Bench to Bedside

October 2013

Children’s Hospital First in Nation to Disallow Dietary Supplements


The Children’s Hospital of Philadelphia has announced that its Formulary — the list of medications approved for use — will no longer include most dietary supplements. The hospital said the action was being taken because the Food and Drug Administration (FDA) does not routinely review the manufacturing of dietary supplements, and therefore cannot guarantee their safety and effectiveness.

The move makes CHOP the first hospital in the United States to discourage patients from using these products without a doctor’s provision as a matter of policy.

Dietary supplements are defined as vitamins, minerals, herbs, botanicals, amino acids, enzymes, and animal extracts meant to “supplement” the diet and are not intended to replace a healthy diet or to treat, diagnose, prevent, or cure diseases. Melatonin, Echinacea, chondroitin sulfate, glucosamine, CoEnzyme Q10, milk thistle, and probiotics are some of the most commonly used supplements.

“Because vitamins and dietary supplements are essentially unregulated, there is no sound information about adverse side effects, drug interactions, or even standard dosing for the vast majority of them,” said Sarah Erush, PharmD, BCPS, Pharmacy Clinical Manager and a member of the hospital’s Therapeutic Standards Committee. “Administering these medications — particularly to children with serious health complications — is unethical when the risks are unknown, and when there are alternative treatments that have been proven in clinical trials to be safe and effective.”

The hospital’s updated policy acknowledges that there are certain medical conditions that may require supplementation of vitamins or nutrients. To that end, the hospital has determined a very limited and carefully selected list of acceptable products that are proven to be of high quality and safe.

Under the hospital’s updated policy, parents or guardians will be asked upon admission whether the patient is taking any medication or supplements. If so, the attending nurse or physician will review the hospital’s policy discouraging the use of supplements and inform parents or guardians of the potential risks associated with the supplement. Potential risks include contamination, mislabeling, interactions with medications, or potential unforeseen adverse effects.

If, after receiving this information, a parent or guardian insists on continuing to give their child a dietary supplement that is not on the CHOP Formulary, they must sign a hospital waiver stating that they agree to be responsible for providing the product.

“Educating families is one of the most important reasons for implementing this new policy. Most people assume that supplements they buy at the health food store or online are strictly monitored or are safe because they are ‘all natural’,” said Dr. Erush. “But supplements are only subject to FDA review if an adverse event is reported, so there are many for which we have no reliable data. We’d much rather treat children with what we know works than provide them with a substance that may at best do nothing, or at worst, cause harm.”

In order to be included in CHOP’s formulary, all products must follow similar guidelines as for FDA-approved medications. If able to meet these criteria, pharmacy will stock and dispense as a formulary medication, avoiding the need for a waiver:

“CHOP has long embraced its responsibility to advance patient safety as the cornerstone to improving children’s health.” said Paul Offit, MD, Chair of the hospital’s Therapeutic Standards Committee. “Patients with chronic diseases use dietary supplements more frequently than the general population and are at greater risk for adverse events and interactions. Better monitoring and regulating the way we distribute these products is one more step we can take to make sure that we’re providing the best possible medical care for our children.”

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Pennsylvania Program to Support Genomic, Developmental Screening Studies


Two recent state awards to Children’s Hospital researchers show the incredible breadth of research being conducted at The Children’s Hospital of Philadelphia Research Institute every day. Supported by the Pennsylvania Department of Health’s Commonwealth Universal Research Enhancement program, Struan Grant, Phd, will lead an investigation of the genetic links between type 2 diabetes and cancer, while Jennifer F. Culhane, PhD, will study developmental-behavioral screening instruments in children.

Established in 2001, the Commonwealth Universal Research Enhancement (CURE) program awards grants to biomedical, clinical, and health services research projects. Through 2011, the program supported 1,672 research and infrastructure projects with a total of $698 million in grants, according to the CURE website.

Last year, CHOP’s Stephen Grupp, MD, PhD, testified before the Pennsylvania State Senate on behalf of CURE after the program’s funding was threatened. Dr. Grupp, whose work received CURE support, in March 2013 published a study showing a two leukemia patients achieved complete responses after receiving T cells engineered to selectively kill cancerous cells.

“These cells don’t come from an insurance company, and they don’t come from a drug company. They come from research dollars,” Dr. Grupp said.

While the CURE funding ultimately avoided being cut last year, Pennsylvania recently announced that due to a sharp reduction in the amount of money it was due to receive through the Tobacco Master Settlement Agreement — which resolved healthcare lawsuits brought by 46 states against tobacco companies — the state would be forced to reduce funds for a number of programs, including CURE. However, Drs. Culhane and Grant’s projects will continue to be supported.

