to be able to afford to bring her from their home in Glennville, N.Y. to CHOP every three weeks thanks to a dedicated donor.

Rheana, 8

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When her local physicians couldn’t help Rheana, she was referred to CHOP’s Division of Otolaryngology and the Center for Childhood Communication (CCC). In collaboration with the Division of Immunology, doctors discovered she had a rare form of an antibody deficiency syndrome

which requires special infusion therapy of intravenous immunoglobulin (IVIG) to boost her body’s immune system. As Rheana’s immune system strengthened, doctors were able to treat her ear infections and her hearing improved, but it is still impaired. The CCC is working with the family’s insurance company to provide Rheana with hearing aids. However, she must continue IVIG infusions to keep the infections at bay. Often when Rheana is at CHOP, she and her mother, Erikka Gravell, also receive behavioral health treatment from Louise Montoya, M.A., L.P.C., A.C.S., C.S.C., a mental health therapist for CCC’s Integrated Behavior Health Services. These sessions help Rheana better deal with her hearing loss and learn how to behave more appropriately. “There is a big difference between ‘hearing’ and ‘understanding,’” Montoya says. “When a child has hearing loss, adaptations need to be made in all the places the child goes — home, school, the car, a sleepover at a friend’s house — so the child can understand what is being said. If a child hears but doesn’t understand, she fills in the blanks, and can get it wrong. Then, if she’s corrected, she feels like someone changed the rules in the middle of the game, and that is extremely frustrating.”

Houses are full — hotels. They are expenses that health insurance doesn’t cover and that Erikka, the family’s sole breadwinner, can’t afford. Families like the Gravells, who accumulate significant nonmedical expenses while their children are being treated, are helped by the Behavioral Sciences Family Fund, which was launched with a generous donation from the McEwen-Fial Foundation. Clinicians from the CCC identify families that could benefit from the fund. For donor Susan McEwen-Fial, CHOP’s stellar reputation and a personal experience triggered her decision to start the fund. “I know that CHOP is a leader in the nation for pediatric care,” she says. “Although I live overseas now, I grew up in the Philadelphia area and still like to serve the community. “Once, while getting occupational therapy services at CHOP for my child, I noticed other families that were stressing about getting access to services. I thought that it must be a terrible feeling to have as a parent. That was the catalyst for my reaching out to CHOP. I feel very fortunate to be able to offer such a service and to work with the dedicated team in Behavioral Health.”

When a child doesn’t do what she is told, people in the child’s life have to constantly figure out: Did the child understand? And if she did, is she not following through because she doesn’t know how or because she doesn’t want to? Behavioral therapy addresses all these issues.

Tami D. Benton, M.D., CHOP’s psychiatrist-inchief and executive director of the Department of Child and Adolescent Psychiatry and Behavioral Science, understands the interplay between parental stress, a child’s stress and the child’s ability to heal.

Despite living three hours away, Rheana comes to CHOP every three weeks for infusions; each treatment takes a full day. Because Rheana has often had negative side effects and significant reactions to treatment, her family needs to stay nearby for three to five days after each treatment. Each trip to Philadelphia costs the family for gas, tolls, food and — when the Ronald McDonald

“This is a tremendous gift to families to support their expenses so they can get their child the treatment the child needs,” Benton says. “Many families don’t have excess money in their budgets to cover the costs of travel, hotel or meals. The fund can reduce a lot of stress for families, and that benefits the child greatly.”

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Assessing and Treating Mood Disorders New program to care for affected children, also jumpstarting research

or just more than a year, psychiatrists, psychologists and researchers have been running a new clinical program focused on mood disorders, depression and bipolar disorders. The program allows children and adolescents to get the care they need while CHOP can track their outcomes to further improve treatments in the future. The Child and Adolescent Mood Program (CHAMP) was established for outpatients in 2012 thanks to the ongoing generosity of donors, including Scott Spiezle and Susan Goldy, who have been contributing to behavioral healthcare at Children’s Hospital for almost a decade. “I’m just proud that we’ve been able to support in a small way the need for mental healthcare,” Spiezle says. “There needs to be more public awareness, and that hopefully would lead to more funding. Most people don’t think about the need for mental healthcare for children and adolescents.” Spiezle says he believes many more people would benefit from mental healthcare and believes the research underway at Children’s Hospital will help children who need care and educate the larger public about the causes and treatment options for mental illnesses.

