Apr 03 2015

PolicyLab Paper Finds Millions of Children Still Impacted by Recession


“Economists say the recession is over, but five years later, it’s still impacting millions of children,” said First Focus President Bruce Lesley.

A new report from The Children’s Hospital of Philadelphia’s PolicyLab and the advocacy organization First Focus found poverty, food insecurity, and housing instability are more prevalent than they were before the Great Recession. Moreover, according to the report, approximately 1.9 million more children live in poverty now than before the recession.

“Economists say the recession is over, but five years later, it’s still impacting millions of children,” said First Focus President Bruce Lesley. “Where national leaders made smart policy choices, kids fared better — where they didn’t, kids are still struggling.”

PolicyLab’s Rachel Meadows, MPA, was the lead author of the report, “The Effect of the Great Recession on Child Well-Being.” First Focus commissioned this new paper from PolicyLab, which is a follow-up to a 2010 study of the same issues.

Defined as the period from December 2007 through June 2009, the Great Recession saw high unemployment, frequent foreclosures, and the downfall of several larger investment banks, such as Lehman Brothers.

Now, more than five years after the end of the recession, approximately one third of American children still live in food insecure households, according to the PolicyLab report. More than 22 million children remain dependent on programs like the Supplemental Nutrition Assistance Program (SNAP), which offers help to low-income families. And roughly 30 percent of children live in “cost burdened” households, in which a disproportionate percent of the family’s income is spent on housing.

The report did find some silver linings. Anti-poverty safety nets such as SNAP, tax credits, unemployment insurance, and the Temporary Assistance for Needy Families program have helped lift millions of children out of poverty. Moreover, thanks to the Children’s Health Insurance Program (CHIP), which provides low-cost insurance to needy families, the number of children with health insurance increased despite the recession.

As of 2013, there were roughly eight million children enrolled in CHIP nationwide. However, CHIP’s funding has not been extended beyond 2015. And the safety net programs that proved so vital during the Great Recession face an uncertain future.

“Our research shows that investing in social safety net programs when times are good can have payoffs for ‘rainy days,’” said PolicyLab co-Director David Rubin, MD, MSCE. “We also know that millions of children are still struggling, and so we risk stalling or even reversing recovery by making budget and program cuts too soon.”

A recent decline in federal spending on children, combined with a reduction in American Recovery and Reinvestment Act (or stimulus) spending, combined with budget cuts across federal, state, and local governments means that many safety net programs may not be strong enough to weather another crisis.

In a blog post that accompanied the report’s release, Dr. Rubin and Kathleen Noonan, JD, the co-director of PolicyLab, note the report points to a need to confront income inequality. “A true recovery would reverse the trends of growing poverty and widening income gaps,” they write.

To read more, see the full report, “The Effect of the Great Recession on Child Well-Being.”

Permanent link to this article: http://blog.research.chop.edu/policylab-paper-finds-millions-children-still-impacted-recession/

Apr 02 2015

Researchers Suggest NTRK3 Gene Mutation Contributes to Heart Defects

heart defectDuring pregnancy, a series of intricate processes takes place for the heart to form correctly. Cells develop, proliferate, migrate, and die frequently, as the heart tissue transforms from a primary heart tube to four cardiac chambers. Scientists suspect that if there is a genetic mishap, those steps may not occur at the right time or place, resulting in congenital heart defects (CHDs).

One in 125 babies in the U.S. is not born with a perfect heart. CHDs are the most common major birth defects, and they range from simple to complex. For example, a heart defect called ventricular septal defect (VSD) involves an opening in the dividing wall between the two lower chambers of the heart. The hole allows an extra volume of blood to be pumped into the lungs, creating increased pressure, stress, and congestion.

Researchers at The Children’s Hospital of Philadelphia are searching for genes that could be linked to the presence of heart defects, and they recently reported on mutations in the gene NTRK3 that may be involved in the development of VSDs. NTRK3 regulates cell survival and encodes a protein called neurotrophic tyrosine kinase receptor C (TrkC).

