The Society of Radiologists in Ultrasound (SRU) honored Children’s Hospital of Philadelphia radiologist, Beverly G. Coleman, MD, for her outstanding service to the society and the field of radiology. Dr. Coleman received the 2018 Distinguished Service Award at the SRU annual meeting in San Diego. Radiologists Edward Oliver, MD, PhD, and Steven Horiim, MD; and Lori Howell, DNP, executive director of the Center for Fetal Diagnosis and Treatment, travelled from CHOP to attend the reception and dinner honoring Dr. Coleman.

“Ultrasound is my passion,” Dr. Colelman enthused. “Throughout my career, I have tried to use this very unique imaging modality to serve patients and in so doing, I have had the pleasure and opportunity to serve many others.”

SRU is the only medical society in North America representing radiologists in the field of ultrasound. The society promotes the advancement of the science, practice, and teaching of the ultrasound specialization in patient care.

Dr. Coleman is the director of Fetal Imaging at CHOP’s Center for Fetal Diagnosis and Treatment, which provides highly specialized care to mothers carrying fetuses with known birth defects. The Center is recognized as an international leader in fetal diagnosis and care, and it is one of the few locations in the world that performs fetal surgery for life-threatening conditions. Dr. Coleman holds CHOP’s Endowed Chair in Fetal Imaging. She is currently the ultrasound commission chair for the American College of Radiology and served as president of the SRU from 2003 to 2005.

Chief Scientific Strategy Officer Beverly Davidson, PhD, was elected vice president for the American Society of Gene and Cell Therapy (ASGCT) for a three year term — positioning her for the presidency in 2021.

“I’m excited to provide support to the ASGCT membership and the broader gene and cell therapy community,” Dr. Davidson said, “and I’m looking forward to the continued advancement and implementation of these life changing medicines.”

Dr. Davidson, a leading investigator in gene therapy, has been an active member in ASGCT, the nation’s largest association of individuals in gene and cell therapy research, since its founding in 1995. ASGCT seeks to advance research, education, and clinical application in gene and cell therapies to treat disease. Its membership includes more than 2,500 scientists, physicians, and patient advocates.

Dr. Davidson is also the Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT), where, in 2018, she oversaw the opening of Children’s Hospital of Philadelphia’s new Clinical Vector Core facilities that produce clinical-grade viral vectors to deliver cell and gene therapy for difficult-to-treat diseases. She holds the Arthur V. Meigs Chair in Pediatrics and is a professor in the Department of Pathology and Laboratory Medicine at the Perelman School of Medicine at the University of Pennsylvania.

Steven Douglas, MD, chief of the Immunology Section in the Division of Allergy-Immunology at Children’s Hospital of Philadelphia, was recognized for his significant contributions in the field of pediatric HIV/AIDS with the 25th Herman and Gertrude Silver Lecture Award.

In his lecture entitled, “HIV/AIDS Virology and Immunology: Toward Durable Success,” Dr. Douglas drew on his 50-year career in immunology to review major milestones in pediatric HIV/AIDS research and shared his optimism that paradigm shifts and new discoveries are ahead.

“We have made great strides over this period of 35 to 40 years,” said Dr. Douglas, who also is a professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. “We know a great deal about the pathogenesis. We have a strong pharmacopeia of antiviral drugs. We have exciting ideas with new ones emerging. It’s a remarkable story that I’m thrilled to have been a part of in my career and to have watched this evolve.”

The Silvers established a fund for the award in 1990, in memory of their children who were treated at CHOP. The award has recognized physicians and researchers from leading academic institutions including the National Institutes of Health, the Centers for Disease Control and Prevention, Oxford University, and past recipients of The Nobel Prize.

Throughout the decades, Dr. Douglas’ research has led to important advancements in the pediatric HIV/AIDS field. In the late 1970s, he established laboratory methods for investigating two types of immune cells —monocyte-macrophages and lymphocytes — which facilitated laboratory research around the world. He further discovered that an important neuropeptide plays a key role in the neurological manifestations of AIDS.

Dr. Douglas also lends his talents to the Philadelphia International Maternal Pediatric Adolescent AIDS Clinical Trials (IMPAACT) Unit, which includes a clinical research site at CHOP. The Douglas Lab is the central immunology laboratory for the IMPAACT Network.