Projects Span the Research Spectrum

Dr. Culhane, an associate professor of Pediatrics at Children’s Hospital and the University of Pennsylvania, will lead a comparison of the screening instrument the Survey of Wellbeing of Young Children (SWYC). Recently developed by Tufts Medical Center’s Ellen C. Perrin, MD, and R. Christopher Sheldrick, PhD, the SWYC is a “freely-available, comprehensive screening instrument for children under 5 years of age,” according to the SWYC website.

Because the SWYC is “brand new” and is still being researched, with her project Dr. Culhane will be running a validation study of the survey to determine its applicability in underserved populations. Dr. Culhane and her team plan to compare the SWYC to previously validated instruments, she said.

The SWYC is comprised of three different “domains,” which together can give a comprehensive overview of a child’s development. Questionnaires related to behavior, development (including an autism screening), and questions related to family are included. That the SWYC is “free and short” could help it become an important screening tool, Dr. Culhane pointed out.

While the Tufts researchers have been focused on “more typical populations” in their validation studies, Dr. Culhane’s study will examine “very unique populations” of children of Spanish-speaking women — many of whom may be undocumented — and children of black women born in Africa who have immigrated to the United States. She and her colleagues, including Children’s Hospital’s Marsha Gerdes, PhD, who produced a Spanish translation of the SWYC, are already recruiting study participants, and hope to evaluate approximately 1000 patients.

Dr. Grant’s project, meanwhile, will examine the genetic commonalities of type 2 diabetes and cancer. In particular, Dr. Grant, who is a faculty member in the department of genetics at CHOP, will investigate the function of key genes that come in both disease settings, in particular TCF7L2, which is the gene most strongly associated with type 2 diabetes. Dr. Grant discovered the association between type 2 diabetes and TCF7L2 in 2006, and “thus far, it is the most strongly associated gene in type 2 diabetes reported to date,” Dr. Grant said.

But researchers do not yet fully understand the role TCF7L2 plays in causing type 2 diabetes. Moreover, TCF7L2 has also been implicated in the pathogenesis of certain types of cancer. For example, Dr. Grant pointed out, “if you get a different flavor of mutation in this gene, you’ll get colorectal cancer. So this gene is clearly both a colorectal cancer gene and a type 2 diabetes gene,” Dr. Grant said.

And follow up studies of genome-wide association study findings have revealed a key relationship between TCF7L2 and multiple forms of cancer. “So TCF7L2 is not only the top story in type 2 diabetes, it seems to be also explaining the top story in multiple cancer settings,” Dr. Grant noted.

Supported by the CURE funding, Dr. Grant hopes to “understand why there is a specific common genetic etiology to type 2 diabetes and cancer, primarily prostate and colorectal cancer.” Dr. Grant has been working with a number of CHOP and Penn faculty on the project, including Center for Childhood Cancer Research investigators Adam Resnick, PhD and Andrei Thomas-Tikhonenko, PhD, as well as Penn geneticist Klaus H. Kaestner, PhD, and Penn diabetes researcher Morris J. Birnbaum, MD, PhD.

To learn more about the CURE program, see the Pennsylvania Department of Health site.

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PolicyLab’s David Rubin, MD, MSCE, Appointed to Child Abuse Commission


Co-director of The Children’s Hospital of Philadelphia’s PolicyLab David Rubin, MD, MSCE, was recently appointed to a government commission tasked with working to end child abuse and neglect-related deaths. Dr. Rubin is one of twelve experts from a variety of fields to be named to the Commission to Eliminate Child Abuse and Neglect Fatalities, and one of just six appointed by President Obama. Notably, Dr. Rubin is the only health care provider appointed to the commission.

The commission was established as part of the Protect Our Kids Act of 2012. By establishing a commission of child welfare experts, pediatricians, and jurists, the Protect Our Kids Act seeks to “develop a national strategy and recommendations for reducing fatalities resulting from child abuse and neglect,” according to the law’s text. A rare example of bipartisan accord, the House of Representatives voted 330-77 in favor of the act before it quickly proceeded through the Senate. President Obama signed the act into law on January 14, 2013.

The Protect Our Kids Act was inspired in part by a 2011 Government Accountability Office (GAO) report on child fatalities from abuse and neglect. According to the National Child Abuse and Neglect Data System (NCANDS) — a voluntary reporting system that collects data from all 50 states as well as the District of Columbia and Puerto Rico — in 2009 there were 1,770 child deaths due to abuse and neglect.