“Finding the potential variations that are involved in heart defects in children is like finding a needle in a haystack, but you’re looking at a field of hundreds of haystacks,” said Petra Werner, DVM, PhD, a senior research associate in the laboratory of Elizabeth Goldmuntz, MD, professor of pediatrics in the Division of Cardiology at CHOP. “We picked NTRK3 as a candidate gene because deletion of this gene in mice will result in heart defects, and we had identified a patient with a VSD that had a large deletion encompassing NTRK3.”

In an article published in the December issue of Human Mutation, Dr. Werner and colleagues described how they screened 467 patients with related heart defects for NTRK3 mutations. They identified four of those patients with VSDs who had a missense mutation, which means an amino acid substitution occurred in the TrkC protein made by the gene that may modify how it works.

Next, the study team conducted experiments to see if the mutated TrkC lost any function. As a receptor, TrkC sits on cells’ membranes and waits for a signal from its ligand, a protein called neurotrophin-3 (NT-3). The results showed that one of the mutations significantly reduced TrkC’s ability to respond to the ligand, and subsequently TrkC failed to activate essential downstream signaling pathways.

In addition, the investigators found that cells expressing mutant TrkC showed altered cell growth. Usually, when NT-3 is present and binds to TrkC, it is a survival signal for the cell to differentiate and migrate. When the NT-3 ligand is absent, the cell begins to die, a process known as apoptosis. The experiments showed that cells with some of the mutant TrkC kept growing, even when they lacked NT-3.

“These results suggest that these mutations might permit increased cell growth under developmental conditions where morphologic changes require cell death,” the investigators wrote in the Human Mutation article.

Dr. Werner and her colleagues hypothesize that if TrkC’s function is impaired and allows the wrong heart cells to differentiate and migrate, then flaws could occur during the rapid remodeling of embryonic heart development.

“They may end up in the wrong location in the heart and be missed in other locations, resulting in malformations or holes,” Dr. Werner said. “But much more research must be done before we fully understand all of TrkC’s functions.”

Permanent link to this article: http://blog.research.chop.edu/researchers-suggest-ntrk3-gene-mutation-contributes-to-heart-defects/

Mar 31 2015

Driven by Patients: CHOP’s Cutting-Edge Fetal Surgery to be Featured on PBS

fetal surgery

Tune into WHYY at 8 p.m. on March 31st, April, 7st & 14th!

Mark your calendars! Starting tonight, the work of The Children’s Hospital of Philadelphia’s N. Scott Adzick, MD, an internationally recognized fetal surgery pioneer, will be featured in the new documentary series Twice Born. Airing March 31 to April 14 on PBS, Twice Born will highlight stories from Children’s Hospital’s groundbreaking Center for Fetal Diagnosis and Treatment.

Children’s Hospital’s surgeon-in-chief, Dr. Adzick is the founder of the Center for Fetal Diagnosis and Treatment, one of the world’s leading fetal care centers. The Center offers everything from testing to fetal surgery to postnatal care, and since its inception in 1995, Center surgeons have performed more than 1,200 fetal surgeries. Its experts treat a wide range of conditions, including congenital diaphragmatic hernia, conjoined twins, spina bifida, and twin-twin transfusion syndrome.

“This can’t be done many places in the world, this not amateur hour, this is very serious stuff,” said Dr. Adzick in a preview of the series.

Twice Born will tell the stories of parents of babies with birth defects who came to CHOP for care. Including rare operating room footage and interviews with CHOP clinicians, the series “serves up both arresting human drama and astonishing medical science,” according to the show’s website.

“We’re driven by patient needs,” said Dr. Adzick. “We’re driven by trying to find solutions to those unsolved problems. It’s a miracle and a privilege to take care of patients, of babies. Babies are the future! What could be more compelling than a baby?”

In other news, Dr. Adzick was recently awarded the Philadelphia Academy of Surgery’s 2015 Samuel D. Gross Prize. Given “every five years for the best original research in surgery by an American citizen,” the prize is named after prominent nineteenth century surgeon Samuel D. Gross, MD. A surgical pioneer and founder of the Academy, Dr. Gross published numerous books on surgery and anatomy, but is perhaps best known for being the subject of the famous Thomas Eakins painting, The Gross Clinic.

