Spring 2013
Children’s
Research Breakthroughs at CHOP
also inside Karabots Center Opens in West Philadelphia
the view from here It is truly exciting to work alongside some of the world’s best medical researchers. What they do unlocks
OFFICERS
the mysteries of the human body and changes children’s lives,
Mark Fishman Vice Chairman
not just at CHOP, but also around the world. I see so many breakthroughs on the horizon, and it inspires me every day. Yet I realize that much of what goes on in our 800,000 square feet of laboratory space is hidden from public view and involves
Mortimer J. Buckley Chairman
Tristram C. Colket, Jr., R. Anderson Pew Honorary Vice Chairmen steven M. Altschuler, M.d. Chief Executive Officer
the most sophisticated level of science and mathematics —
Clark Hooper Baruch Secretary
so technical that it is almost another language.
Anthony A. Latini Treasurer
With this issue of Children’s View, we are excited to take you behind the scenes of The Children’s Hospital of Philadelphia Research Institute. In the pages ahead, we will introduce you to just a handful of the 1,400 people who have devoted their lives to pediatric research. People like Kathy High, M.D., whose work I
Thomas J. Todorow Assistant Treasurer
BOARD OF TRUSTEES n. scott Adzick, M.d. • steven M. Altschuler, M.d. • Clark Hooper Baruch • A. Lorris Betz, M.d., Ph.d. • Aminta Hawkins Breaux, Ph.d. • Mortimer J. Buckley • Reid s. Buerger • dominic J. Caruso • Alan R. Cohen, M.d. • Tristram C. Colket, Jr. • Arthur dantchik • Mark denneen • Mark Fishman • Lynne L. garbose, Esq. • shirley Hill • Anthony A. Latini • James L. McCabe • John Milligan, C.P.A. • Asuka nakahara • Jeffrey E. Perelman • R. Anderson Pew • gerald d. Quill • daniel T. Roble • david B. Rubenstein • Anne Faulkner schoemaker • salem d. shuchman • Kornelis smit • Binney Wietlisbach • nancy Wolfson EX-OFFICIO
Paula Agosto, R.n. • Tami Benton, M.d. • Robert doms, M.d., Ph.d. • Jeffrey A. Fine, Psy.d. • William J. greeley, M.d., M.B.A. • diego Jaramillo, M.d., M.P.H. • Ralph Wetmore, M.d. EMERITUS
Leonard Abramson • Willard Boothby • stephen B. Burke • Ruth M. Colket • Peter C. Morse • george Reath Jr. • stuart T. saunders Jr., Esq. • Richard d. Wood Jr.
Jeffrey d. Kahn, Esq. General Counsel & Assistant Secretary Margaret M. Jones Assistant Secretary
have followed for decades. Her gene therapy techniques (Page 10) have been able to reverse an inherited form of blindness and hold great promise for many different diseases. Meanwhile, CHOP
Children’s
surgeon Alan Flake, M.D., is on the cusp of a revolutionary cure for sickle cell disease (Page 12), which would halt this terrible
EXECUTIVE VP & CHIEF DEVELOPMENT OFFICER
disease before the child is even born.
stuart sullivan
From the common problems of obesity and concussions (Pages 18-19) to serious rare diseases like congenital hyperinsulinism (Page 16), CHOP is working hard to help children live longer and fuller lives. But if not for the exceptional vision and philanthropic commitment of our generous donors, much of this would forever remain theoretical ideas.
ASSISTANT VP, COMMUNICATIONS & DONOR RELATIONS
WRITERS
Louis Bell, M.d. Jerisha gordon Mark Jolly-Van Bodegraven Eugene Myers PHOTOGRAPHY
Ed Cunicelli Kevin Monko graham studios DESIGN
Mark Turbiville
swivelstudios, Inc.
EDITOR
PRODUCTION
Julie sloane
Kimberly Caulfield nicole Keane
Children’s View is produced by The Children’s Hospital of Philadelphia Foundation. COMMENTS AND INQUIRIES SHOULD BE ADDRESSED TO:
The Children’s Hospital of Philadelphia Foundation 34th street and Civic Center Boulevard Philadelphia, PA 19104-4399 givehope@email.chop.edu For information about making a contribution to support CHOP, call 267-426-6500 or visit GiftofChildhood.org. PRINTED BY
LLC
Thank you for helping us invent and engineer new, healthier futures for children all over the world!
Steven M. Altschuler, M.D. Chief Executive Officer
Founded in 1855, The Children’s Hospital of Philadelphia is the birthplace of pediatric medicine in America. Throughout its history, a passionate spirit of innovation has driven this renowned institution to pursue scientific discovery, establish the highest standards of patient care, train future leaders in pediatrics and advocate for children’s health. A haven of hope for children and families worldwide, CHOP is a nonprofit charitable organization that relies on the generous support of its donors to continue to set the global standard for pediatric care.
© 2013 The Children’s Hospital of Philadelphia, All Rights Reserved.
Children’s
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cover story Big ideas: Research at CHOP Want to see the medicine of tomorrow? Go behind the scenes at The Children’s Hospital of Philadelphia Research Institute.
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The Nicholas and Athena Karabots Pediatric Care Center opens in West Philadelphia.
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Today@CHOP
Our two- and three-legged furry friends, a sister’s mitzvah project, learning about Lyme disease and more.
That's no ordinary window washer! A superhero swings by.
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Curing disease at the DNA level? CHOP’s incredible advances in gene therapy.
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After 30 years of research, one surgeon is close to a breakthrough for sickle cell disease.
A radical rethinking of our body’s cells could transform modern medicine.
A season of fabulous fundraising events
On the Cusp of a Cure
mighty mitochondria
volunteers in Philanthropy
ON THE COVER: Yannick, 13, was almost completely blind until he received experimental gene therapy at CHOP.
Read more about the science behind his breakthrough on page 10.
today@CHOP
Paw Power Cyrus
festus
When bright-eyed Cyrus walks the halls of CHoP on his two legs, it is nearly impossible not to smile. His three-legged pal, Festus, gets a similar reaction. After all, most dogs have a full set of four. Cyrus and Festus’ can-do spirits have made them two of the 20 much-loved regulars in CHOP’s Paw Partners Pet Therapy Program, which is generously supported by donor Gerry Shreiber. The dogs’ weekly visits, along with owner Domenick Scudera, help ease the stress of a hospital environment, lighten the mood for patients — many of whom miss their own pets at home — and provide unconditional love and acceptance. “Cyrus and Festus are unique,” says Scudera, a theater professor at ursinus College, who has been bringing the dogs to CHOP for four years. “They are visible symbols to the kids that you can get through adversity — and it’s clear they’ve overcome a lot.” It was love at first sight for Scudera after he spotted both animals’ stories on shelter adoption websites: At just three months old, Festus lost a front leg from injury sustained while living on the streets; little Cyrus was born without his front legs. Despite their challenges, Festus moves just fine on a three-legged gait, while Cyrus motors around on his custom-made wheels. Scudera has noticed that rehabilitation patients in Children’s Seashore House at CHOP seem to push themselves harder to complete their exercises when Cyrus or Festus visit. “It’s really changed my life to volunteer with the dogs and bring them around,” says Scudera. “The dogs love it, the patients love it —JERISHA GORDON and I love it.”