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CHAMP has established an assessment service for children and adolescents presenting with mood symptoms for the first time or for second opinion evaluations and treatments. In addition to giving children and families answers and the beginnings of a treatment plan, the comprehensive assessment service has allowed CHOP to gather systematic information about the children and families treated here. Research on their care and outcomes can show which approaches work best for which children. In addition, the program now includes psychiatry and psychology trainees in the clinic in order to train the next generation of clinicians to perform state-of-the-art clinical assessments of mood and anxiety disorders, as they commonly co-occur. Beyond the assessment service, CHAMP includes treatment as well. In addition to treatment provided by CHOP Psychiatrist-in-Chief Tami Benton, M.D., and psychologist Rhonda Boyd, Ph.D., the program currently has two additional psychologists skilled in evidence-based psychotherapies for mood and anxiety disorders. Benton says they are expanding their mood evaluation and treatment protocols to include all clinicians in the Department of Child and Adolescent Psychiatry to improve recognition and consistency of these evidence-based practices.

“Depression and bipolar disorders get better with treatment,” Benton says. “There is help for young people who believe that they will never smile again.” On the research side, Benton and her team are working to gather the most comprehensive and useful data possible for understanding and treating mental illness. In addition to psychological and psychiatric assessments, they plan to collect blood specimens from patients to analyze endocrine, immune and inflammatory mediators of mental health conditions and to obtain genetic data from parents and youths who present with these conditions. The goal is to better understand the biology underlying the symptoms seen with depression and bipolar disorders. The hope is to eventually secure funding to obtain neuroimaging data as well. The goal of these studies, which are

being conducted with the Center for Applied Genomics and the Center for Autism Research, will be to identify biomarkers for some mooddisordered states. In keeping with CHOP’s emphasis on familycentered care, which provides support for parents and family members to ensure they can give patients the best care at home, CHAMP is collecting family satisfaction data and developing a family resource program by asking parents what services, support and resources are most needed as they seek evaluation, cope with a diagnosis and deal with ongoing treatment. The program is also building a website that will allow families to access mental health resources and information about their children with mood disorders.

“There needs to be more public awareness, and that hopefully would lead to more funding. Most people don’t think about the need for mental healthcare for children and adolescents.” — Scott Spiezle

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Raising Funds, Raising Awareness Buddy Walk® and Family Fun Day More than 3,300 people came out to Villanova University Stadium on Oct. 6, 2013 for the 12th annual Buddy Walk® and Family Fun Day, sponsored by Northwestern Mutual Eastern Pennsylvania. The event raised $247,000 for CHOP’s Trisomy 21 (Down syndrome) Program. Families enjoyed games, athletic activities, moon bounces, arts and crafts, music, sweepstakes, mascot appearances, and more.

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Huddle Up for Autism On April 14, 2013, CHOP partnered with the Philadelphia Eagles to host an Autism Family Fun and Awareness Day at Lincoln Financial Field. The event brought in more than 3,000 participants for field and locker room tours, arts and crafts, games, face painting, and magic shows, and raised $34,000 for CHOPâ&#x20AC;&#x2122;s Center for Autism Research.

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Research and Care from Every Angle Center for Autism Research takes multifaceted approach to studying disorders

he prevalence of autism spectrum disorder (ASD) has risen tenfold in the last few decades, yet the causes for ASD are not clearly understood and there are few truly effective treatments.

The Center for Autism Research (CAR) at The Children’s Hospital of Philadelphia is the largest center of its kind dedicated to understanding the causes of ASD so that treatments can

target biological mechanisms and effect lasting changes. This progress will aid discovery of which treatments work best for which individuals based on genetic, brain imaging and behavioral profiles. CHOP’s multidisciplinary teams in Psychology, Neurosciences and Genetics are spearheading the largest and most innovative autism studies ever conceived. In addition to improving treatments and access to services in the long run, CAR provides families living with ASD now comprehensive, coordinated, family-centered care and support to meet each child’s specific developmental and educational needs — free of charge through the Center’s research protocols and through low- or no-cost seminars for parents and caregivers. Although just 5 years old, CAR has already made substantial contributions to autism research, with more than 250 professional journal publications on autism. CAR has about 100 faculty and staff performing cutting-edge autism research, completing up to 35 new evaluations per week, as part of more than a dozen different research projects.

A student in an AIMS classroom in Lewis C. Cassidy Elementary School in Philadelphia received one-on-one instruction from a team of educators during the clinical intervention trial for ASD.