The award recognizes some of Dr. Adzick’s many accomplishments. In particular, in 2011 Dr. Adzick led a landmark study in the New England Journal of Medicine that showed fetal surgery can significantly improve the outcomes for children diagnosed in utero with spina bifida. More recently, he has contributed to investigation of ex utero intrapartum treatment, congenital diaphragmatic hernia, and a study of extracorporeal membrane oxygenation, or ECMO.

For more information about Twice Born, see the trailer below. And be sure to check your local PBS station for Twice Born showtimes!

Permanent link to this article: http://blog.research.chop.edu/driven-by-patients-chops-cutting-edge-fetal-surgery-to-be-featured-on-pbs/

Mar 30 2015


CANCERThe history of our understanding and approach to cancer are at the center of CANCER: THE EMPEROR OF ALL MALADIES, a film directed by Barak Goodman and executive produced by Ken Burns. The documentary, which features the Cancer Center at CHOP, will air in three parts on March 30, 31 and April 1 at 9 p.m. ET on PBS. This film is based on Siddhartha Mukherjee’s 2010 Pulitzer Prize-winning book, The Emperor of All Maladies: A Biography of Cancer.

The end of the film’s final chapter, airing April 1 at 9 p.m., highlights the incredible story of Emily Whitehead, a patient of The Children’s Hospital of Philadelphia who was the first child enrolled in a clinical trial called CTL019. That trial, which takes an immunotherapeutic approach to the treatment of a form of acute lymphoblastic leukemia, has since been awarded Breakthrough Therapy designation by the U.S. Food and Drug Administration.

More information is available on The Children’s Hospital of Philadelphia website.

View the trailer for the series below.

Permanent link to this article: http://blog.research.chop.edu/watch-cancer-the-emperor-of-all-maladies/

Mar 27 2015

NIH Award Boosts CHOP’s CRISSP Summer Program

CRISSPAn innovative summer program at The Children’s Hospital of Philadelphia Research Institute that exposes undergraduates to CHOP laboratories has received key backing from the National Institutes of Health. CHOP Research was awarded more than $500,000 from the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) to support the CHOP Research Institute Summer Scholars Program (CRISSP).

Started as a pilot program in 2012, CRISSP pairs promising undergraduate scientists with leading CHOP researchers for a 10-week program, from June to August. In addition to offering undergraduates valuable laboratory and clinical research experience, the program also supports their professional development with networking lunches and other events, and CRISSP interns conduct informational interviews to learn more about graduate and medical school. They are given the opportunity to shadow clinicians, and are trained in laboratory safety, animal use, and human subjects protections.

“It has been inspirational to work with CRISSP faculty and students over the past several years,” said Ian Krantz, MD, the program’s principal investigator. “I am amazed at the maturity and drive of the students who we have had the pleasure to host in my lab and the quality of the overall program in providing a unique and valuable research experience to these undergraduates.”

Since its inception, the program has been a runaway success. CRISSP’s applicant pool has nearly doubled since 2012, with 2015 applications coming from more than 164 schools for 25 spots. Moreover, administrators have seen a rise in the overall strength of applicants, making the selection process that much more difficult.

In 2014 CRISSP students hailed from twenty schools around the country, including the University of Virginia, Villanova University, and the University of Pennsylvania. Three spots in the program are reserved for Community College of Philadelphia (CCP) students, and Dr. Krantz, Eric Marsh, MD, PhD, and Adam Resnick, PhD, have all committed to work with CCP students in 2015. Other 2015 faculty include oncologist Lamia P. Barakat, PhD, geneticist Struan Grant, PhD, and emergency medicine expert Mark R. Zonfrillo, MD, MSCE.

“It is has been a true pleasure for me to be part of CRISSP from the very beginning and to see firsthand the incredible degree of commitment and enthusiasm on the part of the CHOP administrators in charge of it and the unwavering support of the faculty,” said orthopedic expert Maurizio Pacifici, PhD, who in 2014 mentored CCP student Adebayo Bello.

CRISSPThe NICHD grant will allow CRISSP’s managers in CHOP Research’s Office of Responsible Research Training to increase their efforts to diversify the program, add faculty and student mentoring services, offer Responsible Conduct of Research training, and add a high school component to the program.

In particular, the funding will support a half-day, “Research in Action” event for Philadelphia-area high school students. The annual event will expose students — starting with those from Philadelphia’s Science Leadership Academy — to the wide array of research performed at CHOP Research every day.

