President Obama appointed Peter C. Adamson, MD, an oncologist at The Children’s Hospital of Philadelphia and an internationally recognized leader in pediatric cancer drug development, to the National Cancer Advisory Board (NCAB).

The appointment follows the President’s announcement during a recent State of the Union address of a Precision Medicine Initiative that will harness research and technology toward developing individualized treatments for many diseases.

Dr. Adamson is the only pediatric oncologist to currently serve on the NCAB, and he works to ensure decision makers in the federal government hear the voices of the pediatric cancer community.

In his new role Dr. Adamson is advising the U.S. Secretary of Health and Human Services, the director of the National Cancer Institute (NCI), and the President on a wide range of issues relating to the national cancer program, including NCI operations.

Notably, the NCAB and the President’s Cancer Panel are the only advisory bodies at either the National Institutes of Health or the Department of Health and Human Services whose members are appointed by the President.

A renowned investigator and leader in cancer drug development for children, Dr. Adamson has served as chair of the Children’s Oncology Group (COG), the world’s largest organization devoted exclusively to childhood and adolescent cancer research, since 2011. Through COG, he works with leading pediatric cancer researchers at CHOP and across the country who are hard at work on the most promising new therapies. Dr. Adamson is also a professor of Pediatrics and Pharmacology at the Perelman School of Medicine at the University of Pennsylvania.

From 1999 to 2014, Dr. Adamson was chief of the Division of Clinical Pharmacology and Therapeutics at CHOP, and he also served as director of the Office of Clinical and Translational Research from 2005 to 2011. Prior to joining CHOP in 1999, he was a member of the Pediatric Oncology Branch of the NCI.

“We are extremely proud of Dr. Adamson on his appointment by President Obama to this distinguished advisory board,” said Joseph St. Geme, MD, physician-in-chief and chair of the Department of Pediatrics. “This recognition is a testament to his dedication and leadership within the medical community in furthering research to advance treatment for childhood cancer.”

In a fight to eradicate all childhood cancers, it helps to have a strong general leading the charge. The Children’s Hospital of Philadelphia welcomed a new leader in pediatric oncology in November 2014.

Stephen P. Hunger, MD, a nationally prominent specialist in children’s cancer, became the new director of the Center for Childhood Cancer Research (CCCR) and chief of the Division of Oncology. He joined CHOP from Children’s Hospital Colorado.

“I am tremendously excited by this opportunity to join one of the premier pediatric cancer programs in the world and to help CHOP develop more and better therapies toward our eventual goal of curing all pediatric cancers,” Dr. Hunger said.

He is a fitting leader for CHOP’s comprehensive program, staffed by internationally recognized experts in pediatric cancer who rely on the most current advances in research and treatment. In U.S. News & World Report’s most recent rankings of pediatric care specialties, CHOP’s cancer care ranked second in the nation.

Dr. Hunger has a particular research interest in acute lymphoblastic leukemia (ALL), the most common childhood cancer. His recent papers include a Nature Communications paper detailing the evolution of genetic changes in ALL from diagnosis through remission and relapse, a New England Journal of Medicine report on the largest study of ALL genetics that identified new potential therapies for a newly described high risk subset, the first invited review on childhood ALL published in the New England Journal of Medicine in 10 years, a Journal of Clinical Oncology pharmacokinetic study in ALL patients, and an Annals of the New York Academy of Sciences paper on the development of asparaginase as an ALL treatment.

A leading member of the Children’s Oncology Group (COG), the national cooperative organization of pediatric cancer centers, Dr. Hunger has sat on numerous COG study committees, and served as chair of the organization’s acute lymphoblastic leukemia committee from 2008 to 2015. He was recently appointed the next Scientific Committee Chair of the International Society of Pediatric Oncology, and also has served on NIH study sections and committees of the National Cancer Institute, the American Society of Hematology, the American Society of Clinical Oncology, and numerous other foundations and groups.

“We are delighted to welcome this internationally renowned pediatric oncologist to CHOP to lead our pediatric cancer program, advancing the treatment of children, adolescents, and young adults with cancer and overseeing pediatric oncology research,” said Joseph St. Geme, MD, CHOP’s physician-in-chief and chair of the Department of Pediatrics. “Dr. Hunger has an impressive background as a clinician, investigator, teacher, and leader in pediatric oncology and will undoubtedly have a major impact at CHOP.”