Congratulations went out this year to Lindsey George, MD, hematologist at Children’s Hospital of Philadelphia and assistant professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania, a recipient of the 2019 Clinical Research Achievement Award from the Clinical Research Forum. Dr. George was recognized for her promising research of the first gene therapy clinical trial to report a phenotypic cure for hemophilia B patients.

Hemophilia B is a hereditary bleeding disorder caused by a lack of the blood clotting factor IX — without it, the blood cannot clot properly to control bleeding. Dr. George served as the lead investigator for the clinical trial, which involved a single intravenous administration of a bioengineered, gene therapy vector that then allows patients to produce their own clotting factor IX to safely and effectively stop bleeding in hemophilia B patients.

“The ultimate goal of gene therapy is to have a one-time, disease altering or curative treatment,” Dr. George said. “We’re thrilled that we’ve made significant progress toward that goal and are cautiously optimistic.”

The 2019 Conquer Cancer Foundation/Anna Braglia Endowed Young Investigator Award in Cancer Supportive Care was awarded to Allison Barz Leahy, MD, attending physician with the Cancer Center at Children’s Hospital of Philadelphia.

As the foundation of the American Society of Clinical Oncology, Conquer Cancer funds research and education across every facet of cancer. The award is a one-year $50,000 grant supported by Helsinn, a Swiss pharmaceutical group focused on building quality cancer products. The award is given to a physician in the final years of training, to assist with the transition from a fellowship program to a faculty appointment promoting quality research in clinical oncology.

Dr. Leahy’s current research focuses on the benefits and drawbacks of using patient-reported symptom monitoring for children with cancer in order to examine the impact of such monitoring on the quality of life, hospitalization rate, and illness severity in childhood cancer.

“I am greatly honored to receive the Anna Braglia Endowed Young Investigator Award in Cancer Supportive Care,” Dr. Leahy said. “This award will provide invaluable resources for the investigation of the use of patient-reported symptom monitoring in pediatric oncology care. Determining how best to incorporate the child’s voice into their medical care is essential — and we are hopeful that this work lay the necessary groundwork to enhance communication with the treating team, increase patient and family engagement in care, and ultimately lead to better clinical outcomes for children undergoing treatment for cancer.”

Michael A. Levine, MD, received the 2018 Frederic C. Bartter Award honoring his work, spanning nearly four decades, to advance the understanding of inherited bone and mineral disorders. The award is given each year to an American Society of Bone and Mineral Research (ASBMR) member for his/her outstanding clinical investigation in disorders of bone and mineral metabolism.

“It is particularly gratifying to receive this award acknowledging the impact of my research from the ASBMR, which I consider my ‘home’ society,” Dr. Levine said. “Given that I have focused on uncommon or orphan diseases over my career, I could not have pursued these studies without the support of the outstanding resources and talented colleagues of the CHOP-Penn scientific community.”

A pediatric endocrinologist, Dr. Levine is the medical director of the Center for Bone Health at Children’s Hospital of Philadelphia, and chief emeritus of CHOP’s Division of Endocrinology and Diabetes. He holds the Lester Baker Endowed Chair in Pediatric Diabetes at CHOP.

Throughout his distinguished career, Dr. Levine achieved scientific breakthroughs by identifying the molecular basis of several inherited disorders of mineral metabolism, including the role of the GNAS gene and associated mutations in patients with pseudohypoparathyroidism and McCune Albright syndrome, and the role of the GCM2 gene as the basis of isolated hypoparathyroidism. His work has advanced the understanding of the parathyroid gland function.

Dr. Levine also studied the molecular pathogenesis of unusual metabolic bone disorders to provide insights into the basis of more common bone diseases. This research demystified the causes of various forms of rickets along with the revelation that genetic variation in some of these genes contributes to vitamin D insufficiency in North America and Europe.

The Society for Pediatric Research awarded Barbara Schmidt, MD, a neonatologist recently retired from Children’s Hospital of Philadelphia’s Division of Neonatology and its former director of Clinical Research, with the Douglas K. Richardson Award for her contributions to children’s health as a perinatal researcher. 