However, the true number of child deaths that year was likely higher, the GAO report states, both because the NCANDS is a voluntary data-collection system, and because “nearly half of states report to NCANDS data only on children who were already known to” child protective services (CPS). However, not all children who die from abuse or neglect are brought to the attention of CPS, the report states.

“As a society, we should be doing everything in our collective power to end child deaths and near deaths from maltreatment, and the collection and reporting of comprehensive data on these tragic situations is an important step toward that goal,” the GAO report concluded.

Commission Mission, Duties

As a first step in preventing child deaths from mistreatment, the Protect Our Kids Act charges the Commission to Eliminate Child Abuse and Neglect Fatalities with conducting a survey of child welfare and protection services, in order to determine the effectiveness of existing policies and services. The commission, which will offer education opportunities to graduate students, will also examine barriers to improving child welfare, child abuse and neglect trends, and methods of improving data collection.

Based on its work, the commission will “develop recommendations to reduce fatalities from child abuse and neglect,” as well as developing data tracking guidelines. The commission is due to submit a report to the President and Congress on its work within two years.

Six members of the commission were appointed directly by President Obama, while three were appointed by the House of Representatives and three by the Senate. In addition to Dr. Rubin, commission members include Theresa Martha Covington, MPH, director of the National Center for the Review and Prevention of Child Deaths, Marilyn Bruguier Zimmerman, MSW, director of the National Native Children’s Trauma Center, and Susan Dreyfus, president and CEO of the Alliance for Children and Families. The Commission will be chaired by David Sanders, PhD, executive vice president of Casey Family Programs.

Along with current co-director Kathleen Noonan, JD, in 2008 Dr. Rubin founded PolicyLab, which works to develop evidence-based solutions for the most challenging health-related issues affecting children. In addition to his role at PolicyLab, Dr. Rubin is an associate professor of Pediatrics as the Perelman School of Medicine at the University of Pennsylvania, a senior fellow at the Leonard Davis Institute of Health Economics, and an associate program director of the Robert Wood Johnson Clinical Scholars Program at the University of Pennsylvania.

“As a pediatrician in Philadelphia, I see the toll that stress has on children and families,” Dr. Rubin said. “I am honored to have the opportunity to serve this commission and hope to bring my experiences serving children and families to think preventatively at the system level about what can be done to avoid unnecessary childhood injuries and fatalities.”

To read more about PolicyLab’s child welfare work, see the PolicyLab site.

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Defense Department Grant to Fund Advanced Transplantation Study


A recent grant from the Department of Defense (DoD) to The Children’s Hospital of Philadelphia will fund a new study of advanced transplantation techniques. The study, which is being led by Children’s Hospital’s Wayne Hancock, MBBS, PhD, FRCPA, will focus on vascularized composite allotransplantation (VCA), a type of transplantation in which multiple tissues — such as an entire hand — are transplanted “as a functional unit,” according to the American Society of Transplantation (AST). In particular, Dr. Hancock and his team will be working to develop “new approaches to immunosuppression,” he said.

Dr. Hancock, the chief of Children’s Hospital’s Division of Transplant Immunology, will act as the project’s principle investigator, while L. Scott Levin, MD, FACS, the Paul B. Magnuson Professor of Bone and Joint Surgery at the University of Pennsylvania, will lead the University of Pennsylvania’s efforts. In addition to Drs. Hancock and Levin, Assistant Professor of Surgery at the University of Pennsylvania Matthew H. Levine, MD, PhD, will contribute to the investigation.

With this four-year, $2 million grant, CHOP and Penn join a consortium of institutions — including the University of Maryland and the Christine M. Kleinert Institute for Hand and Microsurgery — led by Emory University that will examine advanced transplantation techniques through the DoD’s Restorative Transplantation Research program. Overall, the research program seeks to “make a significant impact on improving the function, wellness, and overall quality of life” for wounded members of the military.

A study published last year in the Medical Surveillance Monthly Report, a publication of the Armed Forces Surveillance Center, underscores the DoD’s interest in advancing VCA. The study found that between 2000 and 2011, U.S. military service members underwent 6,144 “traumatic amputations.” Of these, about one third were “major amputations” of a hand or limb.

“Because of improvements in protective equipment and innovations in medical evacuation procedures and battlefield trauma care … many severely injured service members, who might have died in earlier wars/conflicts, survived their injuries with significant disabilities, including amputations,” the study’s authors note.

