After a lifetime of innovation and care, world-renowned pediatric orthopaedic surgeon Robert Campbell, MD, passed away peacefully in July. A true innovator, Dr. Campbell’s thought-leadership and inventions changed the care for children with complex and life-threatening spine and chest wall deformities. He is best known as the inventor of the vertical expandable prosthetic titanium rib, or VEPTR, the first device approved by the FDA to treat thoracic insufficiency syndrome (TIS), a rare congenital condition affecting children in which the thorax cannot support regular growth or breathing. By separating the ribs and helping to straighten the spine, the VEPTR is designed to give children’s lungs room to grow, allowing them to breathe without the aid of ventilators. Left untreated, TIS can be devastating. As children with TIS grow, the condition causes the chest wall to become deformed and can ultimately lead to death due to respiratory insufficiency. However, since Dr. Campbell implanted the first VEPTR in 1989, the device has proven to be a lifesaver. His invention has become the standard of care throughout the world, saving or extending the lives of children with previously untreatable conditions.

Dr. Campbell led a Journal of Pediatric Orthopaedics study that showed the VEPTR treatment improved Jeune syndrome (a severe form of TIS) patients’ survival to nearly 70 percent, compared to a 70 to 80 percent mortality rate without treatment.

But his contributions didn’t stop with the development of VEPTR. After joining Children’s Hospital in 2008, he launched CHOP’s Center for Thoracic Insufficiency, attracting children from around the world, many of whom were told "nothing can be done." He created a team approach to care, collaborating closely with pulmonologists, thoracic surgeons, intensivists and radiologists to optimize treatment of children with severe spine and chest wall deformities. In addition, his innovation formed the basis of a new CHOP Frontier Program to improve outcomes for patients with TIS.

Dr. Campbell traveled worldwide, training surgeons in the use of VEPTR and assisting them with their most difficult cases while consistently and compassionately caring for patients and their families. He was the recipient of numerous awards for his innovations over the course of his career, including the Life Sciences Pennsylvania's (formerly Pennsylvania Bio) Patient Impact Award. Shortly before his passing, the National Organization for Rare Disorders Inc. honored him and his pioneering work with their Lifetime Achievement Award.

The past several decades have witnessed a dramatic shift in the landscape for how curable some pediatric cancers are, especially acute lymphoblastic leukemia (ALL), which was once incurable and now has about a 90 percent cure rate. ALL has been the central focus of the research of Stephen P. Hunger, MD, and his pioneering investigations led the American Society of Pediatric Hematology/Oncology (ASPHO) to award him with the George R. Buchanan Lectureship Award in May 2018.

The award had a particularly special meaning for Dr. Hunger, who serves as chief of the Division of Oncology and director of the Center for Childhood Cancer Research at CHOP. That’s because Dr. George R. Buchanan— past president of ASPHO and a renowned pediatric hematology physician-researcher — was supportive of Dr. Hunger early in his career. “I was both flattered and honored,” Dr. Hunger said of the distinction. “He helped me to get established nationally.” Dr. Hunger accepted the award at the ASPHO Conference in May, where he personally thanked Dr. Buchanan and presented a lecture on improving survival for children and young adults with acute lymphoblastic leukemia.

Dr. Hunger shared his perspective on receiving the award and the future of pediatric cancer research, enthusiastic about the promise of more targeted therapies that have a better “therapeutic index” — meaning they have much more specificity in terms of leukemia cell killing while sparing the normal cells and therefore reducing the side effects to treatment. He also expressed hope about incorporating immunotherapy into ALL treatment to perhaps lead to better cure rates and potentially allowing physicians to move beyond some aspects of current therapy.

Cutting-edge neuroscience techniques are leading to a better understanding of the brain’s role in controlling bladder function, and the novel insights of one CHOP investigator earned him special recognition from the neuro-urology community.

Jason Van Batavia, MD, a urologist and physician-scientist in the Division of Urology, was named the grand prize winner in the 2018 Diokno-Lapides Essay Contest. His manuscript described a research project focused on optogenetic stimulation of specific neurons in a section of the brainstem called Barrington’s nucleus, which scientists think is an important “command center” for controlling voiding (urination).

Identifying these neurons’ role in voiding could help scientists to figure out new approaches to treating voiding dysfunction in children. Forty percent of children — that’s around 2,000 new patients a year — who visit CHOP’s DOVE Center for Voiding and Bladder Function have lower urinary tract symptoms, which include high urination frequency or leakage of urine during the day or night.