Dr. Williams and her team will also use the NICHD funding to develop a mentor training program for CRISSP-associated faculty and staff. Dr. Durbin, who has mentored many junior faculty members and physician fellows over the years, and who has studied research mentoring, will lead the CRISSP Research Mentor Training Program.

Moreover, the NICHD grant will fund the development of a mentorship training program for CRISSP students, to help them learn how to be an ideal mentee as well as how to mentor others. And it will help develop a workforce “pipeline” that encourages talented high school students, particularly under-represented minority students, to pursue careers in biomedical science.

“The support of the NICHD through the R25 grant will allow CRISSP to formalize and extend its efforts in providing one of the most exceptional undergraduate summer research experiences in the country,” said Dr. Krantz.

To learn more about CRISSP, see the program’s website.

Permanent link to this article: http://blog.research.chop.edu/nih-award-boosts-chops-crissp-summer-program/

Mar 25 2015

Technology Helps Overcome Barriers to Vaccination

vaccinationElectronic health records (EHRs) hold promise as tools to trigger reminders to clinicians and families to support on-time vaccination. Making sure that a child is immunized at the right time helps to provide the best defense against dangerous childhood diseases.

Pediatricians capture vaccine data and share these records with immunization information systems, formerly known as registries, which have been implemented in all 50 states to track vaccine receipt by children across practices. With the widespread use of EHRs, practices can use an EHR interface to populate these immunization records, and then the data can be used to expedite efforts to improve health outcomes.

In a recent editorial, Alexander Fiks, MD, MSCE, a faculty member at PolicyLab and the Center for Pediatric Clinical Effectiveness at The Children’s Hospital of Philadelphia, commented about a study appearing in the same issue of JAMA Pediatrics that described an approach to using health information technology to increase vaccination coverage. Allison Kempe, MD, MPH, of the Children’s Hospital Colorado, led the study team that conducted the randomized pragmatic trial.

The researchers targeted families of more than 18,000 children, comparing traditional practice-based vaccine reminders through telephone or mail versus multiple telephone and mail reminders delivered centrally by health departments working in collaboration with practices and using the Colorado Immunization Information System (CIIS) as a data source.

They found that those who received the collaborative centralized reminder not only had higher rates of vaccination, but also lower costs per child vaccinated. Of importance, vaccination rates were higher when reminder messages were specifically endorsed by a child’s practice.

In certain settings where the interests of the practice and public healthcare systems coincide, collaboration with health departments or other outside groups may prove effective, especially if they are built on existing relationships between families and clinicians, according to the JAMA Pediatrics commentary.

“With nearly 80 percent of pediatric practices now capturing data in EHRs, a remarkable opportunity exists to use these data to improve health outcomes,” Dr. Fiks wrote.

Permanent link to this article: http://blog.research.chop.edu/technology-helps-overcome-barriers-to-vaccination/

Mar 24 2015

Babies’ BMI May Predict Childhood Obesity

childhood obesityA new study from The Children’s Hospital of Philadelphia shows body mass index (BMI) during infancy may help to predict if a child will be obese by age four. In work focused on the infant BMI-childhood obesity relationship in a cohort with a majority of African-American children, Children’s Hospital researchers say a better understanding of infant growth patterns may lead to more effective early efforts at obesity prevention.

“Given the public health importance of obesity-related medical problems, we investigated whether BMI in infants could be used as a tool to identify children at increased risk of future obesity, in order to develop better prevention strategies,” said the study’s leader, Shana E. McCormack, MD, a pediatric endocrinologist at CHOP. “We also analyzed ancestry-based differences in growth patterns, and found differences that were apparent at as early as nine months of age were ultimately related to childhood obesity risk.”

As a measure that includes both weight and height, BMI is an approximation of body fat content. BMI increases after birth, reaching its peak in infancy, usually between eight and nine months of age.

The current study, published in the Journal of Clinical Endicronology and Metabolism, analyzed the electronic health records of healthy Philadelphia-area infants, as part of a larger study conducted by the Center for Applied Genomics. Sixty-one percent of the children in the study were African-American, a population that has shown high rates of obesity and diabetes in adulthood. Investigators hope that more reliable, early identification of all infants at increased risk for obesity will offer a unique opportunity to develop and implement targeted interventions.