Weighing Transplantation’s Costs, Benefits

According to the AST, there have been a number of hand and face transplants over the last decade worldwide, with approximately 46 patients receiving 66 hand transplants. However, while clinicians can perform hand or limb transplants, whether they should is in question, as the burden of requiring ongoing immunosuppression when performing such transplants still outweighs their benefits, Dr. Hancock said.

This is because the effects of the immunosuppressive drugs that accompany any transplantation can outweigh the procedure’s benefit. After receiving a liver, kidney, heart or lung transplant, patients must agree to undertake a lifelong regimen of medications to prevent organ rejection, including the immunosuppressives tacrolimus or cyclosporine, mycophenolate, sirolimus, and steroids like prednisone.

There are a number of adverse effects associated with these drugs, which patients must take for the rest of their lives. For instance, some of the many side effects associated with tacrolimus are diarrhea, constipation, nausea, vomiting, dizziness, hypertension, liver and kidney issues, neuropathy, seizures, and depression.

In the case of organ transplants — without which one cannot live — the “risk-benefit ratio weighs in favor of taking the risk, because the benefit is one of life,” Dr. Hancock said. The ethics of performing a limb transplant remain less clear, he noted. And managing the body’s immune response to a transplant can be as tricky, if not trickier, than the surgery itself, Dr. Hancock pointed out.

So with this project Dr. Hancock and his team will work to “come up with some new strategies that don’t involve conventional immunosuppression.” The investigators’ work will begin in mice, eventually moving to humans once the researchers show that their ideas work. “We’re using knowledge that we’ve developed over years to show that we can do limb transplants in small animal models,” he said.

And though the project is not pediatric-specific, Dr. Hancock noted that it was “possible that the techniques developed could eventually be useful for limb transplants in kids.” Researchers might eventually be able to use what they learn to perform limb transplants on children with congenital limb abnormalities.

To learn more about The Children’s Hospital of Philadelphia’s transplant services and research, see the Pediatric Transplant Center.

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Children’s Hospital Neonatologist Elected to Institute of Medicine


Phyllis A. Dennery, MD, FAAP, an internationally prominent neonatologist and researcher at The Children’s Hospital of Philadelphia, has been elected to the prestigious Institute of Medicine (IOM).

Dr. Dennery is the chief of the Division of Neonatology and Newborn Services at Children’s Hospital and holds the Werner and Gertrude Henle Endowed Chair in Pediatrics. She is one of 70 new members elected to the IOM in recognition of their major contributions to the advancement of the medical sciences, health care, and public health.

Dr. Dennery focuses her research on oxidative stress-mediated neonatal lung gene regulation and on the biology of lung injury and repair. She runs a National Institutes of Health-funded laboratory and has published her findings in highly respected peer-reviewed journals such as the Journal of Biological Chemistry, New England Journal of Medicine and Blood, among others. Her clinical interests are in neonatal jaundice, bronchopulmonary dysplasia, and the long-term consequences of prematurity.

Dr. Dennery focuses her research on oxidative stress-mediated neonatal lung gene regulation and on the biology of lung injury and repair. She runs a National Institutes of Health-funded laboratory and has published her findings in highly respected peer-reviewed journals such as the Journal of Biological Chemistry, New England Journal of Medicine and Blood, among others. Her clinical interests are in neonatal jaundice, bronchopulmonary dysplasia, and the long-term consequences of prematurity.

Throughout her career, she has received many awards and honors, including the Andrew Mellon Fellowship, the Alfred Stengel Health System Champion Award from the Perelman School of Medicine at the University of Pennsylvania, and the Mentor of the Year Award from the Eastern Society of Pediatrics.

In 2010, she was appointed to the U.S. Secretary of Health and Human Services Advisory Committee on Infant Mortality, and she has also served on the Community Action Team of the Medical Examiner’s Office in Philadelphia focused on infant mortality.

After receiving her medical degree from Howard University College of Medicine, Dr. Dennery completed her residency at Children’s National Medical Center and a fellowship in Neonatology at Rainbow Babies and Children’s Hospital. She was on the faculty at Stanford University where she served as director of Neonatology Research and associate division chief, before coming to Children’s Hospital of in 2003 to assume the role of division chief of Neonatology.

Established in 1970 by the National Academy of Sciences, the IOM honors professional achievement in the health sciences and serves as a national resource for independent analysis and recommendations on issues related to medicine, biomedical sciences, and health. Current members of the Institute elect new members from a slate of candidates nominated for their professional achievement.