“Those are some of the most common problems that we see in the pediatric urology world,” said Dr. Van Batavia, who also is a clinical instructor of Urology at the Perelman School of Medicine at the University of Pennsylvania. “And yet our understanding of them hasn’t changed much in the last few decades.”

The optogenetic stimulation approach Dr. Van Batavia employed involves using light to selectively control cells of interest. The results of his study point to the CRH expression in the brain, and CRH receptor activity may be a key factor in some dysfunctional voiding patterns. These novel insights led him to receive the prestigious prize May 19 at the American Urological Association Annual Meeting. Dr. Van Batavia’s work involved mentoring and guidance from Stephen Zderic, MD, pediatric urologist at CHOP; Rita Valentino, PhD, former director of the Stress Neurobiology Division within the Department of Anesthesiology at CHOP, as well as support from the Division of Urology under the leadership of Douglas Canning, MD, chief of the Division of Urology at CHOP.

Throughout his decades-long career as a researcher, author, professor, vaccine advocate, and strident spokesperson for accurate science, Paul Offit, MD, has been the recipient of numerous awards. In 2018 he added “gold medalist” to his vibrant list of honors and accolades, when he received the 2018 Albert B. Sabin Gold Medal from the Sabin Vaccine Institute.

The Sabin Gold Medal recognizes individuals who have made significant contributions in vaccinology or complementary fields. The annual award commemorates the legacy of Dr. Albert B. Sabin, renowned scientist and inventor of the oral live virus polio vaccine that nearly eradicated polio worldwide.

As the 25^th recipient of the Sabin Gold Medal, Dr. Offit received the award for his co-invention of an oral rotavirus vaccine, and his leadership as “one of the United States’ most vocal and dedicated advocates for immunization,” according to the Sabin Vaccine Institute.

“It’s an honor to join the distinguished ranks of Albert B. Sabin Gold Medal recipients,” Dr. Offit said. “Though our research may have focused on different diseases, we all share Dr. Sabin’s commitment to reducing human suffering.”

Dr. Offit, who directs the Vaccine Education Center at Children’s Hospital and holds the Maurice R. Hilleman Chair of Vaccinology, worked tirelessly with his colleagues on the vaccine for 26 years before it was brought to the marketplace, where it quickly proved its effectiveness and has since reduced child hospitalizations from rotavirus by 85 percent.

Beyond his work on vaccines, Dr. Offit is equally renowned for speaking up and writing about the importance of clear science communication and critical thinking, particularly when it comes to central issues in children’s health like vaccine safety and immunization.

Winning a trio of major distinctions in one month is certainly a rare feat, clinical geneticist Elizabeth Bhoj, MD, PhD, modestly admits, but one she is proud of, and so are we. In May, Dr. Bhoj, whose area of expertise is in the field of translational genomics, earned the William K. Bowes Jr. Award in Medical Genetics, the Burroughs Wellcome Fund’s Career Awards for Medical Scientists with a $700,000 prize, and the Society for Pediatric Research’s (SPR) Physician Scientist Award.

Dr. Bhoj received the SPR award in Toronto at the Pediatric Academic Societies Meeting where she presented the lecture “Novel Gene Discovery in a Cohort of Patients with Craniofacial and Neurologic Syndromes.”

She has conducted soon-to-be-published research on histone 3.3. Histones are proteins that wrap around DNA to keep it organized. Dr. Bhoj and colleagues found that a mutation in this particular histone is associated with a novel phenotype of developmental delay, neurodegeneration, epilepsy, and facial dysmorphism.

In other research, Dr. Bhoj has played a key role in the CHOP team who discovered a new growth disorder, Mulchandani-Bhoj-Conlin Syndrome. Named in recognition of their work, the condition — characterized by failure to thrive, severe short status, and profound feeding difficulties — is caused by an abnormality of chromosome 20.

Referring to the award triple-play, Dr. Bhoj said, “Having this vote of confidence is even more important than the financial support. Getting the feedback of, ‘Yes, we believe in you; this work has value, and it should continue,’ is an enormous career boost.”

Whether from a mentor or the larger scientific community, recognition and support go a long way toward propelling the careers of new investigators working to establish research programs that may lead to tomorrow’s breakthroughs.

