Jul 30 2014

Unique Collaboration Leads to “Dream Teams” to Advance Pediatric Research

dream teamTwo dedicated “dream teams” of investigators will shape innovative solutions to address unmet pediatric medical needs, as part of a research partnership with The Children’s Hospital of Philadelphia, Drexel University, and The Hebrew University of Jerusalem.

The joint projects each will receive $250,000 over two years in institutional funding, as administrators seek external investors interested in advancing exciting pediatric translational research with commercial viability. The dream teams rose to the top of 20 proposals submitted as a result of a research symposium in January that gave researchers from all three institutions the opportunity to connect and share ideas.

“We are thrilled by the potential for discovery that this portfolio of projects holds, and much of this important work should appeal to future donors,” said The Children’s Hospital of Philadelphia CEO Steven M. Altschuler, MD.

Funding these initial grants internally demonstrates the institutions’ commitment to be a hub of collaboration and exemplifies the inventive, diverse, and entrepreneurial spirit that formed their research consortium initiative. Dr. Altschuler, Drexel President John A. Fry, and The Hebrew University of Jerusalem President Menahem Ben-Sasson signed the research agreement in November as part of a trade mission that Philadelphia Mayor Michael A. Nutter took to Israel.

“These inaugural awards are just the beginning of what the ongoing collaboration between The Children’s Hospital of Philadelphia, Hebrew University and Drexel can accomplish,” said Drexel President John A. Fry. “Together we can create new and unique opportunities that will address unmet needs in pediatrics through innovative commercial pediatric therapeutics and diagnostics.”

One dream team will be based at Drexel with Amy Throckmorton, PhD, as the principal investigator of the “Giving Kids a Chance” project that will investigate a new intravascular blood pump for pediatric patients with congenital heart disease (CHD).

The treatment of single ventricle (SV) anomalies is a formidable and costly challenge for clinical teams caring for patients with CHD, which is the most common major birth defect affecting nearly 1 percent of all newborns. Palliative repair of a SV is generally performed in a series of open-heart procedures over several years. The end result is a man-made physiology in which a SV drives blood flow through the entire circulatory system without the presence of a “right-sided” pulmonary ventricle to pump blood to the lungs. A heart transplant is a treatment option in difficult cases, if the child can survive the waiting period.

As interest is growing about the use of mechanical assistance as a bridge-to-transplant or treatment strategy, the dream team aims to develop a uniquely designed, new therapeutic device for patients with dysfunctional SV physiology. Their goal is to advance the state-of-the-art in blood pump technology, reversing the deleterious characteristics of current approaches and helping to prevent premature congestive heart failure.

dream team

“These inaugural awards are just the beginning of an ongoing collaboration between The Children’s Hospital of Philadelphia, Hebrew University and Drexel can accomplish,” said Drexel President John A. Fry.

The interdisciplinary team will combine its broad expertise in engineering, pediatric cardiology, congenital heart and cardiothoracic surgery, medical device development, and manufacturing for artificial organs. Members from Drexel will include Dr. Throckmorton, J. Yasha Kresh, PhD, and Randy Stevens, MD. They will be joined by four investigators from CHOP: David J. Goldberg, MD; Matthew Gillespie, MD; Kevin K. Whitehead, MD, PhD; and Joseph Rossano, MD. Amnon Hoffman of The Hebrew University of Jerusalem and Amiram Nir of Hadassah Medical Center will round out the team.

They will work with four industry partners: Rotor Bearing Solutions International of Charlottesville, Va.; Cardiac Assist Inc. of Pittsburgh, Pa.; Applied Rapid Technologies of Fredericksburg, Va.; and Laserage Technology Corp. of Waukegan, Ill.

A second dream team will be based at CHOP with Robert J. Levy, MD, as the principal investigator of a research project “Pediatric Transcatheter Valve Replacements: Preventing Device Failure due to Structural Degeneration.”

These investigations also will focus on CHD with a concentration on Tetralogy of Fallot (TOF). These “blue babies” have insufficient oxygen and need cardiac surgery early in life. Postoperatively patients with TOF are left with chronic malfunction of their pulmonary valve.

