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COMMUNITY REPORT 2015-2016 A PLACE WHERE EVERY CHILD CAN ACCESS THE FINEST, MOST ADVANCED MEDICAL CARE FROM SOME OF THE TOP DOCTORS IN THEIR FIELDS. IMAGINE A PLACE WHERE RARE IS FAMILIAR. WHERE THERE’S NO SUCH THING AS UNTREATABLE, UNIMAGINABLE OR IMPOSSIBLE. WHERE EVERY DAY, NEVER-BEFORE-SEEN TREATMENTS BECOME THE GOLD-STANDARD AND OUR PHYSICIAN-SCIENTISTS ARE UNLOCKING NEW POSSIBILITIES FOR FUTURE GENERATIONS OF CHILDREN. IMAGINE A PLACE WHERE ANYTHING IS POSSIBLE. President John Kennedy lit America’s imagination on fire with his bold vision of landing astronauts on the moon in a decade’s time. Imagination is what a child employs when she tells an adult she wants to be a veterinarian or a princess. It’s what our Child Life Department taps into when they do medical play with patients to help them understand a procedure they’re about to undergo. At Children’s Hospital of Pittsburgh of UPMC, and free from stigma. And each is made possible, we value imagination. We want to think big. or made better, with philanthropic support. We want our physicians, families, staff members, volunteers, and donors to think big. “Thinking In short, we couldn’t fulfill our mission without big” developed the polio vaccine, introduced the financial support that comes from our and perfected organ transplantation as a community, both in big and small ways. Your lifesaving therapy, and discovered a cure generosity gives our physicians and researchers for a rare liver disease. the chance to dream. It gives parents the space to hope, at what could be their moment of In this Community Report, you’ll meet some greatest need. It gives our children the ability to of Children’s Hospital’s most imaginative imagine a future like any other child’s. is what a child employs thinkers. They are physicians, scientists, and administrators, and in big and small ways they’re So with gratitude, we ask you to enjoy this changing the world around us. They are fighting glance back at our achievements through brain cancer with a vaccine, regenerating fiscal year 2016. Then join us with eyes diseased heart tissue, and treating kids’ and teens’ forward as we imagine what we might mental health issues in a way that’s full of respect accomplish next. J. Gregory Barrett, President Christopher Gessner, President Children’s Hospital of Pittsburgh Foundation Children’s Hospital of Pittsburgh of UPMC HEALING BROKEN HEARTS A FAMILY HOLDS CHILDREN’S CLOSE TO THEIR HEART When John and Cathy Pelusi’s youngest child, Jodie, came to Children’s for surgery to correct her ventricular septal defect (VSD), a hole in the wall separating the two lower chambers of the heart, she was only 6 years old. The Pelusis say the level of care was outstanding — they realized how lucky they were to have Children’s skilled doctors and nurses repair Jodie’s heart defect, and they knew that other children with even more serious issues were being cured. Today, Jodie is a thriving, caring young adult with a big heart, working as a Peace Corps volunteer in Cameroon. Her family remains grateful for the wonderful cardiac care she received. For more than 15 years, the Pelusi family has supported the Cardiac Research Fund in honor of Jodie to honor Children’s and the wonderful things it does in the community. Bernhard Kühn, MD THE RICHARD KING MELLON FOUNDATION INSTITUTE FOR PEDIATRIC RESEARCH ENABLES PROMISING PHYSICIAN-SCIENTISTS TO PURSUE LONG-TERM, AND SOMETIMES LONG-SHOT, GROUNDBREAKING MEDICAL ADVANCES. IN SHORT, THE PROGRAM BROKEN FUNDS PHYSICIAN-SCIENTISTS WITH BIG DREAMS. Bernhard Kühn, MD, is a Mellon Scholar with a Dr. Kühn realized this stimulant potentially “Regenerative medicine, by definition, should colossal dream: to help the heart regenerate. could be used in therapies to trigger heart cells help you get better permanently. When you absent through disease or congenital defect, come off the medicine, you should stay better,” “As a field, we understand how to fix faulty to grow anew. he says. “This is a game changer in how heart blood flow in the heart,” says Dr. Kühn, “But disease is dealt with because we have the what we have not figured out is how to restore Now, Dr. Kühn is working toward that goal. potential to stop the progression and partially heart muscle that is damaged, diseased, or His lab has already yielded insights into the revert the disease.” might not have grown.” growth mechanisms of heart cells. The next step will be to develop strategies for clinical When asked where he would imagine his work Scientists have long believed that, after a trials in children with damaged hearts or could lead, he responds simply, “Saving lives. baby is born, the human heart cannot certain congenital heart defects. Dr. Kühn How many times does a researcher get to make regenerate itself. says the