The research team identified significantly different growth trajectories between African-American infants and white infants. Peak infant BMI occurred around 12 days earlier in African-American children, and was about 3 percent higher in magnitude than others in the study, who were primarily of European ancestry. Overall, African-American infants appeared to have more than twice the risk of obesity at age four compared to infants of primarily European ancestry.

However, the study team performed statistical analyses to distinguish the effects of ancestry and infancy BMI, while also accounting for other factors such as birth weight and socioeconomic status. Their conclusion was that infancy BMI played a more important role than ancestry in determining the risk of childhood obesity. In addition, socioeconomic factors, inferred from geographic and insurance data, played a role in infancy BMI.

In children under age two, there is currently no consensus definition of obesity, said Dr. McCormack.

“In the absence of an accepted, valid definition of obesity in infancy, we struggle both as researchers and clinicians with how to best individualize recommendations for infants to prevent childhood obesity,” she added. “Our findings suggest that infant BMI pattern could be one additional tool. In addition, infant BMI may be an early metric to use in evaluating the impact of public policy interventions.”

To read more, see the full press release about this study.

Permanent link to this article: http://blog.research.chop.edu/babies-bmi-may-predict-childhood-obesity/

Mar 23 2015

View Amazing Images in Honor of Brain Awareness Week

Brain Awareness WeekNeuroscientists worldwide celebrated the 20th Anniversary of Brain Awareness Week, March 16-22. The outreach campaign aims to educate the public about the brain and the promise of brain research. Future therapies for brain-related diseases such as Alzheimer’s, Parkinson’s and epilepsy depend on a better understanding of the brain and neuroscientists’ innovation.

At The Children’s Hospital of Philadelphia, researchers in the Division of Neurology are looking at the cellular and molecular mechanisms underlying the development of epilepsy. They use sophisticated imaging technology to view and characterize alterations occurring in the epileptic brain at a circuit level. Epileptic seizures emanate from a burst of electrical signals in dysfunctional brain circuits, which are complex collections of interconnected neurons that process information.

Neuroscience researcher Hajime Takano, PhD, who works in Douglas Coulter, PhD’s, epilepsy research laboratory at CHOP, is investigating which specific neurons could be inciting the neural network. Here are some amazing images from Dr. Takano’s work. He modified the original scientific micrographs for the artistic effect:

Brain Awareness Week

Left Image: Interneuronal network in the dentate gyrus captured by two-photon excitation fluorescence microscopy. Image Courtesy of Hajime Takano, Christopher Dengler, Ethan Goldberg, Douglas Coulter (Translational Epilepsy Research Program)
Right Image: Hippocampal neurons with the genetically encoded calcium indicator captured by two-photon excitation fluorescence microscopy. Image Courtesy of Hajime Takano, Fu-Chun Hsu, Douglas Coulter (Translational Epilepsy Research Program)

Brain Awareness Week

Left Image: Fluorescence micrograph of a hippocampal slice expressing red fluorescent protein in dentate gyrus granule cells. Image Courtesy of Hajime Takano and Douglas Coulter (Translational Epilepsy Research Program)
Right Image: Fluorescence micrograph of CA3 hippocampal neurons stained with calcium indicator dye and color coded activity map of individual neurons captured by high speed confocal microscopy. Image Courtesy of Hajime Takano and Douglas Coulter (Translational Epilepsy Research Program)


Permanent link to this article: http://blog.research.chop.edu/view-amazing-images-in-honor-of-brain-awareness-week/

Mar 20 2015

Researchers Honored for Using Video to Better Understand Alarm Fatigue

Christopher_Bonafide_0139ECRI Institute selected an innovative study conducted by patient safety researchers at The Children’s Hospital of Philadelphia who aimed to objectively measure alarm fatigue as a finalist for its 9th Annual Health Devices Achievement Award. The award recognizes an outstanding initiative undertaken by an ECRI Institute member healthcare institution that improves patient safety, reduces costs, or otherwise facilitates better strategic management of health technology.

When healthcare workers experience alarm fatigue, they become overwhelmed, distracted by, or desensitized to the numbers of alarms that monitors activate, which can lead to delayed response times or missed alarms. Addressing alarm fatigue is one of the key factors to improving the management of clinical alarm systems, according to the Joint Commission’s National Patient Safety Goal on alarm safety.