Ongoing research at CHOP has demonstrated that transcatheter pulmonary valve (TPV) therapy — the current best option — is susceptible to oxidative damage and structural failure. This interdisciplinary team seeks to gain a better understanding of the inflammatory and oxidative events responsible and to inhibit the early inflammatory response to TPV. They will attempt to modify the TPV material with an antioxidant as a way to prevent oxidative damage to the TPV leaflets.

Dr. Levy will collaborate with Matthew Gillespie, MD from CHOP and Joseph H. Gorman, MD and Robert C. Gorman, MD, from Penn, along with Kenneth Barbee, PhD, and Kara Spiller, PhD, from Drexel, and Gershon Golomb from The Hebrew University of Jerusalem.

Permanent link to this article: http://www.research.chop.edu/blog/unique-collaboration-leads-dream-teams-advance-pediatric-research/

Jul 28 2014

CIRP Engineering Expert Accepts New Leadership Role

concussions

Most recently, Dr. Arbogast has extended her work to include the study of concussions, not only from motor vehicle crashes, but also from sports

The Center for Injury Research and Prevention (CIRP) proudly announced the promotion of Kristy Arbogast, PhD, to co-scientific director. She was previously and continues to serve as the Center’s director of engineering.

Dr. Arbogast has been with CIRP since its establishment in 1996 to advance the safety and health of children, adolescents, and young adults through comprehensive research that encompasses before-the-injury prevention to after-the-injury healing. She joined Flaura K. Winston, MD, PhD, and Dennis R. Durbin, MD, MSCE, as a co-investigator on the seminal Partners for Child Passenger Safety (PCPS) project, a 10-year-long national study that led to significant policy and safety design advances for child motor vehicle occupants.

“This is a natural transition for CIRP,” said Dr. Winston, scientific director and founder of CIRP. “As Kristy’s career has matured and flourished, so has CIRP. She has helped to grow our Center’s international reputation as a thought leader in child injury prevention and pediatric biomechanics.”

Dr. Arbogast has dedicated her career to safety research, focusing on the development of new auto and restraint safety designs and biofidelic child anthropomorphic dummies. Her work was recently recognized by the receipt of an honorary doctorate from Chalmers University, Sweden.

As CIRP’s co-scientific director, Dr. Arbogast will join Dr. Winston and Ayana Bradshaw, MPH, CIRP’s administrative director, in leading a group of behavioral scientists, clinicians, epidemiologists, biostatisticians, engineers, public health practitioners, and communications professionals that focus on injury prevention, violence prevention, physical and emotional recovery following injury, digital health, and pediatric biomechanics.

“I have benefitted from the interdisciplinary structure of CIRP,” Dr. Arbogast said. “Working with colleagues from diverse academic backgrounds and research interests has expanded my own understanding of child injury prevention. We are all dedicated to translating our research into action to reduce preventable child injuries and deaths.”

Most recently, Dr. Arbogast has extended her work to include the study of concussions, not only from motor vehicle crashes, but also from sports. She served on the Institute of Medicine Committee on Sports Concussions in Youth and is also a member of the National Council on Youth Sports Safety. Since 2011, Dr. Arbogast has co-led a multi-disciplinary project at CHOP called Minds Matter: Improving Pediatric Concussion Management that streamlined and standardized concussion diagnosis, treatment, and follow-up care across the CHOP network.

Dr. Arbogast continues to serve as the co-director of the National Science Foundation-sponsored Center for Child Injury Prevention Studies (CChIPS) at CHOP, University of Pennsylvania and The Ohio State University. She is also research associate professor of pediatrics at the University of Pennsylvania.

Permanent link to this article: http://www.research.chop.edu/blog/cirp-engineering-expert-accepts-new-leadership-role/

Jul 23 2014

Platelet Granule Formation Gives Insight Into Rare Disease

plateletIf you take a normal platelet and examine it under an electron microscope, you will see a bunch of black dots. The specks may not seem like much at first, but cell biologists at The Children’s Hospital of Philadelphia Research Institute speculate that these dense granules hold the key to unlocking the mechanisms behind a rare disease called Hermansky-Pudlak syndrome (HPS) and other forms of bleeding disease.

HPS is estimated to affect one in 500,000 to one in 1,000,000 individuals worldwide. In certain places, like Puerto Rico, it is much more prevalent — about one in 1,800 individuals. People with the disease have a tendency to bruise and difficulties with blood clotting, which can be deadly under certain circumstances such a pregnancy, major surgery, or dental surgery.