With a dedication toward advancing the biomedical sciences, the Burroughs Wellcome Fund (BWF) is an independent foundation that provides highly competitive awards to investigators in the early stages of their research careers. One of those annual awards is the Pathogenesis of Infectious Disease (PATH) Award, which this year BWF awarded to Jorge Henao-Mejia, MD, PhD, assistant professor of Pathology and Laboratory Medicine at CHOP.

The PATH Award provides investigators at the assistant professor level with the opportunity to bring multidisciplinary approaches to the study of human infectious diseases. Supported by a $500,000 grant over five years, Dr. Henao-Mejia is continuing his investigation of the critical role that the microbiota — that vast population of bacteria and other microorganisms living within the human gastrointestinal tract — play in obesity and type 2 diabetes.

It’s well-established that the microbes in our gut help to regulate a number of physiological processes and, when dysregulated, can contribute to the development of highly prevalent disorders. Factors like lifestyle, age, diet, antibiotic exposure, and genetics can all contribute to an individual’s unique microbiota composition.

But despite recent advances in its role in disease, the detailed mechanisms by which the microbiota promotes obesity and type 2 diabetes are still poorly understood, a gap in knowledge Dr. Henao-Mejia is working to fill with the support of the PATH Award. Besides the award’s financial support, Dr. Henao-Mejia said that being selected for the award holds special meaning when it comes to his career trajectory, too.

“To be supported for projects that are considered high-risk, high-reward is very important when [a researcher] is starting an independent career,” he said. “I think it’s very valuable that BWF is willing to invest in young investigators, and it’s a wonderful initiative. I feel very lucky, privileged, and grateful for being part of a select group of people.”

It takes the right mix of innovative thinking, passion, and persistence for young investigators to succeed in research. But another key ingredient for the kind of success that can lead to tomorrow’s discoveries is the support these investigators receive early in their careers. Such support can take numerous forms, including recognition and awards from the scientific community.

The Burroughs Wellcome Fund (BWF), an independent foundation dedicated to advancing biomedical science, bestowed its prestigious Career Award for Medical Scientists (CAMS) to Sarah Henrickson, MD, PhD, for her groundbreaking work on how the immune system functions in pediatric disease, in particular inflammatory diseases like asthma and obesity. Specifically, Dr. Henrickson addresses the question of whether obesity might affect how a child with asthma responds to a vaccine.

The CAMS Award supports scientists early on in their careers, providing significant funding over a five-year period to physician-scientists on an academic career track and who are in the early years of their research careers.

With the support of the CAMS Award, Dr. Henrickson aims to start her own independent research group and continue to identify new hypotheses of mechanisms of immune dysfunction in pediatric disease while starting to validate the mechanisms found in her earlier work. As an allergist/immunologist, Dr. Henrickson’s overarching goal is to improve the outcomes of the patients she sees in the clinic based on a more mechanistic understanding of what underlies their different diseases.

“I’m really focused on bringing together different kinds of data, from clinical to genomic, to learn as much as possible, and always going back to the clinical data so that we’re relevant to the issue of the patient and their disease,” Dr. Henrickson said.

Dr. Henrickson was one of two CHOP investigators to receive the CAMS award this past year, the second going to clinician-researcher Elizabeth Bhoj, MD, PhD, for her research in translational genomics. BWF also recognized Jorge Henao-Mejia, MD, PhD, assistant professor of Pathology and Laboratory Medicine at CHOP, with its Pathogenesis of Infectious Disease Award.

Also during the fiscal year, Dr. Henrickson received a 2018 Faculty Development Award from the American Academy of Allergy, Asthma and Immunology Foundation for her work into immunometabolic alterations in primary immunodeficiency.

The remarkable accomplishments of two neonatal research and medicine pioneers took center stage in May 2018 as the Division of Neonatology celebrated the careers and retirements of Barbara Schmidt, MD, and Haresh Kirpalani, MD. The Division held a clinical research symposium that brought together the world’s leading experts in neonatal research and evidence-based medicine, many of whom had worked closely with Drs. Schmidt and Kirpalani as trainees, co-authors, and colleagues in the last few decades.