Among her career accomplishments and contributions, Dr. Schmidt directed a study entitled “Trial of Indomethacin Prophylaxis in Preterms,” in which researchers followed 1,202 extremely low birth weight infants from five countries through the end of their second year of life. The study found that the high rate of mental and motor deficits in these children is not improved by prophylactic treatment with indomethacin, a nonsteroidal anti-inflammatory drug.

In other work, Dr. Schmidt, professor emeritus of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania, served as the principal investigator of the “Caffeine for Apnea of Prematurity” trial, which enrolled more than 2,000 very low birth weight infants in North America, Europe, Israel, and Australia. The study revealed caffeine therapy for apnea of prematurity improves the rate of survival without any neurodevelopmental disability up to two years after birth.

The Association of Women’s Health, Obstetrics, and Neonatal Nurses (AWHONN) honored Diane Spatz, PhD, RN-BC, FAAN, with an Excellence in Research Award which recognizes members who exemplify the highest standards of service in nursing. Dr. Spatz, nurse scientist and founder of the Lactation Program at Children’s Hospital of Philadelphia, is internationally recognized for her research and education initiatives on the use of human milk and breastfeeding that are making a difference in the lives of mothers and infants in vulnerable populations around the world.

“Receiving this award was so meaningful to me because it recognizes the contributions that my research has made to improve the lives of children and families globally,” Dr. Spatz said. “Human milk is a life-saving medical intervention, and all families deserve evidence-based lactation care and support so that they can reach their personal breastfeeding goals.”

Dr. Spatz is also the founder of the CHOP Mothers’ Milk Bank and serves as the Clinical Director. At CHOP, Dr. Spatz developed the Breastfeeding Resource Nurse (BRN) course, a training program that enables nurses to provide families with evidence-based lactation support and care. More than 1,400 CHOP nurses have taken the course.

Dr. Spatz’s 10-Step Model to Promote and Protect Human Milk and Breastfeeding in Vulnerable Infants helps clinicians and mothers make informed choices about why human milk is a medical intervention and provides comprehensive breastfeeding support to mothers who choose to breastfeed. The model has been implemented in neonatal intensive care units in the United States and countries including Thailand, India, China, Mexico, and Japan.

With having all nurses in the Newborn Infant Intensive Care Unit trained as BRNs and by using her 10-step model at CHOP, the number of infants discharged on human milk has significantly increased over the past 18 years, with human milk rates at discharge being well above 75 percent. Some of her research with surgical infants has demonstrated rates of human milk/breastfeeding at over 86 percent.

Life Sciences Pennsylvania honored Jonathan Spergel, MD, PhD, Chief of the Allergy Program at Children’s Hospital of Philadelphia, with a Patient Impact Award for his work with CHOP’s Food Allergy Frontier Program. The award honors significant contributions to the quality of healthcare or length of life of patients.

Dr. Spergel was recognized for leading research on eosinophilic esophagitis (EoE), a rare chronic allergic inflammatory disease of the esophagus, the muscular tube that carries food from the throat to the stomach. EoE causes certain food allergens to trigger adverse reactions in children, including vomiting, heartburn sensations, and failure to thrive. Eliminating a suspected allergen from a patient’s diet can prove to be difficult, because individuals with EoE are often sensitive to several foods, making it unclear which food triggers a reaction.

His work with families at the Food Allergy Center — the only pediatric program nationally recognized for its expertise in diagnosing and treating all types of food allergies — is answering questions about the nature of EoE. Recently, Dr. Spergel and his team discovered that children enrolled in a clinical trial had outgrown their EoE, indicating that EoE was not a lifelong condition as previously believed.

“I am very honored about the award and it is truly the work of multiple people,” Dr. Spergel said. “But, most importantly, it gives hope to the families for a better treatment, which is the true goal of medicine.”

Photo by Jordan Brian Photography

What — or should we say — who is sweeter than sugar? Answer: Lisa States, MD, pediatric radiologist at Children’s Hospital of Philadelphia with an expertise in nuclear medicine. Dr. States received the 2018 Congenital Hyperinsulinism International “Be My Sugar” Award for Medical Excellence, in recognition of her life-saving work on the FDOPA PET/CT study — an integral component to finding an effective treatment for congenital hyperinsulinism (HI).