Think your canine pal has what it takes to become a therapy dog at CHOP? Visit childrensview.org for more information about Paw Partners. 2 | Children’s view | Spring 2013
kids in the hall
WE WALKED THE HALLS OF CHOP AND ASKED PATIENTS THE SAME QUESTION:
“if you could meet any famous person, who would it be and why?”
Louisa
Samuel
AGE 28 • PULMONOLOGY
AGE 4 • INTEGRATED CARE SERVICES
“Cupid, because he’s a good dancer and I love the Cupid Shuffle.”
“Batman, because he is cool.”
C’Ani
Mike
AGE 10 • NEUROLOGY
AGE 17 • NEUROLOGY
“Sofia, from Disney’s ‘Sofia the First,’ because she is a princess and lives in a big castle.”
“Dwyane Wade, because I love basketball, always looked up to him and how he respects people.”
AJ
Brianna
AGE 14 • DIALYSIS
AGE 10 • PULMONOLOGY
“Drake, because I like to rap.”
“Louis Tomlinson, because he is in One Direction and he is funny and British.”
A mitzvah for Cancer Patients
lEFT TO RIGHT: Remi, Pamela, Abby, Steve and Julia Rosenthal
Pamela and steve rosenthal still well up with emotion as they remember labor Day weekend in 2004 when they brought their 20-month-old daughter Julia to CHOP’s Emergency Department. “At 3:30 a.m., an oncologist walked in and introduced herself,” says Pamela. “And that’s when we realized.” Julia had stage 3 Wilms’ tumor, a cancer of the kidneys. Thankfully, her treatment was a success. In February, 10-year-old Julia celebrated eight years of remission. Neither Julia nor her identical twin, Remi, remember that time at CHOP, but big sister Abby, now 13, hasn’t forgotten. When Abby’s synagogue, Tiferet Bet Israel in Blue Bell, Pa., asked her to do a community service project in conjunction with her bat mitzvah, she didn’t hesitate: “I wanted to give back to CHOP because they saved my sister’s life and I love her,” she says, looking warmly at Julia. “I wanted to help all the other children like her.” Abby’s Auction was born. By asking local businesses to donate items like toys, movie tickets and restaurant
gift cards — Abby asked all the store managers herself — she collected 55 items. Synagogue members, friends and family were all encouraged to bid on the items, many of which went over face value. Her initial goal was $600, which she increased to $2,000 as bids added up. ultimately Abby’s Auction raised $2,701, and Abby decided to contribute $299 of her own money to make it an even $3,000 for the Cancer Center. Abby is not alone. Each year, a number of children choose CHOP as the beneficiary of their bar and bat mitzvah projects, while others have given their time and energy to CHOP for high school graduation or other community service projects. Julia and Remi also plan to raise money for CHOP for their bat mitzvahs in 2015. “I hope the girls will remember this experience,” says Pamela. “What started out as a very tragic situation for us has morphed into something so positive.” Adds Steve, “We’re indebted to CHOP, and we’re going to keep continuing to give in whatever way we can.” —JulIE SlOANE
If you or someone you know would like to give back to CHOP as part of a milestone project, please call 267-426-6496 or email communityfundraising@email.chop.edu. Spring 2013 | Children’s view | 3
today@CHOP
A grand Opening 3
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The atrium at the new Nicholas and Athena Karabots Pediatric Care Center Nicholas and Athena Karabots tour the new facility. CHOP patients present a plaque to the Karabotses. Dancers from Mexicano Folklorica greeted visitors. The Karabotses share a laugh with CHOP CEO Steven Altschuler, M.D., and a young patient. Illstyle dancers wowed the crowd with high-energy dancing.
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Passersby on 48th Street marvel at the building’s rainbow colors. The Karabotses chat with Philadelphia Mayor Michael Nutter. The ribbon-cutting ceremony with (L to R) Steven Altschuler, Councilwoman Jannie Blackwell, Athena Karabots, Nicholas Karabots, Mayor Nutter and CHOP President and Chief Operating Officer Madeline Bell A Reach Out and Read volunteer shares a story with a little girl in the Marjorie and David Rosenberg Reading Room.
The sun momentarily burst through the overcast winter sky on Feb. 6 as donors and dignitaries cut the ribbon on the new Nicholas and Athena Karabots Pediatric Care Center in West Philadelphia. The event was nearly as colorful as the building’s rainbow-hued facade, with greeters in traditional Mexican garb, city officials, trustees, TV crews, hip-hop dancers and even a children’s choir. It was a big day for CHOP and the City of Philadelphia. But it was also a big day for Jason and Kiara, who were at the Karabots Center that day for their son’s 6-month well visit checkup. CHOP’s expert pediatricians would check the baby’s growth rate, administer vaccinations and provide the family with a book to read with him. “We’ve never had anything like this place in our neighborhood before,” said Kiara. “It’s a blessing.” The Karabots Center will get 60,000 patient visits each year and will also be the core location for CHOP’s top-ranked medical residency training. At the same time, it represents a new model for a healthcare facility, also serving as a hub for community-based programs like Early Head Start, Reach Out and Read, and the Homeless Health Initiative. Aiding those efforts are two donor-supported spaces, the Marjorie and David Rosenberg Reading Room for Reach Out and Read as well as the
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Romano Parent Resource Room. The aim is to create an environment that improves the whole lives of children and positions them for future success. Mayor Michael Nutter, speaking at the event, called it “a tipping point” for the revitalization of West Philadelphia. “There’s no greater calling, or challenge, than taking care of our kids. This investment, combined with others in the area, demonstrates that this community truly cares about children, their families and the people who work here.” “None of this would have been possible without Nick and Athena Karabots,” said CHOP CEO Steven Altschuler. Giving back is a personal mission for Nick Karabots. “I grew up in New York in a neighborhood a lot like this,” he said. “I was fortunate enough to escape a difficult life, and now Athena and I have the privilege of giving back and providing opportunities to struggling kids and families, so that they too can become successful and productive members of society. And hopefully one day, they too will be in the position of supporting the place that gave to them.” “The Karabots family has given a remarkable gift to the children of West Philadelphia,” said Altschuler. “We couldn’t ask for better friends and partners with CHOP.” —Mark Turbiville
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today@CHOP
license to
Thrive! To learn more, visit giftofchildhood.org/licenseplate.