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CAR geneticists have led national efforts to discover the genetic underpinnings of ASD, discovering a range of genetic associations, including the strong role for metabotropic glutamate receptor (See page 26) and cadherin pathway genes. CAR neuroimagers have led pioneering brain mapping research on key brain systems that map onto core dimensions of autism,

including difficulties with social communication and language. Moreover, CAR researchers are part of a national effort to define the first signs of autism in the developing brain and have found that the brains of infants who go on to develop autism are already developing differently at 6 months of age, before any symptoms emerge. CAR is at the forefront of using technology to help youth with autism. Researchers at CAR have developed a set of computerized games proven in clinical trials to effectively teach specific social skills. Other research projects at CAR are evaluating the effectiveness of behavioral and pharmaceutical treatments and innovative approaches to combining therapies. CAR researchers also recently concluded the largest autism intervention study ever conducted anywhere in the world. In the Autism Instructional Methods Study (AIMS), CAR partnered with the School District of Philadelphia and other Philadelphia agencies to try to determine the best ways to move evidencebased autism interventions into practice. The goal of the study was to improve educational outcomes for children with ASD. Although the study has ended, CAR staff continue to provide professional development training and in-classroom consultation to more than 120 classroom teams in kindergarten through 5th grade and autism support classrooms across the City of Philadelphia. A number of new studies have grown out of the initial project. Recognizing that ASD can have a devastating effect on families, scientists at CAR are also conducting research on autism’s economic and psychological effects on the family and how parents make treatment decisions for their children with ASD. All of CAR’s outreach and education programs are funded through private giving, and donations subsidize the writing of the evaluation reports

that are given to families that take part in research. Most notably, though, many of the research endeavors that take place every day at CAR would not have begun without the generosity of donors. Since its inception, CAR has been allocating money received for the general support of the Center — gifts both large and small — to provide seed money to promising research that otherwise would have no money to begin. One project that has benefited greatly from the support of private donors is the study of anxiety in ASD. Many of the children with ASD who come to CAR and to the clinical centers at CHOP are plagued by anxiety that causes significant child and family stress and often affects school performance. Medication is often prescribed, however families and doctors alike recognize that these medications do not work as effectively for children with ASD. Donor support allowed CAR to launch a research program focused on the neurobiological underpinnings, manifestations and measurement of anxiety in children with ASD, making enough progress to earn a $2 million grant in 2012 to continue the work. Without the generosity of CAR’s supporters, this project would never have been launched. Similarly, CAR is using donor funding now to do a pilot study of oxytocin as a potential new treatment and is looking for support for many other promising projects.

For more information about the Center for Autism Research, contact Lynn Johnson-Porter at 267-426-6511 or JohnsonPorterL@email.chop.edu and request the Center’s most recent annual report.

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Annie, 9, with her mother, was diagnosed at 18 months with autism.

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Navigating Autism A roadmap for families

magine you are bringing your son or daughter to a regular checkup at the pediatrician. As you walk in, your brain is racing: Don’t forget to buy milk and pick up the dry cleaning. Did you pay the credit card bill? You feel the buzz of a text message. It’s just another busy day, and you’re happy to scratch this checkup off the to-do list. But then you get some unexpected news: The pediatrician thinks your child is showing signs of autism. For so many parents, it is a moment of pure shock. With one in 88 children now thought to be affected by autism spectrum disorder (ASD), more and more parents are struggling to understand an autism diagnosis, find the resources their child needs, and help him or her through all the normal milestones, from toilet training and school to adolescence and adulthood. “There’s no comprehensive, one-stop shop to find this information,” says Gail Stein, L.S.W., A.C.S.W., a social worker at CHOP’s Center for Autism Research (CAR). “You can figure out how to find a psychologist or speech therapist, but where do you go for social skills groups? An education program? There’s no clear path. There is just so much on the Internet — where do you begin?” When Susan Wenger received the news that her 18-month-old grandson was flagged as showing signs of autism, she went into overdrive trying to connect him with an array of therapists and services that CHOP specialists recommended. But even for a savvy businesswoman, it was incredibly difficult. That’s why Wenger and her family have provided the financial support for CAR to create an

“autism roadmap.” When the roadmap goes live this spring, it will be a comprehensive website providing families with accurate information about ASD, directories of service providers at CHOP and elsewhere, and other information ASD families will need at various stages of life. Based in part on the in-person Next Steps workshops for families and professionals that CAR offers several times a year, the website has a fun graphic interface showing a cartoon-like town. By clicking on various “buildings” in the town, visitors can navigate through sections including education, home and family, community support, government and legal, and employment. Already Stein and her team have written or compiled more than 300 articles for the site, and they expect it to continue to grow. “The content will never be finished,” says Stein. “We’ll always be adding to it and updating it. Every time we turn around, we find more to say!” The site will launch with a searchable directory of service providers, both CHOP and non-CHOP, within a 50-mile radius of the Hospital, and creators hope to expand it steadily over time. “A tool like this is just so needed,” says Wenger. “It will make life so much easier for parents and grandparents, and help get children who are red-flagged for autism the resources they need as quickly as possible.”