“Those dense granules don’t get made in a set of patients, and the consequence of that is the patients bleed too much, and they’re not able to make blood clots efficiently,” said Michael S. Marks, PhD, who received a grant in May from the National Heart, Lung, and Blood Institute to better understand how platelet dense granules form and why their creation is disrupted in HPS.

Dense granules are lysosome-related organelles (LROs) within platelets that act as a storage compartment for small molecules such as calcium, adenosine diphosphate, and serotonin. These molecules are released when platelets begin to accumulate at sites of blood vessel damage.

“We know that there are important things stored in those granules, but we have no idea how they get there,” Dr. Marks said. “This is the first time anybody’s taken an approach to try and understand how dense granules are put together and how those molecules get stored in them.”

HPS can be difficult to diagnose because it affects multiple organs, and the symptoms can be variable and nonspecific. Mutations in the genes associated with HPS prevent the formation of LROs or impair their performance in platelets, pigment cells, and lung cells. Subsequently, in addition to excessive bleeding, a main feature of the disease is oculocutaneous albinism, which causes abnormally light coloring of the skin, hair, and eyes. By the time people with HPS reach their 30s, a lung defect called pulmonary fibrosis appears that rapidly worsens, and the lung scarring often is fatal.

“Getting a good diagnosis early would be really important,” Dr. Marks said. “That would be the most immediate impact of this grant.”

The study aims to achieve a better understanding of two dense granule integral platelet membrane proteins that Dr. Marks’ team at CHOP and a collaborating group in Colorado recently identified — SLC35D3 and VMAT2 — which may act as vehicles to import dense granule contents. The investigators suspect that this delivery is impaired in HPS, and the dense granules do not form completely. Necessary machinery could be missing at a crucial time when a dense granule’s membrane coalesces from other membranes in megakaryocytes, which are platelets’ precursor cells that reside in bone marrow. It appears that this occurs during a late stage of differentiation of platelets from megakaryocytes.

In order to test this hypothesis, scientists in Dr. Marks’ lab will collaborate with another group of experts in megakaryocyte and platelet formation from the lab of Mortimer Poncz, MD, division chief of hematology at CHOP. They will take megakaryocytes from mice and then modify them by adding fluorescent proteins that hopefully will allow the investigators to visualize dense granule formation using live cell imaging. The next step will be to put the megakaryocytes back into the mice where they will produce platelets, and then take the platelets out to analyze them.

Dr. Marks also will compare the same mutations in human cells by using megakaryocytes created from stem cells of patients with HPS. Deborah L. French, PhD, a specialist in making induced pluripotent stem cells, will assist with this part of the project. Dr. French is director of the Human Stem-Cell Vector Core within the Center for Cellular and Molecular Therapeutics at CHOP.

The investigators will determine if SLC35D3 and VMAT2 live on dense granule membranes and whether or not they are expressed on platelet membranes in both the human and mouse HPS models. What they discover could guide new diagnostic approaches.

“If we’re right, then we could make antibodies to those proteins,” Dr. Marks said. “Then it should be a very simple test to look for exposure of that protein when you activate platelets.”

Such a diagnostic test would be helpful for identifying HPS as well as other forms of bleeding disease due to mutations in genes that encode proteins on these dense granules. As researchers learn more about the proteins’ structure and the jobs that they perform, this knowledge could be applied to designing drug therapies that either enhance the proteins’ activity, as would be the case in HPS, or decrease the proteins’ activity, which could potentially modulate diseases that cause too much blood clotting.

“Ultimately, maybe, we’ll be able to come up with some kind of treatment,” Dr. Marks said. “That will depend on whether we’re right with some of our guesses about the proteins that are on dense granules and whether we can characterize them further and identify steps that would be downstream from them.”

Dr. Marks also is looking forward to working on a future study with Susan Guttentag, MD, that recently received grant funding. That investigation will focus on the formation of another LRO, the lamellar body, in lung epithelial cells. Dr. Guttentag and colleagues have shown that HPS models interfere with this process which underlies the lung fibrosis in HPS.