The Amazing
Spider-man Swings By CHOP
last fall, hundreds of children and their parents crowded the Hospital’s windowlined bridges to watch our friendly neighborhood Spider-Man take a break from his crime-fighting duties for an important mission: delighting the young patients at CHOP. Doing whatever a spider can, Spider-Man spent three hours peering in through the glass at so many smiling faces. It was a plot hatched by CHOP Environmental Services after seeing photos of Spider-Man visiting Great Ormond Street Hospital in london. Brought to life by Jenkintown Building Services — CHOP’s window cleaning vendor and Peter Parker’s local employer — Spider-Man also followed the lead of Child life specialists to visit the windows of children unable to leave their rooms. Reports in The Daily Bugle indicate that Spider-Man had such a good time that he has persuaded a few of his amazing friends to drop by in the future. Stay tuned. —JulIE SlOANE
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Pennsylvania drivers can now get a specialty license plate celebrating Children’s Hospital! A portion of each $45 plate funds vital programs in our community and makes a statement about your support for CHOP.
ask Dr. Bell Louis Bell, M.D., chief of the Division of General Pediatrics at CHOP, explains the latest in medical thinking on an important topic: LYME DISEASE. BY lOuIS BEll, M.D.
Shutting Out Lyme Disease Most kids can’t wait for all the outdoor fun of spring and summer, but warmer temperatures are also attractive to ticks, the tiny pests that spread lyme disease. The very name has come to strike fear into the hearts of parents — the dreaded lyme disease! — but fortunately it is easier to prevent and treat than you might think. My colleague Jason Kim, M.D., M.S.C.E., an infectious disease expert at CHOP, explains that although early spring through late fall is generally considered tick season, ticks can occasionally attach to our skin during mild winters. Ticks are also found all over the country, in every state but Alaska, infesting suburban neighborhoods, wooded areas, and even parks and other green spaces in cities. Only certain ticks — those carried by whitefooted mice and white-tailed deer — may carry the bacteria that cause lyme disease. It’s important to remember that not all ticks will be infected, and not every tick bite will lead to lyme disease. The good news is that even if a tick attaches to the skin, it takes 36 to 48 hours for it to transmit disease-causing bacteria to a person — ample time to discover and safely remove the parasite. Ticks prefer to attach in darker, warmer regions of the body (e.g., under the arms or on the scalp). Therefore, make a habit of checking your kids all over for ticks after they have been outdoors. When the tick is full, it disengages and drops off, so if you miss it you may never know it was on your child — unless he or she displays symptoms of lyme disease, around four to 10 days after being bitten. The first sign of the disease is typically a slightly raised, circular ring at the bite site with a bull’s-eye at the center of a painless, non-itchy rash. later stages of infection may include multiple bull’s-eye rashes, facial paralysis, meningitis or arthritis. Seek immediate medical attention for any of these symptoms, even if you never saw a tick bite.
The great news is that lyme disease can be treated easily and completely with oral antibiotics and has no lasting effects — regardless of whether the illness is discovered right away or in one of the later stages of infection. After treatment with antibiotics, the lyme germ is totally eliminated. It will not “hide” in the body to recur at a later time. Prevention is even more effective. Ticks can’t bite through fabric, so the best protection is to wear long-sleeved shirts tucked into pants, long pants tucked into socks and closed-toe shoes. If you can’t cover exposed skin, insect repellents containing DEET can help ward off ticks.
Even if a tick attaches to the skin, it takes 36 to 48 hours for it to transmit disease-causing bacteria to a person.
Be sure to inspect your kids and pets daily and learn to remove ticks properly: use sharp, narrow-nose tweezers to carefully grasp the tick by the head, close to the skin, and slowly pull upward without twisting or squeezing it. Then clean the wound with rubbing alcohol. Ticks are a health threat but a manageable one, and lyme disease is not at all a chronic or debilitating illness for children. Simply keep a vigilant watch for ticks and symptoms, and have fun playing, hiking, camping and picnicking outdoors with your kids!
For more of Dr. Bell’s columns, please visit childrensview.org. Spring 2013 | Children’s view | 7
Fraser Wright, Ph.D. Director, Clinical Vector Core
Rajiv Sharma Predoctoral Fellow Rebecca Loomis, Ph.D. Senior Research Associate
Daniel Hui, Ph.D. Postdoctoral Fellow
cover story
It takes place inside gleaming glass towers, using high-tech instruments and complex mathematical calculations. But at its core,
medical research is all about
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Tom Curran, Ph.D., F.R.S. Deputy Scientific Director
Lyam Vazquez, M.D. Research Technician
people.
More than 1,400 people work at The Children’s Hospital of Philadelphia Research Institute, making it one of the largest pediatric research programs in the country. They came to Philadelphia from all over the globe, and their life’s work is to solve the medical mysteries of childhood disease. They do it for their children and grandchildren. For the hundreds of thousands of CHOP patients. For future generations around the world.
F. George Otieno Research Associate Hakon Hakonarson, M.D., Ph.D., Director, Center for Applied Genomics
Xilma Ortiz-Gonzalez, M.D., Ph.D. Research Fellow
Philip Johnson, M.D. Chief Scientific Officer
They spend countless hours hunched over lab benches, studying data, completing regulatory paperwork and brainstorming new answers to age-old problems. At the cutting edge of science, their work is unfathomable to most of us. But it is also fundamentally human — learning how our bodies work and giving children longer, healthier lives. — J u l ie S l oane
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qI MA, PH.D., POSTDOCTORAl FEllOW
SIlMARA MONTAlVÃO, VISITING STuDENT
MARTIN PICARD, D.HOM., PH.D., POSTDOCTORAl FEllOW
Repairing DNA, After decAdeS of being “the next big thing,” gene therApy iS here. And IT’s WORKIng.