The key to the success of a clinical research investigation is the contribution of time, energy, and effort of patients willing to participate. Without them, the hard work of clinical research teams to find better treatments and cures could never get done.

The Children’s Hospital of Philadelphia Research Institute established a new centralized resource this year focused on helping busy investigators with the often-challenging effort in finding and recruiting study participants.

The Recruitment Enhancement Core (REC) led by Chris Gantz offers assistance with recruitment plans, creating marketing and promotional materials, and giving investigators access to a registry of potential recruits. The REC falls under CHOP Research Institute’s Clinical Research Support Office.

Much of the REC’s work draws on Gantz’s decade of clinical research experience and marketing background. For example, the REC team works with investigators to create recruitment letter content and appearance — even the envelopes — all with the goal of making sure letters are actually opened once they are sent out. Likewise, Gantz is setting up a centralized, accessible space in the Hospital to display recruitment flyers. Another REC goal is to create a version of CHOP Research Institute’s Clinical Research Finder tool for the Hospital’s website.

The REC team also is building a robust registry of people interested in being involved in research, in part by encouraging investigators to start the dialogue with their patients. Indeed, according to a study by the Center for Information & Study on Clinical Research Participation, 95 percent of study volunteers would consider participating in another clinical research study in the future.

“The problem is that we haven’t always been asking,” Gantz said.

The REC team works with the University of Pennsylvania to identify opportunities for collaboration to enhance recruitment across both institutions, as well as participating in community engagement activities to raise awareness of CHOP and clinical research participation opportunities.

With the REC’s help making the match between participants and studies, CHOP study teams can recruit participants for clinical research more efficiently and effectively than ever before — and investigators and participants can move together swiftly toward better treatments and better outcomes from childhood disease.

More than 30 years ago, The Children’s Hospital of Philadelphia pioneered life-saving early surgeries for children with complex heart defects. Today, researchers are investigating ways to intervene even sooner — before birth — to protect these infants’ brain development and prevent brain injury.

The Fetal Neuroprotection and Neuroplasticity Program at CHOP launched in June builds upon growing evidence of the interaction of heart disease and brain development in the fetus. This research, much of it developed at CHOP, shows that in utero brain development is abnormal in fetuses with congenital heart disease (CHD), leading to delayed maturation, poor growth, and white matter injury.

Neurodevelopmental disability is now recognized as the most common complication of critical CHD — those patients requiring cardiac surgery in infancy — and has the most negative impact on quality of life, academic performance, and opportunity for independence as an adult.

“The Fetal Neuroprotection and Neuroplasticity Program is another innovative initiative in a long series of identifying opportunities to ensure that children with CHD not only survive, but truly thrive, as they grow into adulthood,” said J. William Gaynor, MD, cardiac surgeon and director of the Fetal Neuroprotection and Neuroplasticity Program, and an assistant professor of Surgery at the Perelman Medical Center at the University of Pennsylvania. “This program allows us to enhance our continuum of care from conception through adulthood.”

The focus of the new program is to investigate the factors that cause abnormal brain development in the fetus with CHD and, for the first time, to conduct clinical trials of fetal interventions to determine whether novel prenatal treatments can reduce brain injury and improve neurodevelopmental outcomes in newborns with CHD who subsequently undergo cardiac surgery. One such study aims to evaluate whether the hormone progesterone, administered prenatally to the mother, has a neuroprotective effect on brain development.

The program is a joint project of the Hospital’s Cardiac Center, the Fetal Heart Program, the Center for Fetal Diagnosis and Treatment, and the Division of Neurology.

“We now have an opportunity to not only offer the best diagnostic care to the fetus with heart disease, but to also begin to explore ways in which we can optimize long-term outcomes from the neurocognitive perspective,” said N. Scott Adzick, MD, surgeon-in-chief at CHOP, and a professor of Pediatrics, Obstetrics and Gynecology at the Perelman School of Medicine.