Permanent link to this article: http://www.research.chop.edu/blog/platelet-granule-formation-gives-insight-rare-disease/

Jul 21 2014

Putting a Dollar Amount to Autism Pays Off

autismAutism spectrum disorders (ASDs) have an enormous price tag, and experts at The Children’s Hospital of Philadelphia are hoping that all those dollar signs add up to increased attention on new research and more coordinated ways to support patients with autism throughout adulthood.

One in 68 children in the United States has ASD, making it the fastest-growing disability. It is a complex neurological and developmental condition characterized by marked deficiencies in social interaction and communication and various behavioral issues. While individual presentations may vary, the signs of ASD usually appear during the first three years of life.

David S. Mandell, ScD, associate director of the Center for Autism Research at CHOP, and co-investigators in London updated estimates of the economic effects of ASDs on individuals and their families in the U.S. and U.K. They published their results online recently in JAMA Pediatrics. The lifetime cost of supporting a patient with an ASD and intellectual disability was $2.2 million in the U.S., and the lifetime cost for a patient with an ASD without an intellectual disability was $1.4 million in the U.S.

“They are staggering numbers in many ways,” said Dr. Mandell, who is also an associate professor of psychiatry and pediatrics at the University of Pennsylvania’s Perelman School of Medicine, where he directs the Center for Mental Health Policy and Services Research. “What is most intriguing about the study is what is driving those costs.”

For families, when their child with autism is young, special education costs were the largest cost component. Lost parental wages — especially for mothers who often must leave the workplace in order to care for their children — was another hefty economic hit. Productivity loss was estimated to be $18,720 annually for U.S. caregivers of children with ASD.

A key finding, Dr. Mandell pointed out, is that most of the expenses associated with autism accrue in adulthood. Society tends to think of autism as a pediatric concern, but it is a lifelong disorder. Residential care represents a substantial portion of those annual costs in the U.S.: $36,000 for adults with an ASD who have an intellectual disability, and about half that amount for adults with an ASD without an intellectual disability.

While some adults with autism have significant impairments and require expensive 24-hour residential care in a stable, supervised environment, many others who have participated in early interventions continue to develop their skills when given social coaching and job training so that they can work and live in less restrictive housing arrangements.

“I hope that advocates will use this information to support the importance of paying for intensive, evidence-based care for children with autism that has the goal of increasing their potential for full participation in their communities, which also may come with cost-savings,” Dr. Mandell said.

Part of the challenge to conducting this study, Dr. Mandell said, is that no gigantic spreadsheet listing all of these costs in one place exists. In addition to performing an extensive literature review conducted in 2013 concentrating on U.S. and U.K studies, Dr. Mandell and his co-authors relied on a number of sources to estimate cost-related data. For future studies, he encourages multiple systems to collaborate and perform longitudinal, population-based data collection so that researchers have easier access to metrics that can be used to assess the efficiency and effectiveness of autism care on a more regular basis.

Autism Speaks, the world’s leading autism science and advocacy organization, supported this study.

Permanent link to this article: http://www.research.chop.edu/blog/putting-dollar-amount-autism-pays/

Jul 17 2014

Study Shows Evaluators Can Screen for Eye Disease Remotely

retinopathy of prematurityA study recently published in JAMA Ophthalmology shows trained evaluators who studied retinal images transmitted to computer screens successfully identified newborn infants likely to require a specialized evaluation for retinopathy of prematurity (ROP), a leading cause of treatable blindness. Findings from a new study strengthen the case for using telemedicine to address unmet medical needs of preterm babies worldwide who cannot be initially evaluated by ophthalmologists.

“This study provides validation for a telemedicine approach to ROP screening and could help prevent thousands of kids from going blind,” said lead investigator Graham E. Quinn, MD, MSCE, a pediatric ophthalmologist at The Children’s Hospital of Philadelphia. Dr. Quinn is the corresponding author and principal investigator of the study of remote evaluation of ROP conducted by the e-ROP Cooperative Group, a collaboration among neonatal intensive care units in 13 North American centers.