OF ALL THE ACCOMPLISHMENTS A SCIENTIST CAN MAKE, Katherine High, M.D., can claim one of almost Biblical proportions: She made blind people see. High would protest that it’s a bit more complicated — she worked together with Jean Bennett, M.D., Ph.D., and Albert Maguire, M.D., at the University of Pennsylvania, and their team restored only some of the patients’ sight — but the fact remains: This work allowed people who were legally blind to now recognize faces, see objects and better navigate the world. High is one of the world’s leading experts in gene therapy, which has long been a “next big thing” in medicine: Take a person with a devastating genetic disease and replace their nonfunctional gene with a normal one — a cure built right into your DNA. It sounds elegantly simple, but it is among the most complex endeavors in all of medicine. Finally, after decades of work, the future has arrived. High and her team at CHOP’s Center for Cellular and Molecular Therapeutics have produced clinical trials that show real
it turns out gene therapy also has something in common with real estate: location, location, location. results in humans, first with a type of inherited blindness called Leber’s congenital amaurosis (LCA), and more recently with the blood clotting disorder hemophilia B. Both are relatively rare conditions, but they are clear proof of concept — gene therapy works. At its most basic level, gene therapy is all in the delivery. To move microscopic snippets of DNA into the body, High’s team uses adeno-associated virus (AAV) vectors, essentially envelopes of molecules engineered to look like a common virus. But instead of delivering a nasty payload of viral DNA, 10 | Children’s view | Spring 2013
it delivers a normal gene that the recipient needs. They are so essential to gene therapy research that when a biotechnology company stopped making them in 2004 — not enough short-term profits — CHOP created its own clinical-grade production facility. (It also produces lentivirus vectors used to treat cancer patients like Emma Whitehead. See Page 16.) Precisely engineering these particles is just one challenge; preventing the immune system from destroying them is another. “In a sense, viruses and the human immune response have been conducting a war for millions of years,” explains High. “The human immune system evolves to fight the virus, and then the virus makes some change to get around the immune system. It’s like the arms race.” It turns out gene therapy also has something in common with real estate: location, location, location. It has worked well in the eye, where the immune system is relatively less aggressive, but has been more difficult elsewhere. “It has become a set of problems where each tissue — liver, skeletal muscle, blood — has a different set of answers,” High explains. High’s breakthrough clinical trial came in 2007. Twelve patients with LCA-related blindness, including four children, received a gene therapy injection in one eye and demonstrated dramatic improvement in vision. A follow-up study last year treated the second eye in the same patients, with similar success. The team now has approval for a new trial to treat both eyes simultaneously. If successful, the treatment could be submitted for Food and Drug Administration approval as soon as 2015, and treatments for numerous other retinal diseases could follow. High has also been working since the mid-1980s on gene therapy for hemophilia. The liver, where the blood’s clotting factors are made, is a trickier proposition. She led a clinical trial
One Cell at a Time in 2001 that partially cured a man with hemophilia B, however the effects lasted only four weeks. His body’s immune system rallied to defeat the replacement gene. High, described by her colleagues as “energetic” and “dynamic,” returned to her lab and kept working. In 2011, she was a collaborator in a study that took place in London that finally achieved the desired result: Six patients with hemophilia B are now producing enough clotting factor on their own to reduce or eliminate the need for clotting factor infusions. High hopes to extend this success with a new trial of 10 to 15 hemophilia patients that began at CHOP in January. A mother of three, an avid late-night reader and a World War II buff, High grew up in North Carolina. She considered becoming a chemist like her grandfather — he taught at Penn, and a photo of him boating on the Schuylkill River sits atop her office shelves — but instead pursued medicine. As a hematology resident, she became interested in hemophilia A and B, the genes for which had just been identified. “When I started in the 1980s, I thought it was going to be a big deal if we could just cure the disease in animals,” High says with a smile. “Now it’s much more fun because while there are still many problems that need to be solved, we know gene therapy works in certain subgroups of people. We are working to develop solutions that will allow us to extend these therapies to ever larger numbers of patients. It’s really exciting.” —julie sloane
opposite:
Adeno-associated viral vectors (actual size: 20 nanometers) deliver “corrected” DNA to the body’s cells. this page, top to bottom:
Katherine High, M.D., in her lab
To learn more about High’s gene therapy work for blindness, watch a video at childrensview.org.
High (center) with Penn research collaborators Albert Maguire, M.D. (left), and Jean Bennett, M.D., Ph.D. (right) Yannick Duwe, 13, was blind before receiving gene therapy, which restored much of his sight.
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JAMES SNYDER, RESEARCH TECHNICIAN
RAN lIN, VISITING STuDENT
MARY CONNEll, lAB DIRECTOR
On the Cusp
of a Cure
After 30 yeArS of reSeArch, one SUrgeon iS CLOsE TO A BREAKTHROUgH for SicKLe ceLL diSeASe.
THIS PAGE, TOP TO BOTTOM:
People with sickle cell disease have hard, sickle-shaped red blood cells (right) that are different from normal soft, round ones (left). Children who have sickle cell disease, like Shannon, 12, are often admitted to CHOP many times a year for fevers, pain and illness. OPPOSITE PAGE, TOP TO BOTTOM:
Alan Flake, M.D. (left), with Kim Smith-Whitley, M.D. (right), director of CHOP’s Comprehensive Sickle Cell Center Montana, 18 months, is one of more than 1,000 children seen at CHOP for sickle cell disease.
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IT’S A GOOD RULE OF THUMB that when it comes to sick kids, the sooner a problem is identified and treated, the better the prognosis. Many of CHOP’s patients are toddlers, infants or even newborns. But one group of surgeons and researchers at CHOP works on a whole different timeline: They treat babies before they are even born. In 1981, surgeon Alan Flake, M.D., was part of the very first open operation on a fetus still in the womb. Today, Flake is not only one of the world’s top fetal surgeons, but he is also director of the Center for Fetal Research at CHOP. There he has spent most of his 30 years in the field working on what could be one of the biggest medical breakthroughs in a generation: a cure for sickle cell disease.
Sickle cell disease affects millions of people worldwide and is the most common inherited disorder in African Americans, of whom 1 in 375 is born with the disease. Instead of the normal soft, red blood cells that most of us have, people with the disease have stiff, sickle-shaped cells that clump and block blood vessels, leading to a host of problems from debilitating pain crises and fatigue to potentially fatal heart attacks and strokes. Sickle cell patients are commonly hospitalized three or four times a year — every fever means straight to the Emergency Department. Heartbreakingly, most people with the disease will succumb to it before their 50th birthday. Although bone marrow transplants can cure sickle cell disease, the procedure is so risky that it’s usually considered worse than the disease. What Flake is developing could be a true cure — a way for children to be treated before birth and grow up completely symptom-free. Because red blood cells are made in bone marrow, the procedure involves transplanting stem cells from the mother’s bone marrow to the child. The first transplant is done during a special window in early pregnancy when the fetus’ immune system is not yet fully developed; at this stage, the fetus’ body tolerates its mother’s cells as if they are its own, avoiding the toxic chemotherapy typically required by bone marrow transplantation. After the baby is born, a second transplant cements the process. He or she will be able to produce normal red blood cells, without any symptoms of sickle cell disease. The procedure is so advanced that Flake is even helping design the instruments needed to do it, including a narrow needle to inject cells into
What flake is developing could be a true cure — a way for children to be treated before birth and grow up completely symptom-free. the fetus in a minimally invasive way. He plans to enter clinical trials for the procedure within three years and sees the potential for it to cure a broad range of related disorders, including thalassemias. Since 2007, the Albert M. Greenfield Foundation has been a major supporter of Flake’s work, but the doctor’s team still needs significant funding to make this pioneering research a clinical reality. To that end, CHOP Foundation created Hope on the Horizon, a volunteer-driven campaign working to raise those critical funds through private philanthropy. Together we can make a big difference for children with this devastating disease. —EuGENE MYERS
To learn how you can get involved, please visit hopeonhorizon.org. Spring 2013 | Children’s view | 13
WHAT CAUsEs dIsEAsE? WHY dO WE gET sICK? doUgLAS WALLAce’S AnSWerS coULd SpArK A reVoLUtion in MedicAL thinKing.