In the U.S., approximately one in every 120 newborns is diagnosed with CHD, making it the most common birth defect. As recently as the 1960s, only 20 percent of newborns with critical CHD survived to adulthood. Thanks to better prenatal diagnosis, advances in surgery, and improved postoperative care, early survival is over 90 percent.

While the Fetal Neuroprotection and Neuroplasticity Program initially focuses on the fetus with CHD, it will expand in the future to include fetuses with other birth defects, such as congenital diaphragmatic hernia and pulmonary hypoplasia.

Children with advanced cancers often do not have a known curative pathway because their tumors may acquire different genetic alterations that resist current treatments. A new research opportunity supported by the National Cancer Institute (NCI) called Pediatric MATCH (Molecular Analysis for Therapy Choice), which will be conducted by the Children’s Oncology Group (COG), aims to use the power of precision medicine to potentially provide investigational therapies for these stubborn cases.

“Pediatric MATCH will try to match genomic changes in certain children’s cancers with drugs that are either approved for adult cancers or with drugs that are still under investigation and not yet approved,” said Peter Adamson, MD, chair of the Children’s Oncology Group and a pediatric oncologist at CHOP. “Based on what we currently know about the genomics of childhood cancer, we estimate that perhaps 15 percent of children with relapsed cancer may have a finding for which there is an investigational drug that could be studied to determine its effectiveness.”

When a child participating in Pediatric MATCH undergoes a tumor biopsy at relapse, some of the tissue will be sent to a centralized center for specific genomic testing. Once investigators define the tumor’s genomic profile, they will sort through a group of approximately 10 or more targeted drugs to see if there is a drug that specifically targets the pathways identified by the genomic profile. Those children with matches may then receive the investigational new drug as part of this phase 2 trial.

The Pediatric MATCH trial is a combined effort of the NCI the COG, and a range of pharmaceutical companies that already have committed to providing drugs to be tested in the adult NCI MATCH Trial that began in July 2015. Dr. Adamson anticipates that many of this counterpart trial’s pharmaceutical agreements will carry over to Pediatric MATCH.

“That is of great importance to this project because all childhood cancers are rare and ultra-rare diseases. Pharmaceutical companies generally do not have an economic incentive to study childhood cancers, and thus partnership with the NCI and pharmaceutical companies is essential for success of this project,” said Dr. Adamson, who also is a professor of Pediatrics and Pharmacology at the Perelman School of Medicine at the University of Pennsylvania.

The Pediatric MATCH study team will share tumor analysis results with the child’s cancer specialists to help them guide the family’s treatment choices. In addition, the tissue samples will be highly valuable to future researchers as they try to explain the basis of cancer treatment failure and relapse.

Pediatric MATCH is a high priority for the COG, the world’s largest organization devoted exclusively to childhood and adolescent cancer research. Five working groups are focused on launching the trial in 2016, Dr. Adamson said.

A new center at The Children’s Hospital of Philadelphia Research Institute seeks to support vulnerable patients by conducting research to better understand the root of disparities — be they racial, gender-based, or caused by geography.

Though clinicians are tasked with doing their best to extend the same level of care to all patients, the fact remains disparities exist in care and health outcomes, especially in pediatric patients.

Led by Scott Lorch, MD, MSCE, the Harriet and Ronald Lassin Endowed Chair in Pediatric Neonatology, the Center for Perinatal and Pediatric Health Disparities Research (CPHD) will work to “identify, describe, and understand disparities in care and care practices among perinatal and pediatric patients.”

“Research in adult patients has shown that there are extensive disparities in the care received by minority patients, particularly Hispanic and African-American patients,” Dr. Lorch said. “CPHD, through multidisciplinary academic and clinical research, aims to understand how these same disparities apply to the perinatal population, where the mother-fetal interaction is of primary importance, and the pediatric population, where family/mother-child interaction is of primary importance.”

Dr. Lorch is an ideal choice to lead the new Center, because his work — focused on health disparities, the economics and geography of healthcare, and perinatal epidemiology — dovetails nicely with the Center’s mission.

Dr. Lorch is also director of the Neonatal-Perinatal Medicine Fellowship Program in the Division of Neonatology and deputy director of the Center for Outcomes Research at CHOP, as well as an associate professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania.