ROP involves an abnormal growth of blood vessels in the retina that may lead to scarring, retinal detachment and, in severe cases, blindness. Some degree of retinopathy of prematurity occurs in more than half of all infants born at 30 weeks gestation or earlier, but only five to eight percent of cases become severe enough to warrant treatment. Because early detection and prompt treatment are essential to identifying high-risk eyes, the American Academy of Ophthalmology recommends routine screening for all infants born at or before 30 weeks gestation or weighing less than 3.3 pounds.

In recent years, the U.S. has seen a decline in the number of ophthalmologists who conduct ROP screening examinations. To address the public health issue of detecting potentially serious ROP, the e-ROP Cooperative Group tested the validity of a telemedicine approach by comparing evaluations by ophthalmologists with those done independently by trained non-physician image readers.

Study Examined Patients Across the U.S. and Canada

The study team analyzed results in 1,257 premature infants in neonatal intensive care units (NICUs) at centers in the U.S. and Canada from 2011 to 2013. On average, the babies were 13 weeks premature and all weighed less than 1251 grams (about 2.75 pounds) at birth.

The infants all received regularly scheduled diagnostic examinations by an ophthalmologist who determined whether their ROP had a severity that warranted referral for further evaluation (designated RW-ROP). In addition, NICU staff members, called certified retinal imagers, took retinal photographs of all the infants, and those images were transmitted to trained image readers at the University of Pennsylvania. The image readers, all of them non-physicians, followed a standard protocol to assess whether features of RW-ROP were present in retinal images.

The image readers were unaware of which infants had been designated by the ophthalmologists as needing referral. The two groups had broadly similar results: the image readers identified 90 percent of the infants that ophthalmologists rated as having RW-ROP. When the readers did not find RW-ROP on grading, 87 percent of the time the ophthalmologist had not noted RW-ROP on the examination either.

Among the 244 babies that the ophthalmologists identified as having findings consistent with RW-ROP, 162 subsequently received treatment. Of these 162 infants, the non-physician image readers identified RW-ROP in 159 of them, meaning that 98 times out of a hundred, the eye was identified as a high-risk eye.

The investigators pointed out several potential advantages of telemedicine screening for ROP. Non-physician imagers could perform retinal imaging more frequently than ophthalmologists, and NICU staff can implement an imaging schedule individualized to specific babies. Grading of retinal photographs could allow a more standardized approach to ROP screening, while reducing the numbers of babies needing to be examined by ophthalmologists could thus lower the costs of routine retinopathy of prematurity screening. Finally, remote screening could decrease the number of unnecessary patient transfers to larger nurseries with more on-site ophthalmologists.

To see a video about e-ROP, visit the National Eye Institute YouTube channel at

Permanent link to this article: http://www.research.chop.edu/blog/study-shows-evaluators-can-screen-eye-disease-remotely/

Jul 15 2014

CHOP Researchers Discover Postsurgical Pain Gene Variants

painIn the first genome-wide analysis of postsurgical pain in children, pediatric researchers identified variations in genes that affect a child’s need for pain-control drugs. The findings suggest that at some point physicians may calibrate pain-medication dosages according to a child’s individual genetic makeup.

“Although this research is only a first step for our team, it provides tremendous new insight into the biological mechanisms and brings us a little closer to personalizing medicine for pain control,” said Scott D. Cook-Sather, MD, a pediatric anesthesiologist at The Children’s Hospital of Philadelphia. Dr. Cook-Sather and colleagues — including Hakon Hakonarson, MD, PhD, the director of the Center for Applied Genomics — published the study recently in the journal Pain.

The study team performed a genome-wide association study (GWAS) of more than 600 children between ages 4 and 18 who had tonsils and adenoids removed in day long surgery procedures. The retrospective study analyzed whether gene variants were associated with the need for higher or lower than average dosages of morphine for pain control. The researchers also analyzed genetic links to postoperative pain scores. The GWAS identified one gene location linked to increased morphine requirement.

“While scientists already know that morphine works by binding to specific opioid receptors in the nervous system,” added Dr. Cook-Sather, “we don’t know exactly why there is, in this setting, a tenfold variation in how much morphine patients require for pain relief.” The study team found that two single-base gene variants at the TAOK3 locus were associated with approximately 8 percent of that tenfold variance in morphine requirement, comparable to that portion of the variance associated with age, body mass and overall health status combined.

Dr. Cook-Sather explained that multiple genes are assumed to contribute to these analgesic effects, and that further investigations, with larger numbers of patients, are needed to understand and prioritize the full array of genes that modify morphine response.