HI is a rare genetic disorder in which the insulin cells of the pancreas secrete too much insulin. The excess insulin causes low blood sugar, also known as hypoglycemia, which can lead to seizures, brain damage, and even death if left untreated. The Congenital Hyperinsulinism Center at CHOP cares for approximately 80 percent of children in the United States who require surgery for HI.

Dr. States has played a key role in studying the effectiveness of F-DOPA, a radioactive tracer drug that is used in PET scans to capture images of the pancreas. By highlighting areas of the pancreas that overproduce insulin, researchers are able to use F-DOPA to diagnose HI in newborns and detect and localize the focal form of the disease.

“One of the most rewarding aspects of my research is my collaboration with Congenital Hyperinsulinism International,” Dr. States said. “This group unites clinicians and families from across the globe to support patients and families, educate clinicians, and share research ideas.”

Chief of the Division of Neurosurgery Phillip (Jay) Storm, MD, received an inaugural Healthcare Hero Award for his work at Children’s Hospital of Philadelphia in the research and treatment of pediatric brain tumors, the leading cause of cancer-related death in children. With these awards, TEVA Pharmaceuticals recognizes leaders in healthcare research and treatment whose accomplishments have benefitted their patients, their field, and the global community.

“It is a great honor for our team, families, supporters, and collaborators to be the first recipient of this prestigious award,” Dr. Storm said. “I know that our efforts will continue to accelerate discovery, foster innovation, and lead to cures for our patients.”

In 2009, Dr. Storm partnered with fellow surgeons, oncologists, pathologists, foundations, and patients and their families to launch the Children’s Brain Tumor Tissue Consortium (CBTTC) at CHOP, a multi-institutional research program dedicated to the study and treatment of pediatric brain tumors. CBTTC provides free and open-access data to find a cure for pediatric brain cancer, with 6,400 samples provided to researchers to date, including data from 1,446 patients. It is the world’s largest pediatric brain tumor repository of information.

Building on the CBTTC model, Dr. Storm led the 2017 launch of the Philadelphia Coalition for a Cure (PC4C), a first-of-its-kind collaborative program that bridges discoveries between adult and pediatric cancer care. PC4C assesses, promotes, and facilitates the diagnosis and treatment of both adult and pediatric patients with brain tumors and other cancers, with the goal of streamlining research and precision medicine efforts.

Mary Ellen Vajravelu, MD, MSHP, pediatric endocrinologist at Children’s Hospital of Philadelphia, earned the Endocrine Society’s Early Investigators Award for her fellowship research on congenital hyperinsulinism and type 1 diabetes.

The Early Investigators Awards were established to promote the development of early-career investigators and recognize their accomplishments in endocrine-related research. Recipients receive a monetary award to assist in their development and public recognition of their research accomplishments.

“I’m continuously inspired by the innovative research going on throughout CHOP,” Dr. Vajravelu said, “and I’m grateful to have the opportunity to continue to learn from and collaborate with researchers across the institution.”

Dr. Vajravelu’s work focuses on improving clinical effectiveness of therapies for children with endocrine-related diseases by combining her experience in epidemiology, biostatistics, and qualitative research with her advanced training in quality improvement and patient safety. Her current research centers on the use of innovative healthcare delivery strategies, such as mobile health applications, to facilitate behavior change in adolescents with prediabetes and type 2 diabetes.

Douglas Wallace, PhD, was honored with the 2019 Charles L. Hoppel, MD, Award for Outstanding Contributions in Mitochondrial Research from Case Western Reserve University School of Medicine.

A pioneer and internationally renowned scientist in human mitochondrial genetics, Dr. Wallace is the director of the Center for Mitochondrial and Epigenomic Medicine (CMEM) and the Michael and Charles Barnett Endowed chair in Pediatric Mitochondrial Medicine and Metabolic Diseases at Children’s Hospital of Philadelphia.

More than 35 years ago, Dr. Wallace and colleagues founded the field of human mitochondrial genetics. He’s dedicated his career to the understanding of, and potential treatments for, a variety of disorders and diseases by focusing on mitochondria, tiny structures within human cells that produce 90 percent of the body’s energy.