drug is most likely to be effective in a direct impact on patient care? For many the youngest patients, whose hearts are still researchers, once in a lifetime; for most, never. developing. If I can help bring a new therapy to children That was, until a few years ago, when Dr. Kühn — that would be something I could look back published a landmark paper in the prestigious on and say, this was a professional life worth journal Cell, which showed heart muscle cells “We’re hoping developments continue to living.” do proliferate during early development be positive, we maintain a good safety profile, after birth — raising the possibility that and to have clinical trials soon. But this drug scientists could stimulate new cell growth has theoretical, not proven benefits, so we in human infants. must be very careful. We want to be quick, but we must be safe,” he says. He identified the growth factor neuregulin-1 as the stimulant that encourages heart muscle While Dr. Kühn is cautious about the cells to divide. overstating the benefits, he admits the drug has the potential to be revolutionary. IMAGINEHARNESSING THE POWER OF A CHILD’S IMMUNE TO FIGHT THE SCARIEST SYSTEM BRAIN TUMORS Ian Pollack, MD WWE FAN INSPIRES HEAVYWEIGHT RESEARCH WWE (World Wrestling Entertainment Inc.) executives Stephanie McMahon and Paul “Triple H” Levesque’s lives were forever changed by super fan Connor “The Crusher” Michalek, an 8-year-old battling a rare brain and spinal cord tumor called medulloblastoma. When Connor lost his battle to medulloblastoma, Stephanie and Paul decided to keep the fight going by creating Connor’s Cure. In 2015, 1,328 donors, including WWE fans across the globe, contributed $578,219.56 to Connor’s Cure. Today, in partnership with The V Foundation for Cancer Research, Connor’s Cure provides support to leading research hospitals from across the country including Children’s Hospital. WHAT IF A VACCINE COULD BOOST THE BODY’S ABILITY TO FIGHT CANCER? IMAGINE THE HOPE IT WOULD BRING TO THOUSANDS OF CHILDREN WHO WEREN’T RESPONDING TO STANDARD THERAPIES, AND THEIR FAMILIES. (Left to right) Brent, Olivia, Lance, and Christine Hupkovich. Lance is a participant in one of the vaccine trials. Sounds like pie-in-the sky stuff, but Brain tumor treatment is very difficult. One little girl from Minnesota has responded immunotherapy is here. And for years, Chemotherapy and radiation treatment can really well to immunotherapy. She’s now 2. “The Children’s Hospital of Pittsburgh of UPMC has only do so much, and surgery often is not an tumor has shrunk over time, and she’s been been leading the way. option. Immunotherapies show promise for able to walk and get better since being on the brain tumors that are difficult to treat. vaccine. It’s been wonderful to see.” In 2009, the lab of Ian Pollack, MD, chief of the Division of Pediatric Neurosurgery, first Unlike chemotherapy, immunotherapy has few What does the future hold for immunotherapy? identified antigens on tumor cells that could be side affects. The most common are a minor rash Maybe it will become a first-course therapy used as a vaccine. When an early study of this at the injection site and fever within the first for brain tumors, instead of a last-ditch effort. approach proved successful in adults, the team 24 hours. Maybe it will work synergistically with other moved on to kids. standard therapies. Whatever the case may be, Pediatric patients come from all over the Children’s is committed to moving the Today, Children’s Hospital has three open nation to participate in Children’s clinical trials, needle forward. clinical trials — for high-grade glioma, including Oregon, the Midwest, and North low-grade glioma, and ependymoma — all Carolina. Since Dr. Pollack’s first trial, more than types of brain tumor. To qualify, a patient must 70 patients, ages 1 to 21, have enrolled. have failed up to five other therapies. STRONG ALLIES The Alliance for a Healthier Generation, founded by the American Heart Association and the Clinton Foundation has been an instrumental partner in initiating the Healthy Schools program locally. The Alliance takes a comprehensive approach to building healthier learning environments and offers free technical support to participating schools. E G TS O A H N W A for their efforts? Pittsburgh Arsenal 6–8 Pittsburgh Arsenal PreK–5 Pittsburgh Brashear 9–12 Pittsburgh Dilworth PreK–5 Pittsburgh Faison K–5 Pittsburgh Langley K–8 Pittsburgh Minadeo K–5 Pittsburgh Montessori PreK–5 Pittsburgh Sterrett 6–8 Pittsburgh Woolslair PreK–5 WHAT IF ALL STUDENTS HAD ACCESS TO ENVIRONMENTS THAT PROMOTED HEALTHY EATING AND ACTIVE LIVING? Megan Perfetti is a Brashear health and NO WHAT IFS. WE’RE physical education instructor who supervises health and physical education in the district.

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