Together, Drs. Schmidt and Kirpalani devoted more than 30 years to driving evidence-based neonatal research forward and training the next generation of the field’s researchers. Dr. Schmidt, who was an attending neonatologist at CHOP and director of Neonatology Clinical Research at Penn Medicine, had led a host of collaborations and clinical trials in newborns, including the International Trial of Caffeine for Apnea of Prematurity. In 2017, the American Academy of Pediatrics honored Dr. Schmidt with the William A. Silverman Lectureship Award, and in 2015, she received the prestigious Order of Canada.

Dr. Kirpalani, who was an attending neonatologist at CHOP and Emeritus Professor CE of Pediatrics at Penn Medicine, made critical contributions to advancing neonatal clinical trials as well as to ethics and decision-making in newborn medicine. Dr. Kirpalani co-founded the International Society for Evidence-Based Neonatology, a nonprofit organization that promotes the belief that neonatal care should be firmly built on the best available evidence. In 2015, Dr. Kirpalani was named CHOP Mentor of the Year.

The symposium in their honor featured investigators from institutions around the world and concluded with another honor when Eric Eichenwald, MD, chief of the Division of Neonatology, announced the new Schmidt-Kirpalani Mentorship Award. The award, to be presented annually, is an ode to the extensive mentoring work of Drs. Schmidt and Kirpalani and celebration of their decades-long research legacy.

His groundbreaking research over nearly two decades has led to numerous awards for Stephan Grupp, MD, PhD, a pediatric oncologist who maintains an unwavering commitment to improving the lives of children battling different forms of cancer by changing the standard of care for these diseases. He recently added another honor to his growing list when the Citizen Diplomacy International of Philadelphia, a nonprofit international relations organization, awarded him with the 2018 Citizen Diplomat of the Year Award.

The award recognized Dr. Grupp for “raising Philadelphia’s international profile through his innovative advancements in medicine and his commitment to ensuring that people all around the world have access to this treatment.”

In collaboration with the Perelman School of Medicine and Novartis, Dr. Grupp led U.S. global clinical trials of the innovative life-saving chimeric antigen receptor (CAR) T-cell therapy for children with advanced acute lymphoblastic leukemia. This breakthrough garnered international attention and brought newfound promise to those with cancer, and in August 2017 the Food and Drug Administration approved this game-changing cellular therapy to treat cancer with a patient’s own immune system. Tisagenlecleucel, the first therapy and the first engineered cell therapy ever approved by the agency, targets patients age 25 and under who have an aggressive form of this type of leukemia, the most common cancer of childhood.

Dr. Grupp directs the Cancer Immunotherapy Program at CHOP, as well as the translational research of the Center for Childhood Cancer Research and the Stem Cell Laboratory. He continues his innovative research into new cell therapies while maintaining an active schedule with his patients, and traveling the world to educate doctors and other providers about safe use of this new cancer immunotherapy.

The National Kidney Foundation’s National Young Investigator’s Forum proved to be a powerful and rewarding experience for Melissa Meyers, MD, for two reasons. First, she won first place in the clinical research section for her talk about increased risk in children receiving renal transplants. Second, the third-year nephrology fellow sat in exclusive company as the only pediatrician in the room.

“Increased Risk” refers to a donor’s increased risk of transmitting a blood-borne infection — especially hepatitis B and C and HIV. This broad Increased Risk category, which has evolved over the years, now could contain anything from a history of incarceration for more than 72 hours over the last year to IV drug use, an unfortunate result of the nation’s current opioid epidemic.

About 20 percent of the deceased donor pool is flagged nationally as Increased Risk, and in some regions the rate jumps to nearly one-third. Despite having a lot of information on how adults receive these organs, there is a paucity of similar information for children, and Dr. Meyers wanted to see how often kids receive these organs. What she found was that Increased Risk kidneys were more frequently transplanted into adult patients. Her study led the National Kidney Foundation to give her top honors during their April 2018 forum after an open competition judged by independent clinical experts.

In her talk during the event, Dr. Meyers expressed her enthusiasm to focus on what kids are going through, and to share those insights with practitioners who primarily care for adult patients.

“I told the audience, ‘I want to take you in another direction and look at things through a different lens. We want to protect our kids as much as possible and offer them the best possible organs,’” Dr. Myers said.

Children’s Hospital of Philadelphia’s Ignacio Tapia, MD, was humbled to receive the first Carole L. Marcus Outstanding Achievement Award from the American Thoracic Society (ATS). The award is named in honor of Dr. Tapia’s mentor and CHOP colleague who was a leader in pediatric sleep medicine research.