The autism roadmap will be available from CAR’s website at www.research. chop.edu/programs/car.

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Brock, 11, was diagnosed with autism spectrum disorder at the age of 6.

Understanding Autism Donor-funded study identifies potential cause of autism spectrum disorder

ne in 88 children has autism spectrum disorder (ASD), a dramatic increase over the last 15 years. Yet little is known about the causes of ASD and more research is needed to devise effective treatments. Hundreds of different genes have been identified as contributing to ASD, and it is clear the environment plays a role as well, albeit a smaller one. In addition, each child with ASD has a unique presentation of the condition and a smaller subset of those hundreds of genes behind his or her particular case.

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As a result, figuring out which genes are responsible for ASD has been a complex and often frustrating challenge. But Robert Schultz, Ph.D., and his team at CHOP’s Center for Autism Research (CAR) have made a significant advance in identifying the exact combination of risk factors that produce autism. A joint study by CAR and researchers within CHOP’s Center for Applied Genomics (CAG), one of the world’s largest genetics research programs, has focused on other diseases that often cause ASD, in particular the 22q11.2 deletion syndrome. This

involves a group of genes that are accidentally deleted on one chromosome during early development, including a gene that affects the biological pathway mediated by glutamate, a neurotransmitter in the brain. This study found that too much glutamate signaling during brain development increases a child’s chances of developing ASD. This finding is significant because it points to a clear mechanism that can cause ASD. Such knowledge is critical for the development of new therapies. This breakthrough was made possible by a recent $2.5 million gift from Jeffrey Lurie and his family, which has provided transformational support of autism research at CHOP now totaling nearly $4.5 million. The Luries’ recent gift enabled CAR to collect and analyze genetic samples from 1,000 families and supported hiring a genetic counselor to work with families interested in participating in genetics research.

children with ASD process sounds a fraction of a second slower than other children, providing what could become a way to diagnose ASD based on a biological signature, rather than the current method using observation of behaviors. Such a diagnostic tool could allow earlier intervention and improved outcomes for children. “Open-ended, foundational gifts like those from the Lurie family and Allerton Foundation give us more freedom to build and support programs than is the case with federal grants,” says Schultz. “They are vital to the continued progress and success of our autism research programs, allowing us to start new areas of research that are essential for innovation.”

“Now that we have this guidepost, we have a roadmap to work from to create much more effective treatments: medications that can reverse or prevent the onset of symptoms,” says Schultz, CAR’s director, who holds the Regional Autism Endowed Chair, created by the Allerton Foundation, which has given $3.8 million to autism research at CHOP over the last decade. In addition to providing the endowment for Schultz’ chair, the Allerton gifts provide unrestricted funding that covers salaries for several staff members and support various endeavors that aren’t totally grant funded. Philanthropy from the Lurie family also supports research using MEG (magnetoencephalography) technology at the Lurie Family Foundation’s MEG Imaging Center at CHOP to measure the brain activity of school-aged children to better understand differences in the way the brain processes signals in children with ASD. Earlier advances from that research, led by Timothy Roberts, Ph.D., helped show that

Patients with ASD undergo brain imaging at the Lurie Family Foundation’s MEG Imaging Center.

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Building Hope The campaign for the Buerger Center for Advanced Pediatric Care

nyone who has been to the Hospitalâ&#x20AC;&#x2122;s Main Campus in West Philadelphia recently has seen that the new outpatient care building, the Buerger Center for Advanced Pediatric Care, is well underway, with steel girders rising above Civic Center Boulevard. This project, the largest endeavor in CHOPâ&#x20AC;&#x2122;s history, was designed with the input of patients and families and consists of a 12-story outpatient care facility, five levels of underground parking to accommodate 1,500 cars, and a 2.6-acre landscaped plaza.