“A revolution is occurring in medical genetics,” Dr. Wallace said. “With the elucidation of the rules of mitochondrial genetics, not only has a new class of genetic diseases been identified, but a totally new perspective is emerging on the etiology of the common metabolic and degenerative diseases. Thanks to the support provided by CHOP for the Center for Mitochondrial and Epigenomic Medicine and the Mitochondrial Medicine Frontier Program, CHOP is taking the leadership in this exciting new branch of medicine.”

Dr. Wallace discovered that the mitochondria’s own DNA (mtDNA), which encodes the blueprint for the cell’s power generators, is inherited exclusively from the mother. Dr. Wallace’s findings on mtDNA revealed women left Africa about 65,000 years ago to colonize Eurasia, and from Siberia, they crossed the Bering land bridge to populate the Americas.

He also proved that genetic alterations in the mtDNA can result is a wide range of metabolic and degenerative diseases, and he continues to study the communication between the mitochondria and nuclear DNA. This communication is mediated by the epigenome, inherited modifications in gene expression caused by tags or proteins that bind to DNA.

Under his leadership, CMEM researchers are investigating mitochondrial and epigenomic dysfunction in a wide range of medical conditions such as autism, epilepsy, heart disease, diabetes and obesity, forms of blindness, Alzheimer disease, Parkinson disease, cancer, and aging.

Children’s Hospital of Philadelphia Research Institute recognized Babette Zemel, PhD, professor in the Division of Gastroenterology, Hepatology, and Nutrition, with the 2019 Faculty Mentor Award. On top of Dr. Zemel’s impactful contributions to the field of bone health and growth research, she has supported many mentees in their journey to make their own scientific contributions, achieve funding, and carve out career paths by securing academic appointments.

The award is a special honor given to faculty investigators whose mentorship has helped their colleagues become the next generation of brilliant researchers at CHOP. Dr. Zemel received the award at the annual Scientific Symposium, an event which celebrates the Research Institute’s remarkable scientific community: a diverse group of thought leaders, innovators, experts, and early career scientists committed to advancing children’s health.

“Mentoring is, by far, the most enriching and fulfilling thing that I do,” Dr. Zemel enthused. “I have the most extraordinary, talented, dedicated people to mentor here at CHOP, coming from diverse disciplines within pediatrics, as well as nursing, and anthropology. As I work with them to establish their research programs, I am also learning and expanding my knowledge and experience. It’s a journey that we take together. The Faculty Mentor Award is the ultimate recognition of all of those things, and I am deeply, deeply honored.” 

Special recognition went out to the 22q and You Center at Children’s Hospital of Philadelphia and the Perelman School of Medicine at University of Pennsylvania for its outstanding, longstanding, exemplary, and unwavering commitment and contributions to the chromosome 22q11.2 community. Donna McDonald-McGinn, MS, CGC, director of the Center, and colleagues were in British Columbia at the 22q11.2 Society’s international conference to accept the inaugural Special Service Award.

Individuals with 22q11.2 deletion are missing a small piece of chromosome 22. This genetic condition is the most common cause of DiGeorge syndrome. Until CHOP identified the chromosomal etiology, it had also gone by other names such as velocardiofacial syndrome, among others. It may cause a wide variety of health problems, ranging from heart defects and cleft palate, to feeding difficulties, immune problems, speech delay, a unique pattern of learning disabilities and behavioral differences such as ADHD and autism.

The condition is difficult to diagnose because it affects each child differently, can display varying symptoms, and manifests in features associated with many other conditions. But the internationally recognized 22q and You Center continues to lead in supporting early detection, basic science research, and improved care toward better outcomes for all patients.

“The 22q and You Center was launched in the early 1990’s at the behest of families desperate for condition specific, experienced, coordinated multidisciplinary care following the longstanding clinical and basic science research efforts of Drs. Elaine Zackai and Beverly Emanuel,” McDonald-McGinn said. “The success of the Center can only be credited to the dedication of healthcare providers, coordinators, laboratory technicians, researchers, and students both within the Division of Human Genetics and across the entire institution. Although the physical plaques were presented to the Center leads, the award was accepted collectively as each and every breakthrough is the result of this singular focus — to help our patients and families in whatever way we can by working as a team.”