Other faculty associated with the new Center include Nadia Dowshen, MD; Kristen Feemster, MD, MPH, MSHP; Chén Kenyon, MD, MSHP; and Saba Khan, MD. The Center’s associate director is Ashley E. Martin, MPH, while Molly Passarella, MS, will perform statistical programming for the CPHD.

And though it was only recently established, the CPHD has already announced its first round of pilot project funding for junior investigators associated with CHOP. Designed to support pediatric and perinatal health disparities projects, the CPHD Pilot Grant Program “aims to engage fellows and junior faculty in HD research and to assist established faculty in developing new lines of research in this area,” Martin said.

Recent recipients of the pilot grant program include Whitney V. Cabey, MD, Dr. Feemster, Megan S. Ryerson, PhD, and Douglas Wiebe, PhD, who are exploring transportation access as a barrier to on-time immunization; and Stephanie Doupnik, MD, Chris Feudtner, MD, PhD, MPH, and Alexander Scharko, MD, who are investigating the prevalence of mood and anxiety disorders among hospitalized school-age children and adolescents.

The CPHD has also partnered with a number of community organizations and other partners to advance its goals of identifying and addressing pediatric and perinatal disparities. They range from the governmental like the Philadelphia Department of Behavioral Health and Intellectual disAbility Services (DBHIDS), to those in higher education such as the University of Pennsylvania’s Netter Center for Community Partnerships, and nonprofit organizations like Public Citizens for Children and Youth.

All of the CPHD’s research seeks to better understand and confront disparities. Ultimately, Dr. Lorch said, with its work the Center hopes to inspire the next generation of pediatric medical researchers to conduct health disparities research, and to start a dialogue about pediatric and perinatal health disparities, with the ultimate goal of improving outcomes for patients.

Children living with degenerative inherited white matter diseases, known as leukodystrophies, now have access to state-of-the-art diagnostics and integrated multidisciplinary care at The Children’s Hospital of Philadelphia’s new Leukodystrophy Center of Excellence.

Leukodystrophies affect the white matter in the brain and spinal cord resulting in developmental regression and premature death. Although these debilitating diseases do not currently have cures, if detected in the newborn period, a stem cell transplant may be a therapeutic option for some patients. Early detection also allows for preventative care strategies to improve quality of life for children and their families.

CHOP’s Leukodystrophy Center of Excellence is a diagnostic, clinical care, and research initiative launched in response to legislation in Pennsylvania and New Jersey that has added leukodystrophies to the newborn screening panel. This is expected to result in an increase of patients requiring specialized care to address their complex needs.

“This type of comprehensive approach is a critical component in the treatment of children living with inherited white matter diseases,” said Amy Waldman, MD, a CHOP pediatric neurologist and medical director of the Leukodystrophy Center of Excellence.

CHOP will offer a clinic specifically for infants identified by expanded newborn screening programs. They will have access to state-of-the-art diagnostic imaging studies and top specialists across CHOP, including experts from neurology, metabolics, rehabilitative medicine, physical therapy, occupational therapy, and complex care pediatrics.

The Hospital announced the launch of the Leukodystrophy Center of Excellence May 15 at the Calliope Joy Foundation’s “An Evening with Jim and Jill Kelly” gala at the Rittenhouse Hotel in Philadelphia. Hall of Fame quarterback Jim Kelly and his wife, bestselling author Jill Kelly, established Hunter’s Hope in memory of their son who died from Krabbe disease, a type of leukodystrophy. For the past decade, the Kelly’s have been staunch advocates for newborn screening for Krabbe disease and have been instrumental in creating Leukodystrophy Care Centers across the nation.

Funds raised at the gala will support clinical programs, design treatment plans, create imaging and laboratory diagnostics, establish the newborn screening evaluation clinic, and form a pilot research program.

“This is a game changing moment for the care and treatment of leukodystrophies, and we are so pleased the doctors and researchers at CHOP believe they can give hope to the children and families that was unimaginable not long ago,” said Maria Kefalas, co-founder of Calliope Joy Foundation, a nonprofit organization that raises funds to help children living with leukodystrophy through improved care, research, and family support.