Within their initial sample of 617 children, the researchers found that the association between the variants in TAOK3 and the morphine dose needed for pain relief held up for children of European ancestry but not for African-American children. In both groups, however, the gene variants correlated with increased postoperative pain. “Future investigations,” said Dr. Cook-Sather, “may help us predict which patients will need more pain medicine than the standard dose. We could customize an appropriate dose while the child is still under anesthesia in order to minimize the pain when the child regains consciousness.”

“We have identified a novel biological pain target, and even though the variants we identified in this study explain only about 8 percent of the difference in pain sensation between individuals, they give us a strong lead in developing new therapies,” said Hakonarson. “This proof-of-concept study may advance the process of individualizing pain therapy in children.”

Permanent link to this article: http://www.research.chop.edu/blog/chop-researchers-discover-postsurgical-pain-gene-variants/

Jul 10 2014

Electrocardiogram Use Varies for Infants With ALTEs

ElectrocardiogramWhen frantic parents arrive at the emergency room and report that their infant experienced a frightening combination of symptoms including a prolonged lapse in breathing, change in color or muscle tone, or coughing or gagging, clinicians likely will describe the episode as an apparent life-threatening event (ALTE). Yet, it can be a frustrating conclusion because often these symptoms have resolved by the time the patient arrives at the ER, and the clinician is left to discern the exact cause.

The most common underlying diagnoses related to ALTEs are gastroesophageal reflux and upper respiratory illness. Clinicians also consider cardiac causes, but no standardized method is used to evaluate patients with ALTEs, especially with regard to electrocardiograms (ECGs) as a diagnostic tool. An ECG measures a heart’s electrical activity and generates a graphic representation, or tracing, that can indicate heart-related conditions.

A study team of physicians from The Children’s Hospital of Philadelphia’s Department of Pediatrics and Cardiac Center wanted to determine the prevalence of cardiac diagnoses in children who present with ALTEs and how often ECGs are used.

“This is the largest study of its kind and the first multicenter study looking into the evaluation of cardiac etiologies of ALTEs,” said Matthew D. Elias, MD, a CHOP pediatric cardiology fellow physician. “Hopefully it will contribute to the knowledge base of those providing for patients with ALTEs of what their institutions are using as a diagnostic workup for these patients and what other institutions are doing.”

Dr. Elias conducted the study with V. Ramesh Iyer, MD, MRCP, an attending electrophysiologist, and Meryl S. Cohen, MD, an attending cardiologist and director of the echocardiography laboratory. They reported their findings in the April issue of Pediatric Emergency Care.

The study team analyzed data from the Pediatric Health Information System database and found that ALTEs are relatively common, accounting for 2,179 hospital encounters at 43 children’s hospitals during a 15-month period from 2009 until 2010. Their analysis showed that 16 percent of these patients (355) had a cardiac diagnosis; however, due to the database’s limitations, the study team could not determine that cardiac pathology was related to the ALTEs.

The study’s main finding was that the hospitals had a wide range in the use of diagnostic ECGs, which highlights the absence of a systematic approach to ALTEs.

“Some hospitals almost never ordered an ECG, and others almost 100 percent of the time ordered an ECG,” Dr. Elias said. “On average, ECGs were ordered 43 percent of the time.”

When the study team looked deeper into the demographic information to determine any reasons why some patients with ALTEs would receive an ECG and some would not, the one statistically significant finding was that those patients who were older at presentation were more likely to have an ECG ordered.

“ALTEs typically present within the first three months of life, and if a patient were to present later on at several months of age, we think that those providing for the patients might expand their differential diagnosis and start suspecting a possibility of a cardiac disease,” Dr. Elias said.

This study raises a larger question for future research of whether or not ECGs could be helpful as a screening tool to detect or rule out particular cardiac diagnoses for patients with ALTEs.

Permanent link to this article: http://www.research.chop.edu/blog/electrocardiogram-use-varies-infants-altes/

Jul 08 2014

60 Minutes Revisits Cancer Therapy Pioneered at CHOP

curing cancerAustralia’s 60 Minutes recently revisited its story from last year about the revolutionary immune therapy treatment spearheaded by The Children’s Hospital of Philadelphia’s Stephan A. Grupp, MD, PhD. The therapy, in which modified versions of patients’ own immune cells are used to treat acute lymphoblastic leukemia (ALL) has led to dramatic, inspirational results, with several patients achieving complete responses.