BEN TWEDDAlE, RESEARCH TECHNICIAN
FENGXIANG WANG, PH.D., RESEARCH ASSOCIATE
AlESSIA ANGElIN, PH.D., VISITING POSTDOCTORAl FEllOW
mighty mitochondria FOR THOUSANDS OF YEARS, practitioners of Eastern medicine have linked human health to the concept of energy, or “qi” — its presence, absence and flow through our bodies affecting everything from heart disease to mental clarity. Ironically, Douglas Wallace, Ph.D., one of CHOP’s most internationally renowned experts in Western medicine, has spent his career proving that they just might be right. Wallace studies mitochondria, tiny structures that serve as our cells’ “power plants,” converting food and oxygen into energy. Mitochondria are actually symbiotic bacteria that invaded our cells more than 2 billion years ago. Each of the body’s 100 trillion cells contains hundreds, or even thousands, of mitochondria, and each generates about 0.2 volts of potential energy — accounting for 90 percent of the energy the body needs. The health of our mitochondria, Wallace hypothesizes, is our health. In fact, Wallace passionately believes a whole spectrum of human disease — everything from diabetes to autism to obesity — is caused or influenced by the mitochondria. It is nothing short of a revolution in medical thinking, and as the evidence mounts, the world is taking note.
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“I really believe that life is about the interplay between anatomy and energy. Without energy, everything stops, so why shouldn’t that be the most important thing?” asks Wallace, who came to CHOP in 2010 as the director of the newly created Center for Mitochondrial and Epigenomic Medicine. “This is the vital force that people have been wondering about for probably three to 5,000 years. And it’s a straightforward biophysical process.” Wallace founded the field of human mitochondrial genetics in the late 1970s when he and his team made a fundamental discovery: Mitochondria, which have their own unique DNA (often abbreviated mtDNA) that is separate from the DNA in the nucleus of our cells, are inherited only from our mothers. Completely unchanged from mother to child, your mtDNA is the same as your mother’s mother’s mother. This remarkable discovery allowed Wallace to trace 200,000 years of human migration patterns around the world. In November, the Gruber Foundation awarded Wallace its prestigious Genetics Prize for his work in mitochondrial genetics, but for decades, he worked under a cloud of skepticism from the scientific community. That began to
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Prasanth Potluri, Ph.D., shows off frozen tissue samples at the Center for Mitochondrial and Epigenomic Medicine. Olga Derbeneva, Ph.D., prepares samples for study. THIS PAGE:
Mitochondria are the “power plants” of our cells. Douglas Wallace, Ph.D. (center), consults with his team.
turn around about 20 years ago when he published two landmark papers demonstrating that glitches in mtDNA cause disease. Even the smallest variation in mtDNA can cause a subtle change in mitochondrial energy output. Multiplied by trillions of mitochondria, this can have a tremendous impact on the body, particularly the brain, which requires more mitochondrial energy than any other organ. Leigh syndrome, for example, is a severe neurological disorder caused by a mutation in mtDNA.
brain function that disproportionately affects boys more than girls and is sometimes accompanied by gastrointestinal problems, immunological problems and other issues that might result from a lack of mitochondrial energy. “We might have a real window into helping a lot of kids that have trouble in school or have problems with behavior,” Wallace says. Wallace and his team are currently developing diagnostic tests that can more easily detect mitochondrial defects, using nothing more
it is nothing short of a revolution in medical thinking, and as the evidence mounts, the world is taking note. While diseases like Leigh syndrome are rare, a stunning paper published in Cell in October indicates that even common neurological conditions, even something as simple as learning disabilities, may connect to the mitochondria. Wallace’s study showed that by combining two normal mitochondrial DNAs in the laboratory mouse resulted in neurological disorders and learning problems. This finding may shed new light on one of the most common and most mysterious childhood disorders, autism, which affects 1 in 88 American children. Wallace says autism fits the profile of mitochondrial diseases: It is a subtle neurological problem in
than the patient’s breath blown into a can. He feels a strong sense of mission to change the way people think about medicine because the resulting treatments for disease could be game-changing for human health. He sees connections to mitochondria in everything from heart disease to obesity to diabetes to Alzheimer’s disease. “We’re on the verge of a major revolution of medicine,” Wallace says. “We’re going to change from an anatomically based medicine to an energetically based medicine, and more than half of all the complex diseases that we’re worried about right now may be solved that way.” —EuGENE MYERS
Spring 2013 | Children’s view | 15
A New Approach
A chop phySiciAn dIsCOVERs A nEW dRUg thAt MAy chAnge her pAtientS’ LiVeS.
to a Rare Disease
XAVIER ANGuElA, PH.D., POSTDOCTORAl FEllOW
MuNIR KHAN, RESEARCH TECHNICIAN
KIERSTIN KEllER, RESEARCH TECHNICIAN
“IT’S LIKE THE OPPOSITE OF DIABETES.” That is the simplest way
to explain congenital hyperinsulinism (HI), a rare disease in which the pancreas makes too much of the hormone insulin, causing blood glucose to plummet. Depriving the brain of the sugars it needs to function can damage it quickly, so identifying and monitoring kids with HI is urgent and active work. Of the approximately 100 children born with HI each year in the United
States, half seek treatment at CHOP’s Congenital Hyperinsulinism Center. In fact, the center’s expertise is so renowned that it draws children with HI from around the world. “In this country, CHOP is the only center that has a multidisciplinary team dedicated to caring for these children and doing research to improve the management of their condition,” says center director Diva De León, M.D. Currently, about half of HI cases can be treated with medication or cured
by surgically removing portions of the pancreas. For the other half, the only option is to remove most or all of the pancreas. It’s an imperfect solution; many of these children will need a feeding tube to control blood sugar until they’re 5 to 7 years old, and almost all will eventually develop diabetes. But De León and her colleagues are investigating an alternative approach that could revolutionize treatment for HI: a new drug that can help manage glucose levels in even the most severe
Defeating Cancer thAnKS to reSeArch on iMMUnotherApy, EMMA WHITEHEAd’s OWn BOdY KILLEd HER CAnCER.