Dr. Marcus, who died in 2017, served as director of CHOP’s Sleep Center and the Clinical and Translational Research Center/Center for Human Phenomic Science. She was associate director of the Institute for Translational Medicine and Therapeutics at the University of Pennsylvania. Dr. Tapia and Dr. Marcus frequently collaborated on research projects and publishing papers reporting their results.

Dr. Tapia, an attending pulmonologist in the Division of Pulmonary Medicine at CHOP, is committed to helping shape the future of pediatric pulmonary and sleep medicine. His research focuses on understanding the central nervous system complications of obstructive sleep apnea (OSA) in children. He also serves as the director of CHOP’s Pulmonary Medicine Fellowship Program and is an assistant professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.

Dr. Tapia accepted the award in May at the 2018 ATS International Conference in San Diego. “It was a great honor to receive this award named after my mentor. I am working hard every day to live up to the expectations,” he said.

The path to becoming an internationally recognized bioengineer with a focus on brain injury mechanics wasn’t straightforward for Kristy Arbogast, PhD. She had to find creative ways to apply her scientific skills as a non-clinician researcher at a pediatric hospital. Today, when she mentors research trainees with diverse backgrounds and interests, she not only teaches them how to conduct excellent research, but also how to be adaptable and resourceful.

In recognition for being a consummate researcher-teacher who has influenced countless undergrads and mentored dozens of graduate students, post-docs, and fellows, Dr. Arbogast received the Award for Excellence in Mentoring Research Trainees. The award recognizes faculty members who effectively guide the training and professional development of early career investigators.

Her mentoring style, as reflected in one of her nomination letters, gives guidance while encouraging young researchers to pursue their independence: “An effective combination of autonomy and supervision is difficult for any mentor; however, Professor Arbogast is able to achieve the appropriate balance, which in my case allows me the freedom to explore my own research interests within the confines of a larger project.”

Dr. Arbogast knows firsthand the value mentors have in shaping how CHOP investigators learn to interrogate a scientific problem, analyze the data, and use the results to improve the world. In 1997, she was one of the first research scientists hired by Flaura Winston, MD, PhD, and Dennis Durbin, MD, MSCE, who were starting the Center for Injury Research and Prevention (CIRP). Dr. Arbogast also had excellent mentorship from Kathy Shaw, MD, MSCE, who was chief of the Division of Emergency Medicine when Dr. Arbogast started her faculty appointment.

“All of these individuals as mentors treated me as a person who could have a substantial contribution,” Dr. Arbogast said. “I never once felt that my voice wasn’t heard or they weren’t interested in what I had to say. That carries over to how I conduct my mentor-mentee relationships. I want to hear from the mentees and welcome what they have to say.” Dr. Arbogast is the co-scientific director and director of Engineering at CIRP, and co-director of the Center for Child Injury Prevention Studies. She also is R. Anderson Pew Distinguished Chair of Pediatrics at CHOP and a professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.

Chief of the Division of Allergy and Immunology at Children’s Hospital of Philadelphia, Kathleen Sullivan, MD, PhD, received recognition — both inside and outside of CHOP — for her research and contributions in the field of primary immunodeficiency diseases, and for her mentorship of up-and-coming investigators.

During the 2017 Immune Deficiency Foundation National Conference in Anaheim, Calif., Dr. Sullivan was given the Boyle Scientific Achievement Award. The recognition was for her accomplishments in investigating common variable immunodeficiency, chromosome 22q11.2 deletion syndrome, and her work in defining the role of epigenetics in inflammation.

“The Boyle Award is the highest honor in the field of primary immune deficiency, and I was thrilled to be recognized,” Dr. Sullivan said.

Closer to home, Dr. Sullivan, who also holds the Frank R. Wallace Endowed Chair in Infectious Diseases, and is a professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania, was the recipient of the 2018 CHOP Faculty Mentor Award. The Research Institute’s Office of Faculty Development presents the award as an honor to faculty investigators whose mentoring has helped to produce the next generation of investigators dedicated to child health discoveries and clinical practice breakthroughs.

In a nomination letter from current and former divisional faculty of Allergy and Immunology, Dr. Sullivan’s colleagues wrote, “Although always stage-specific, her approach to mentorship is consistently thoughtful and practical: Set goals, prioritize tasks, and collaborate across disciplines whenever possible. It is a winning strategy.”