“It could be one of the greatest medical breakthroughs of the 21st century: a cure for childhood cancer,” said host Michael Usher.

The most common form of leukemia found in children, ALL has a roughly 85 percent cure rate. However, the remaining 15 percent of ALL cases resist standard therapy. The 60 Minutes piece focused on the researchers’ use of HIV, which the program called “one of the most notorious killers of our time,” to treat ALL. Dr. Grupp and his team have used HIV to deliver engineered T cells — the workhorses of the immune system — to selectively kill another type of immune cell called B cells, which had become cancerous.

HIV “is a terrible virus,” Dr. Grupp said, “but there’s a good property, and the good property of the virus is its ability to put a gene into cells. We isolate just that property and we get rid of all of the bad stuff, so yes, HIV’s been retasked to do good in this kind of treatment.”

The piece is also focused on one of Dr. Grupp’s patients, 5-year-old Austin, whom 60 Minutes profiled in its original piece. Diagnosed with ALL when he was 2, Austin is now in complete remission for the first time. “To see him doing normal things is a relief, and a dream come true, because those were things that were put on hold for awhile,” said Austin’s mother Kim, who called the treatment “a miracle.”

“I think we do have a potential new treatment that didn’t exist before that we hope will be available to other patients and other hospitals soon, and across the world, in the next year or two,” Dr. Grupp said.

To watch the 60 Minutes report, see below!

Permanent link to this article: http://www.research.chop.edu/blog/60-minutes-revisits-cancer-therapy-pioneered-chop/

Jul 07 2014

PolicyLab Reports on Students Involved With Child Welfare

child welfare

“Improving outcomes for these students starts with identifying and deploying the appropriate resources that these schools need to support all students,” Dr. Rubin stated.

Educators and policymakers in Philadelphia knew that meeting the complex needs of the most vulnerable students in struggling public schools was a big challenge, but they did not know exactly how big. So they turned to PolicyLab at The Children’s Hospital of Philadelphia to do the math.

A report released in June by PolicyLab revealed that 17 percent of all Philadelphia public school students have been involved with the child welfare or juvenile justice systems, and it described the educational barriers that these students face.

“This report illustrates the magnitude of youth in our school district who have been involved with the child welfare system — 20 percent by the high school years, and for many schools more than a third of their student body,” stated David Rubin, MD, MSCE, co-director of PolicyLab, in a news release.

The analysis showed that students who had interaction with the Philadelphia Department of Human Services (DHS) had poor academic outcomes, were less likely to be promoted to the next grade on time, earned fewer credits during the year, had lower scores on standardized assessment tests, were more likely to receive special education services, and were absent more days from school.

The report — “Supporting the Needs of Students Involved With the Child Welfare and Juvenile Justice System in the School District of Philadelphia” — was commissioned by the Mayor’s Office of Education, School District of Philadelphia (SDP), Philadelphia School Reform Commission, and DHS to help inform policy decisions to align resources to best meet the needs of the diverse student population across the SDP. PolicyLab collected and analyzed data in a targeted cross-system review of students in the third, seventh, ninth, and twelfth grades from the 2011-2012 academic year across all schools within the SDP.

In addition to determining that a substantial percentage of the school population had involvement with DHS, the report also found that these students tend to cluster in certain school types. Within these struggling schools, the report noted, performance was poor even for those students without DHS involvement, demonstrating the need to transform all students’ educational experience in these difficult environments.

“Improving outcomes for these students starts with identifying and deploying the appropriate resources that these schools need to support all students,” Dr. Rubin stated.

At a press conference announcing the report’s key findings, DHS Commissioner Anne Marie Ambrose announced an action plan that will include stationing 27 social workers as educational liaisons in public schools with high concentrations of DHS-served youth to help support their learning needs in the 2014-2015 school year. But the report’s partners admitted that they alone cannot address all of the challenges that PolicyLab uncovered.