CHOP pediatrician and patient safety researcher Christopher Bonafide, MD, MSCE, submitted the entry, “Using Video to Evaluate Physiologic Monitor Alarm Characteristics and Nurse Responses to Alarms.” It described how the study team used a video-based approach to gather data on staff response times and false alarm rates.

After obtaining permission from nurses and patients’ parents or guardians, the team mounted cameras strategically in patient rooms to capture simultaneous views of the patient room, a close-up view of the patient, a full view of monitor screens and ventilator displays, and views of the caregivers responding to the alarms. Additional software generated a time-stamped list of alarms that occurred during the video sessions.

The team performed 40 video sessions and then analyzed the data generated by 4,962 alarms. They determined that caregiver response times increased as the number of false, or nonactionable, alarms increased. In the intensive care unit, 86.7 percent of alarms in heart and lung failure patients were characterized as false. In the general wards, 99 percent of alarms were false.

An article published in the ECRI’s online journal Health Devices described how Dr. Bonafide and a multidisciplinary team of biomedical engineers, physicians, nurses, researchers, and administrators designed and conducted the study.

“Studies such as these can help drive and inform interventions to reduce alarm burden and fatigue, thereby improving system and staff performance and ultimately improving the quality of care,” the article states.

Dr. Bonafide shared his reaction to the runner-up award: “Our team was thrilled to hear that the ECRI Institute selected our project for this tremendous honor. We are committed to using the findings of this study to inform new initiatives to eliminate alarm fatigue and improve the outcomes of hospitalized children.”

The National Heart, Lung, and Blood Institute recently awarded a five-year career development award to Dr. Bonafide, who is also an assistant professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania, to learn more about the science behind alarm fatigue and discover new ways to combat it.

See a PDF of the Health Devices article describing the CHOP initiative that was selected as a finalist for the 2014 Health Devices Achievement Award.

Permanent link to this article: http://blog.research.chop.edu/researchers-honored-using-video-better-understand-alarm-fatigue/

Mar 18 2015

Vaccinating Against Diseases, Without a Vaccine

vaccineOn the heels of his being consulted about a novel study led by the Scripps Research Institute’s Michael Farzan, PhD, the work of The Children’s Hospital of Philadelphia’s Philip R. Johnson, MD, was highlighted in a new The New York Times article about immunoprophylaxis by gene transfer (IGT), a technology spearheaded in part by Dr. Johnson.

Last month, Dr. Farzan published a study in Nature that describes his team’s creation of a new molecule that prevents monkeys from being infected with simian/human immunodeficiency virus. That research builds on Dr. Johnson’s work with simian immunodeficiency virus (SIV) to develop proteins that act like antibodies called immunoadhesins. These proteins, which Dr. Johnson and his team delivered via recombinant adeno-associated virus (rAAV) vectors, successfully blocked SIV infection.

As The New York Times article notes, this approach “is altogether different from traditional vaccination.” Instead, it is more akin to gene therapy, for in the case of HIV the AAV-delivered antibodies “can latch onto many different strains of the virus and keep them from infecting new cells.”

“The new study reinforces the potential promise of the AAV-mediated immunoprophylaxis approach as an alternative to vaccination to prevent HIV infection,” said Wayne C. Koff, chief scientific officer of the International AIDS Vaccine Initiative (IAVI), after Dr. Farzan’s study was published. “We look forward to further studies that will show how well any of these approaches can work in humans and will bring us all closer to a world without HIV/AIDS.”

Following that, Children’s Hospital last year partnered with IAVI to launch a clinical trial to study the safety of the rAAV vector carrying the broadly neutralizing antibody PG9, which has been shown to protect against HIV. The study team hopes to collect data later this year.

“For Dr. Johnson,” the Times article notes, “the growing interest in IGT is gratifying. ‘It’s catching on, but it’s certainly not mainstream,’ he said. That seems likely to change, and soon.”

“The sky’s the limit,” said Dr. Farzan.

Read the whole thing at The New York Times.

Permanent link to this article: http://blog.research.chop.edu/vaccinating-against-diseases-without-a-vaccine/

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