“I’M SORRY, BUT WE’RE ALL OUT OF OPTIONS.” It’s the most heartbreaking thing the family of a sick child could hear, and a devastating moment for the doctor as well. At most hospitals, it’s a conversation Emma Whitehead’s family would have had with her oncologist. Despite every treatment in the book, Emma’s acute lymphoblastic leukemia (ALL), the most common type of childhood cancer, had relapsed twice. She was among the 15 percent of children with ALL whose disease is resistant to chemotherapy. At just 6 years old, she really was out of options. Fortunately, the Whiteheads had come to CHOP for a second opinion and learned of a clinical trial for patients just like Emma, that would use her own immune system to fight the cancer. CHOP’s robust, cutting-edge research program and its association with a large research university can be a great benefit to patients whose diseases have imperfect treatments. CHOP researchers largely focus on translational medicine — investigations that will yield new treatments for patients like Emma as quickly as possible. Under the supervision of CHOP oncologists Susan Rheingold, M.D., and Stephan Grupp, M.D., Ph.D., the study leader at CHOP, Emma enrolled in a clinical trial known as CTL019. It is an experimental treatment developed by Carl June, M.D., at the University of 16 | Children’s view | Spring 2013
Diva De león, M.D., with congenital hyperinsulinism patient Jennifer luu, 4.
forms of the disease — without surgery. It builds on a decade of research by De León to understand how insulin-secreting pancreatic cells work. Armed with that knowledge, she ultimately zeroed in on an investigational drug called exendin-(9-39), which showed success in decreasing insulin production in mice. Exendin-(9-39) is a modified form of exendin-4, a synthesized version of a protein derived from Gila monster saliva that is currently used to treat diabetes. The results of De León’s clinical trial, the first using this drug in humans, were published in the journal Diabetes. They showed that exendin-(9-39) successfully increased fasting blood glucose and inhibited insulin secretion in nine patients. This study, funded by grants from the National Institutes of Health and
Pennsylvania that had shown promising results in several adults. Emma was the first child anywhere to receive this treatment, which modified her own T-cells, a type of white blood cell that attacks invading viruses, so that they could recognize and destroy her cancer. It was experimental, her parents knew, and it might not work. But it was her only hope. It was not smooth sailing. After complaining of flu-like symptoms, Emma suddenly fell gravely ill and was rushed to intensive care. She spent her 7th birthday on a ventilator, heavily medicated. Her medical team found that levels of a certain protein in her blood were extremely high. They were so far into experimental territory that there were no guidelines on what to do. In a bid to save Emma, June proposed giving her a medication for rheumatoid arthritis that was also known to lower that protein. It was doctor’s
generous donations from the Lester and Liesel Baker Foundation and the Clifford and Katherine Goldsmith Foundation, provides proof of concept that will allow for larger studies in the future and hopefully FDA approval of the medication. The center is preparing an expanded clinical study for 2014. “It is a challenge to get funding for HI drug trials because pharmaceutical companies want to develop medications that can be used in larger populations,” explains De León. “A big part of what we have been able to do is because of philanthropic support. It allows us to do the research that we’re doing.” —EuGENE MYERS
For a video about the groundbreaking work being done at CHOP to treat HI, please visit childrensview.org.
intuition. And it worked. Almost overnight, Emma began to recover. Several weeks later, the Whiteheads got some terrific news: Emma was cancer-free. She has been able to return home, go to school, play soccer and walk her dog, Lucy. Along with her hair, her smile has returned. More testing will be needed to determine whether CTL019 is a viable, safe, long-term treatment for this type of cancer, but Grupp is optimistic about its potential in the future. By participating in the trial of CTL019 Emma’s life was saved, and she was able to make a big contribution to cancer research, paving the way for more children with no other option to find success with immunotherapy. —MARK JOllY-VAN BODEGRAVEN
To learn more about Emma’s story, watch the video at childrensview.org.
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Emma Whitehead, 7, is healthy today thanks to an experimental cancer therapy. Stephan Grupp, M.D., Ph.D., checks on Emma during her treatment. Grupp was the study leader at CHOP for the CTl019 trial that saved Emma’s life.
Spring 2013 | Children’s view | 17
The forces at Play
PRASANTH POTluRI, PH.D., SCIENTIST
OlGA DERBENEVA, PH.D., SCIENTIST AND lAB MANAGER
NADA ABDEl-MAGID, RESEARCH TECHNICIAN
c h o p RECR UITs H O CKEY TEA M s t o he Lp St Udy concUSSionS.
WHAT REALLY GOES ON IN THE HEADS OF TEENAGERS? Generations of parents have asked that question, but researchers at the Center for Injury Research and Prevention (CIRP) at CHOP are studying one important aspect of it literally: What happens when a teenager gets a concussion? A blow to the head, or even elsewhere on the body, can jostle the brain inside the skull. Whether this impact leads to a brain injury like concussion is determined by factors such as acceleration, rotation and angle. A new CIRP study, funded with grants from the National Science Foundation, the Toyota Motor Corporation and the National Highway Traffic Safety Administration, aims to better understand concussions by embedding sophisticated sensors in the helmets of 13-year-old ice hockey players, wirelessly monitoring every impact during games and clinically assessing the players’ health before, during and after the season. People commonly associate concussions with sports injuries, but only about 50 percent of concussions occur from sports, according to Kristy Arbogast, Ph.D., engineering core director at CIRP, who is conducting the study along with Mari Allison, a bioengineering Ph.D. candidate at Penn. There are many opportunities for concussions in everyday life, such as car accidents and falls; however, hockey players are ideal subjects for the study because they are more likely to experience high-impact hits. Analyzing the data they collect from hockey games and practices will help Arbogast and her team, which includes pediatric sports medicine specialists Christina Master, M.D., and Matthew Grady, M.D., understand what kinds of impact cause concussion and what factors affect a child’s recovery time. (Master has significant skin in To study concussions, Kristy Arbogast, Ph.D., the game: Her son is on one of the hockey teams being studied.) is using hockey helmets embedded with precision sensors that record impacts. They will also mine the data for ways to prevent, diagnose and manage concussions. “One of the unique things about concussion — and one of our big challenges — is that it’s not something you can see on an imaging test. You can’t look at it like a broken bone on an x-ray and say it exists or doesn’t exist, or it’s better or not better,” Arbogast says.
