Peter Adamson, MD, a pediatric oncologist at Children’s Hospital of Philadelphia and chair of the Children’s Oncology Group, joined the Blue Ribbon Panel for the National Cancer Institute (NCI), as a part of former U.S. Vice President Joe Biden’s national Cancer Moonshot initiative.

The panel served as a working group of the presidentially appointed National Cancer Advisory Board, which Dr. Adamson was named to in June 2015 by President Obama. Panel members considered how to advance new approaches in cancer research, and in September, they issued issue 13 recommendations for the Cancer Moonshot, three of which focused on childhood cancer.

“For the Panel’s pediatric working group, we assembled leaders from around the country in a very short timeframe to identify innovative areas of science today that could accelerate progress in pediatric cancer,” Dr. Adamson said. “And we’re fortunate that many of the leaders in childhood cancer research are right here at CHOP, including Stephen Hunger, MD, chief of the division of Oncology and director of the Center for Childhood Cancer Research at CHOP, and John Maris, MD, a pediatric oncologist at CHOP and co-head of the Pediatric Cancer Dream Team, who are pursuing new treatments for the most challenging childhood cancers. As part of the working group, they helped us focus the discussion and think about transformative research ideas.”

Read more about the pediatric research in the Moonshot era.

Bryan Wolf, MD, PhD, Chief Scientific Officer of CHOP’s Research Institute, will contribute valuable insight from 25 years of working with pediatric health researchers and scientists in his new role as a member of Life Sciences Pennsylvania’s (LSPA) Board of Directors.

LSPA, formerly Pennsylvania Bio, is a statewide trade association representing 700 members that aims to ensure the economic vitality of the life sciences industry by promoting collaboration and public policies that support innovation in the pursuit of improving human health. As a board director, Dr. Wolf will foster supportive relationships within this vibrant community of scientists, biotechnology companies, drug manufacturers, and entrepreneurs.

“A key role of the Research Institute is to take the discoveries and breakthroughs from the research labs into everyday clinical practice, in order to have the greatest impact on the care of our children,” Dr. Wolf said.

As one of Pennsylvania’s top industries, the life sciences sector employs about 78,000 people directly and is known for its highly skilled workforce. LSPA has honored several leading visionaries in this scientific arena for their groundbreaking research and medical engineering performed at the Research Institute and at the Perelman School of Medicine at the University of Pennsylvania.

Read more in Cornerstone.  

Marni Falk, MD, an attending physician in the division of Human Genetics who directs the Mitochondrial Disease Clinical Center at Children’s Hospital of Philadelphia, participated in an expert committee convened by the Health and Medicine Division (formerly Institute of Medicine) of the National Academies of Science, Engineering, and Medicine to evaluate the ethical, social, and policy considerations of emerging techniques that could provide a new reproductive option for mothers who face the risk of passing mitochondrial disease on to their child.  

Mitochondria have their own genetic material called mitochondrial DNA (mtDNA) that babies inherit only from their mother and can cause various medical disorders if mutations occur. Through the modification of an egg or fertilized egg, mitochondrial replacement techniques (MRTs), could help prevent mtDNA disease by replacing the intended mother’s mitochondria that contains a pathogenic mtDNA mutation with mitochondria containing healthy mtDNA from a female donor.

Dr. Falk and committee members issued a report in February 2016 that recommended clinical research into MRTs proceed with careful oversight, and they contributed to a New England Journal of Medicine perspective article on MRTs’ implications for clinicians.

“It was a great experience because of the unique opportunity to get to interact with such thoughtful people having deep expertise in policy and ethics and have them learn and carefully consider the broader implications of quite intricate aspects of science and medicine,” said Dr. Falk, who is also an associate professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.

Read more in Bench to Bedside.

Alexander Fiks, MD, MSCE, FAAP, a primary care pediatrician at Children’s Hospital of Philadelphia, became director of the Pediatric Research in Office Settings (PROS) network in January 2016. The American Academy of Pediatrics developed PROS to help foster research through partnership between practicing pediatricians and researchers. Once developed, questions could be researched throughout this national network of primary care practices.

In his new leadership role, Dr. Fiks aims to broaden the range of PROS studies to include large clinical trials, secondary analyses of electronic health record data from practices, and longitudinal surveys of practitioners.

“The key to achieving these goals will be making sure that researchers and practitioners around the country know that PROS is interested in learning about their research and ideas,” Dr. Fiks said. “In this way, PROS can best serve as a resource to improve the effectiveness of primary care.”

Dr. Fiks is associate medical director for CHOP’s Pediatric Research Consortium; associate director of the Center for Pediatric Clinical Effectiveness; a founding member of the Department of Biomedical and Health Informatics; and a PolicyLab faculty member. He also mentors multiple faculty and academic fellows, and he is an associate professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.

Read more in Cornerstone.

Christopher Forrest, MD, PhD, a professor of Pediatrics at Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, wants to generate faster, cheaper, and better clinical research. As newly appointed chair of the research committee for the national patient-centered clinical research network called PCORnet, he is helping research studies take shape to include a participant population of up to 80 million Americans who are part of 33 large research networks.

“PCORnet’s sweet spot is taking existing treatments and understanding how well they work in the real-world population,” Dr. Forrest said. “A lot of times decisions are made based on gut feelings or based on physicians’ practices that were handed down to them from their teachers, and there may not be good research that underpins them. Clinical trials that show a medication actually does work, don’t take account of all the diversity we have in patient populations. PCORnet, with 80 million people, is able to address that diversity.”

Dr. Forrest was already principal investigator of one of PCORnet’s component networks, PEDSnet, which brings together data from eight children’s hospitals including CHOP to accelerate discovery in pediatric clinical research.

Read more about Dr. Forrest’s work with PCORnet and PEDSnet in the Collaboration section.

Children of military families face special circumstances as a result of their parents’ deployments and frequent relocations. David Rubin, MD, MSCE, director of PolicyLab at Children’s Hospital of Philadelphia, is using his expertise in a new role to ensure that they have timely and efficient access to pediatric healthcare services.

The United States Department of Defense (DoD) appointed Dr. Rubin to its Military Family Readiness Council, which makes recommendations to the Secretary of Defense about policies and programs for servicemen and servicewomen so that they are well-supported while focusing on their mission.

Dr. Rubin’s team at PolicyLab has been conducting research for the DoD’s Defense Health Program (DHP) since 2011, specifically examining the stress families experience when soldiers return home from deployment. Their findings published January 2016 in the American Journal of Public Health illustrate the need to support families throughout the deployment cycle.

“As a pediatrician at CHOP, I care for children from all different kinds of families, including those in the military,” Dr. Rubin said. “And as a researcher, I know that these children face unique challenges and are greatly affected by the deployment of their parent. I look forward to serving military families as a member of the Military Family Readiness Council.”

Read more in a CHOP press release.