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Through modern technology and design elements, the Buerger Center will enable CHOP to offer unparalleled outpatient care and the ideal patient experience, both medically and emotionally. Scheduled to open in 2015, the building will centralize outpatient services and amenities that are currently scattered throughout the Wood Center, Childrenâ&#x20AC;&#x2122;s Seashore House and the Main Hospital. But we need help from the community. Philanthropy is critical to funding the construction, and many opportunities remain to have your generosity recognized.

Buerger Center: Naming & Recognition Opportunities

$5 million n Rooftop Garden

$100,000 n Elevator Lobbies on

A stunning green space, this 14,000-squarefoot rooftop, accessed from the sixth floor, will provide unique healing areas for physical and horticultural therapies, as well as a peaceful place of reflection. Landscape architects will create perennial gardens, a water feature and more, all safely enclosed by a high glass wall.

Patients will travel through the building using a central elevator bank. The light-filled elevator lobbies on each floor will face the landscaped plaza below.

$2 million n Physical Therapy Gym A new physical therapy gym will add more than 5,600 square feet for rehabilitation programs. This state-of-the-art facility will have a two-story rock climbing wall, sports medicine area and mirrored studio space.

$1 to $2 million n Clinical Areas The Buerger Center ultimately will bring together CHOP’s outpatient services currently in the Main Hospital, Children’s Seashore House and the Wood Center. Unit naming opportunities include Radiology, Orthopedics, Rehabilitation Therapies and more.

$500,000 n Welcome and Information Desks This area will be the first thing visitors see as they enter a floor. The desks will be located on every level and staffed with greeters to offer directions, general assistance and information.

$250,000 n Water Features in the Plaza Children naturally love water, and the 2.6-acre landscaped plaza will be accented with three unique water features, including two water walls and a winding canal feature. Each can be named for a gift of $250,000.

Clinical Levels

$75,000 n Oncology Infusion Pods Within the Alex Scott Day Hospital, there are numerous “pods” where children sit for hours as they receive infusions. Outfitted with recliners and televisions, these pods are a home away from home for the day.

$15,000 n Roof Garden Planter Beds Individual spaces within the roof garden can be named in honor of a loved one. These include several perennial and planter gardens.

$10,000 n Patient Exam Rooms Many clinicians and families have provided their input to help us design comfortable, functional and attractive consultation and examination rooms.

$1,000 to $10,000 n Plaza Paving Stones Outdoor paving stones of varying sizes located in four specified areas within the landscaped plaza provide an opportunity to pay tribute to or recognize a loved one. Donors may reserve a paver with a gift of $1,000, $2,500, $5,000, $7,500 or $10,000.

$500 n Inscription on Main Lobby Walkway Gifts of $500 will be recognized with the names of donors on the glass railing of the landing areas located on the Buerger Center’s Main Lobby walkway to the second floor.

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Let Your Legacy Be the Gift of Childhood

Zena, 15, Neurology patient, with her father Jorge

Our care, our research, our teaching. They all rely on gifts from Children’s Hospital supporters, now and in the future. Many of the most important and enduring gifts to CHOP have been legacy gifts. Including Children’s Hospital in your will, or as a beneficiary of a retirement plan, is one of the easiest ways to invest in the health and well-being of future generations of children. These gifts won’t affect your current cash flow and may even help reduce taxes for loved ones. If you have already made a planned gift to CHOP, thank you, and please let us know. We can help you designate your gift to a specific Hospital program, and we’ll invite you to join the Lewis Society, a select group of supporters who have included Children’s Hospital in their plans. Visit us at giftofchildhood.org/plannedgiving to learn more, or contact Tom Yates, director of planned giving, at 267-426-6472 or yatestg@email.chop.edu.

w w w. g i f t o f c h i l d h o o d . o r g

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On the cover: Caitlyn, 6, a patient in the Sleep Study Program

Thank You The children you see throughout this report are our patients. It is for them, and for the many others in our care, that we relentlessly pursue the next advances in neuroscience. Your support is vital to this work. To learn more about how you can help, please contact one of our directors of development:

For Neurology: Meghan Tumolo at 267-426-6498 or TumoloM1@email.chop.edu

For Psychiatry and Behavioral Sciences: Dan Agoglia at 267-426-6461 or AgogliaD@email.chop.edu

For the Center for Autism Research: Lynn Johnson-Porter at 267-426-6511 or JohnsonPorterL@email.chop.edu

The Children’s Hospital of Philadelphia is ranked No. 1 in the nation by Parents magazine and is No. 1 on U.S.News & World Report’s 2013–2014 Honor Roll of the nation’s Best Children’s Hospitals.

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