“one of the unique things about concussion is that it’s not something you can see on an imaging test.”
To learn more about how children recover from injuries, visit CIRP’s website, aftertheinjury.org.
Clinical assessment by an expert is crucial to diagnosing concussions. And because children’s brains are still developing, their concussions are different from adults’. Even children at different ages experience concussion differently. “Ultimately we hope to learn about what leads to concussion and either prevent the injury in the future or accelerate the child’s recovery when it does occur,” says Arbogast, “so they can get back to doing all those things that they were doing before: going to school, playing sports and generally leading active lives.” —EuGENE MYERS
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lEFT:
Senbagam Virudachalam, M.D., M.H.S.P. (right), checks in on cooks Kiana Freeman (left) and Nina Ollison (center). BElOW, lEFT TO RIGHT:
Virudachalam at CHOP Early Head Start in West Philadelphia Class participant Silver Black dices tomatoes for chana masala.
A Recipe for Healthier Children TO PREVEnT CHILdHOOd OBEsITY, A pediAtriciAn heAdS to the Kitchen FOR SOME RESEARCHERS, tools of the trade include pipettes, petri dishes and microchips. For Senbagam Virudachalam, M.D., M.S.H.P., the main ingredients are ginger, garlic, a cutting board and a skillet. On a Thursday morning, Virudachalam is at CHOP Early Head Start in West Philadelphia, teaching 15 young moms and dads how to make one of her favorite childhood dishes, a chickpea curry called chana masala. She peers over their shoulders, offering suggestions as they chop vegetables and measure spices. But this isn’t just a cooking class — it’s serious research. A third of all children in the United States are overweight or obese, and as a pediatrician, Virudachalam knows the importance of a child’s diet to prevent disease later in life. She cites a scary statistic: Half of all AfricanAmerican and Latino children born in the year 2000 will develop diabetes or heart disease, and diet is a big factor. But there’s more to healthy eating than just learning about nutrition. “For the most part, parents understand the difference between healthy and unhealthy food,” she says. “Where they struggle is integrating that knowledge into busy, complicated lives.”
As a fellow in Academic General Pediatrics, Virudachalam studies the barriers to healthy eating among families and looks for ways to overcome them. In studying the problem, she zeroed in on eating at home, where kids get 70 percent of their meals. “Over the past generation or two, there has been a shift away from preparing food in the home, either eating out or getting processed foods that are really easy to put together at home,” says Virudachalam. “Today’s parents don’t necessarily have the skills or confidence to be able to prepare fresh, healthy foods for their children at home.” Cooking classes are not new to Early Head Start, but this six-week series, supported by the American Beverage Foundation for a Healthy America, is specifically designed as an obesity intervention. Classes focus on simple dishes like eggs, skillet chicken, salads and turkey chili and incorporate practical lessons about shopping on a budget and buying seasonal vegetables. The goal is to give parents a foundation of skills where they feel more comfortable and confident in the kitchen. Importantly, the classes include a peer
mentor for every two students, as studies have shown that people who are successful in overcoming challenges in their community often have better solutions than those devised by outsiders. They can also support one another after the class ends. “I’ve seen people exchanging phone numbers at class, setting up play dates for their kids while the moms cook together,” says Virudachalam. “They can build relationships out of this activity, which is the way it used to be when more people cooked and cooking was a bigger part of day-to-day life.” As the students ate their chana masala together at the end of this, their final class, they reflected on what they had learned. “There were a lot of ingredients I never saw before, so this class really opened my eyes to a different culture of food,” said one woman. “It was a good beginning,” said another. “I have a very long way to go — I’m a beginner beginner — but I enjoyed it. It was very practical. We had adversity from day one, but each meal was completed and was a success. I think that’s a wonderful thing.” —JulIE SlOANE
For Dr. Virudachalam’s chana masala recipe, visit childrensview.org.
www.research.chop.edu/blog CHOP’s researchers have been driven to “go first and be first” for more than 80 years, making pioneering discoveries that are changing lives and changing the world. now you can “be first” to hear about their latest breakthroughs at Cornerstone, a new blog
that offers regular updates about new technologies, treatments and therapies — directly from the investigators who are doing this inspiring work at the Research Institute at CHOP.
Spring 2013 | Children’s view | 19
volunteers in philanthropy s ig natu re eve nts
The Children’s Hospital of Philadelphia Buddy Walk ® and Family Fun Day On Oct. 7, 3,500 people gathered at Villanova University Stadium for the 11th Annual Buddy Walk® and Family Fun Day. The event raised $260,000 for CHOP’s Trisomy 21 (Down syndrome) Program. Families enjoyed a day full of games, music, food, moonbounces and fun to promote acceptance and inclusion of people with Down syndrome. Northwestern Mutual – Eastern Pennsylvania was the lead sponsor.
Women’s Committee of The Children’s Hospital of Philadelphia Holiday Boutique The 47th annual Holiday Boutique hosted by the Women’s Committee of The Children’s Hospital of Philadelphia on Nov. 29 and 30 raised more than $350,000 to support patient care, research and education programs at CHOP. Trion was the presenting sponsor for the event, which attracted more than 750 attendees to the Merion Cricket Club in Haverford, Pa.
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“All In” for Kids Poker Tournament On Jan. 17, the annual “All In” for Kids Poker Tournament was held at the Mandarin Oriental New York. The event was rescheduled from Nov. 2 due to Hurricane Sandy. Susan and Don Wenger and Family served as honorary chairs, and poker legend Phil Hellmuth hosted. More than $784,000 was raised for the evening’s featured beneficiary, autism research and care at CHOP.
volunteers in philanthropy u PC O m i Ng eve NTS
THe 2013 PHilADelPHiA AuTO SHOW BlACk Tie TAilgATe PRevieW gAlA
57 T H A n n U A L Daisy Day® Luncheon Benefiting the Pain Management Program at The Children’s Hospital of Philadelphia
THURsdAY, MAY 2, 2013 Hyatt at The Bellevue Chair: Nancy A. Wolfson Honorary Co-Chairs: Tina Snider and the Snider Family On Jan. 18, 4,000 guests enjoyed cocktails, dancing and gourmet cuisine among the finest automobiles in the world at the Pennsylvania Convention Center in Philadelphia. The event, produced by the Auto Dealers CARing for Kids Foundation, the charitable arm of the Automobile Dealers Association of Greater Philadelphia, raised $450,000 for the Division of Gastroenterology, Hepatology and Nutrition.
daisydayluncheon.org Fashion show sponsored by Saks Fifth Avenue, featuring the Stella McCartney Fall 2013 Collection
The Children’s Hospital of Philadelphia Golf Classic See you on the links!