“This can only be one part of the solution,” wrote the project’s director, Sophia Hwang, MSED, in a PolicyLab blog. “In order for our most at-risk students to achieve academically, they also need safe school environments, appropriate behavioral health services, and caring and supportive mentors.”

As a former SDP high school science teacher, Hwang brought a unique perspective to the report. She recalled not knowing to what extent the approximately 160 students who she taught every day were touched by the child welfare or juvenile justice system. Based on the report’s data, she estimates it is likely that one in every three students on her roster was involved with DHS in some way.

“Now, as a researcher, I ask myself, ‘What supports did my children deserve? What resources would have helped me as their teacher?’” Hwang wrote.

With data from this new report in hand, those making educational policy decisions in Philadelphia and in large cities throughout the country can address those questions more strategically.

“I hope that our report will inspire conversations in other urban school districts about the resources and supports needed for students in their communities,” Hwang wrote.

For more information and to view the full report, please visit Sophia Hwang’s blog post on PolicyLab’s website

Permanent link to this article: http://www.research.chop.edu/blog/policylab-reports-students-involved-child-welfare/

Jul 03 2014

Researchers Examine Goal Pursuit in Teens with Cancer

cancerSetting and achieving goals is a significant milestone in adolescent and young adults’ (AYA) development that paves the way for independence in adulthood. Teens with cancer often experience fatigue, pain, and other symptoms that can interfere with these personal goals, which psychologists refer to as health-related hindrance (HRH).

Researchers at the Center for Childhood Cancer Research at The Children’s Hospital of Philadelphia are interested in identifying those most at risk for higher HRH because it is a potential marker of poor psychological adjustment during cancer treatment and into survivorship.  One of their studies that appeared in the March issue of the Journal of Clinical Psychology in Medical Settings focused on the interaction between race, ethnicity, and income in predicting HRH.

“This is one of the first studies to look at the psychosocial components of lower income and ethnic minority status on quality of life outcomes,” said Lauren C. Daniel, PhD, a psychology fellow in the division of oncology at CHOP.

Ninety-four study participants between the ages of 13 and 19 were categorized into four groups: lower income minority status, higher income minority status, lower income white, and higher income white. On average, it was 1.65 years since their initial diagnosis, and the majority had received chemotherapy.

The study participants wrote down up to 10 personally meaningful goals. Some examples included spending more time with friends and getting into a good college. Participants rated the difficulty and importance of the goal as well as the impact of pain, fatigue, and other symptoms on achieving each goal.

The research team examined differences in goal-related variables across the four groups, and their findings were not what they had anticipated. Based on previous research on health care disparities, they had hypothesized that adolescents with cancer of minority status living in lower income families would experience the highest amount of HRH. The results showed the opposite: Lower income minority patients had the least amount of HRH, while high income minority patients had the most.

“It may be that higher income minority families are in schools with higher expectations so patients may be more aware of the impediments that cancer poses,” Dr. Daniel said.

On the flip said, the researchers suggested that those adolescents with cancer in the lower income minority groups may rely on a “shift and persist” adaptive coping style that increases their resilience and persistence toward valued goals.

“This study highlights that goal setting may be different across patient populations,” Dr. Daniel said. “Their goals also might be different than what clinicians are expecting. So it’s important for them to talk with patients about things that they want to achieve, especially understanding how fatigue, pain, and symptoms impact their goals.”

During these conversations, clinicians can work with AYA to set realistic goals in the context of cancer to help motivate them to look toward the future. They also can connect them with organizations that offer information and support or scholarships and financial aid. At CHOP, many unique resources are available for AYA who are on and off treatment.

A larger scale study across multiple centers is needed to fully understand these preliminary findings and further explore the impact of sociodemographic factors on HRH.

“We’re hoping to work toward implementing interventions to improve health-related hindrance as an important part of quality of life of AYA with cancer,” Dr. Daniel said.

This study was part of a larger study on developmental outcomes of AYA with cancer by Lisa A. Schwartz, PhD, assistant professor of clinical psychology in pediatrics at CHOP and the psychologist for the Cancer Survivorship Program. Lamia P. Barakat, PhD, a CHOP psychologist, and Lauren D. Brumley, also contributed to the study.

Permanent link to this article: http://www.research.chop.edu/blog/researchers-examine-goal-pursuit-teens-cancer/

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