PHOTOS BY MARK BARAG
Monday, June 17, 2013 Seaview Golf Resort, Absecon, N.J.
giftofchildhood.org/golf Spring 2013 | Children’s view | 21
volunteers in philanthropy co m m u n ity eve nts fall - wi nt e r 2 0 1 2
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Connie Mac’s Cancer Sucks Fundraiser June 6, Connie Mac’s Irish Pub, Pennsauken Township, N.J. • The day of auctions, raffles, and food and drink specials raised more than $7,000 for the Cancer Center.
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Kayla’s Hope for Kids 12th Annual Golf Tournament Aug. 20, Talamore Country Club, Ambler, Pa. • The golf outing, held in memory of Kayla Romaelle, raised more than $50,000 for brain tumor research.
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Rowan University Engineers Field Day for CHOP Sept. 15, Rowan University, Glassboro, N.J. • Students in the College of Engineering at Rowan University organized a field day that included food, music and T-shirt sales, raising nearly $1,000 for the Cardiac Center.
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Rock ‘n’ Roll Philadelphia Half Marathon Sept. 16, Benjamin Franklin Parkway, Philadelphia • As members of Team CHOP, neurofibromatosis families completed the Rock ‘n’ Roll Philadelphia Half Marathon and raised more than $38,000 for the Neurofibromatosis Program.
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Rite Aid’s 50th Anniversary Sept. 27, Nationwide • In honor of its 50th anniversary, Rite Aid celebrated with “50 Acts of Wellness” across the country and delivered 100 new family kits to CHOP’s Garbose Family Special Delivery Unit.
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Christopher’s Cure 9th Annual Golf Tournament Sept. 28, Downingtown Country Club, Downingtown, Pa. • This golf tournament, dinner and silent auction in honor of Christopher Muth raised $35,000 for musculoskeletal tumor research at CHOP.
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Spike Down Cancer Oct. 19, Academy of Notre Dame de Namur, Villanova, Pa. • Academy of Notre Dame de Namur volleyball players teamed up with the Miriam’s Kids Research Foundation for a volleyball match that raised more than $1,500 for brain tumor research.
volunteers in philanthropy co m m u n ity eve nts fall - wi nt e r 2 0 1 2
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Walk for Hope Oct. 21, Veterans Park, Hamilton Township, N.J. • Preston Elliot was the honorary chair for the 4th Annual Walk for Hope sponsored by the IBD Family Research Council. More than $68,000 was raised for the IBD Center at CHOP.
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Ron’s Gardens Hayrides for Hope Oct. 27, Ron’s Gardens, Hammonton, N.J. • The day raised $3,700 for CHOP and included a corn maze, hayrides, farm tours and a pumpkin carving contest.
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Marriott’s Monte Carlo Night Nov. 6, Courtyard Marriott Downtown, Philadelphia • Marriott Business Council hosted its 7th Annual Monte Carlo Event, raising $96,000 for Children’s Miracle Network at CHOP.
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PhilaU Thon Nov. 9, Philadelphia University, Philadelphia • Students at Philadelphia University hosted their first annual Dance Marathon, which raised more than $8,000 to support Children’s Miracle Network at CHOP.
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Giant Food Stores – Outstanding Corporation of the Year Nov. 14, Loews Philadelphia Hotel, Philadelphia • Giant Food Stores was recognized as the Outstanding Corporation of the Year at the 2012 Association of Fundraising Professionals (AFP) National Philanthropy Day Luncheon. Rick Herring, president of Giant Food Stores, accepted the award from AFP Greater Philadelphia board chair Jaime Howard.
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American Heritage Federal Credit Union Check Presentation Dec. 3, Children’s Seashore House, Philadelphia • American Heritage Federal Credit Union with the Kids-N-Hope Foundation presented a check for $100,000 to support the Music Therapy Program.
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Craft Show for a Cause and Lunch with Santa Dec. 8, The Gin Mill and Grille, Northampton, Pa. • The holiday craft show raised more than $1,000 for the Center for Fetal Diagnosis and Treatment.
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volunteers in philanthropy co m m u n ity eve nts fall - wi nt e r 2 0 1 2
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Excel Academy North Family Night Dinner and Auction Dec. 13, Excel Academy North, Philadelphia • The dinner and auction event raised nearly $5,000 for CHOP.
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Kohl’s Cares Check Presentation Dec. 13, Kendrick Recreation Center, Philadelphia • CHOP received a $620,436 donation from Kohl’s, through its Kohl’s Cares® program, to support the Hospital’s injury prevention programs.
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Conestoga Bank’s Sharing Card Check Presentation Dec. 19, Liberty Place, Philadelphia • Conestoga Bank donates to CHOP any balance from its Visa Sharing Cards unused after 18 months. This year’s donation totaled $8,000.
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Drop for CHOP Jan. 8, Varga Bar, Philadelphia • Varga Bar General Manager Rich Colli accepted a weight loss challenge fundraiser, losing an amazing 33.2 pounds and raising more than $3,000 for CHOP.
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Kortney Rose Foundation Check Presentation Dec. 21, Oceanport, N.J. • The Kortney Rose Foundation presented its sixth annual donation to CHOP: a $100,000 gift that will support brain tumor research. This year’s events included Team Kortney, Kortney’s Challenge, Day at the Races and Turning Point fundraisers.
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New Hope-Solebury Dance-A-Thon Jan. 11, New Hope-Solebury High School, New Hope, Pa. • Fourteen teams participated in a 12-hour dance-a-thon that raised $6,000 for the Cancer Center.
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We saved a baby from a lifetime of disability in an unusual operating room.
His mother’s belly. GO BEHIND THE BREAKTHROUGHS: LEARN ABOUT THIS AND OTHER ADVANCES BEING PIONEERED TODAY AT THE CHILDREN’S HOSPITAL OF PHILADELPHIA.
C H O P. E D U / F E T A L - B T B ©2013 The Children’s Hospital of Philadelphia, All Rights Reserved.
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THINK
BIG The Children’s Hospital of Philadelphia has been a leader in pediatric medicine for 158 years. That’s because from the very start, CHOP’s mission has been to explore big ideas that can benefit children everywhere.
Please join our lasting effort to make the world a healthier and safer place for children. Consider naming CHOP in your will or as a beneficiary of a retirement plan. These gifts won’t affect your current cash flow and may even help reduce taxes for loved ones. Help us continue to think big. Create your own legacy of hope and healing by visiting giftofchildhood.org/plannedgiving. Or contact Tom Yates, director of planned giving: 267-426-6472 or yatestg@email.chop.edu.
GIFT of CHILDHOOD.org/plannedgiving
Jason
Age 8 months | Nephrology