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CHOP Genetics Expert Co-Leading NIH-Funding Psychiatric Illness Consortium


An international collaboration will delve into why patients with chromosome 22q11.2 deletion syndrome have an elevated risk of schizophrenia and other psychiatric illnesses. Genetics experts from The Children’s Hospital of Philadelphia are among the top leaders of this major collaboration, which aims to discover the genes implicated in the syndrome and to shed light on the biological causes of mental illness in the general population.

Found in approximately 1 in 4,000 live births, 22q11.2 deletion syndrome (22q11.2 DS) involves birth defects and developmental and behavioral differences. Caused by the absence of a DNA sequence on the long arm of chromosome 22, the syndrome carries many possible signs and symptoms that may affect the heart, palate, the immune and endocrine systems, and the kidneys. Some patients may have seizures, hearing loss, scoliosis, or feeding and swallowing problems.

In addition, many children with 22q11.2 DS have developmental delays, including learning disabilities and delays in language emergence, or may have an autism spectrum disorder, attention-deficit hyperactivity disorder, anxiety or obsessive-compulsive disorder. As they enter adolescence or young adulthood, approximately 25 to 30 percent of patients are at risk of developing schizophrenia, which represents a much higher rate than the one percent rate found in the general population.

Donna McDonald-McGinn, MS, CGC, program director of the 22q and You Center at CHOP, is the co-director of the International Consortium on Brain and Behavior in 22q11.2 Deletion Syndrome, which has just received a $12 million, four-year grant from the National Institute of Mental Health, part of the National Institutes of Health. The consortium brings together caregivers and scientists from 22 clinical centers and five genotyping sites throughout the world.

The consortium’s research tools will include whole-genome sequencing to uncover genetic variation among patients with chromosome 22q11.2 DS. Children’s Hospital, which has a long-standing research and clinical program focused on this condition, has evaluated more than 1,200 patients, making it the world’s largest center of its kind.

Two genetics researchers with global prominence in studying this diagnosis, Beverly S. Emanuel, Ph.D., and Elaine Zackai, M.D., are co-investigators at CHOP’s site in the Brain and Behavior Consortium. Among many other accomplishments, in 1995 both scientists were on the team that created an early genetic map of chromosome 22, setting the stage for it to become the first chromosome sequenced in the Human Genome Project.

While focusing on the genetic roots of psychiatric disorders in 22q11.2 DS, the consortium leaders expect to identify biological pathways leading to schizophrenia in the broader population, with the hope that such data may inform novel, more effective treatments.

For more information, please visit the Research Institute’s website.

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New Toolkit Offers Innovative Data Resource for Biomedical Researchers


Biomedical researchers often confront large quantities of information that may be amassed in many forms: vital signs, blood cell counts, lengthy DNA sequences, bar graphs, MRIs, patient demographics, and so much more. How do researchers assemble, access and analyze all that data without having to become specialized database technicians themselves?

A team of informatics experts and biomedical researchers from The Children’s Hospital of Philadelphia (CHOP) have launched a new software toolkit to help researchers manage data. Their open-source, highly interactive framework, known as Harvest, is designed to let users to navigate quickly among different types and levels of data.

“We want to help researchers explore their data, not their database,” said Byron Ruth, lead developer of Harvest at CHOP’s Center for Biomedical Informatics (CBMi). Ruth is one of the co-authors of a paper introducing CBMi’s Harvest framework, which was published recently in the Journal of the American Medical Informatics Association.

“Research institutions typically work through their information technology staff to provide a single data warehouse that may be too general-purpose for all its projects, or develop one-off solutions on a case-by-case basis for each project,” co-author Michael J. Italia added. “Our approach in Harvest is different,” said Italia, CBMi’s manager of Applications Research. “We decided to focus on end-users, generalizing the toolkit for application to any biomedical study with multiple collaborators, but also allowing individual software developers and data managers to customize the software for specific projects.”

Harvest, said Italia, “isn’t just shrink-wrapped, ready-to-go software.” He estimates that Harvest typically provides 80 percent of the work, leaving it to any institution’s software developer to adapt the framework to a project’s needs, in collaboration with each project’s principal investigator. Harvest is open-source, so users are free to see bug reports, check software patches, and share fixes and customizations with a wider community of users.

A key feature of Harvest is the ability to maneuver smoothly among various levels of data, from individual patient records to aggregated reports of all patients in a database, and to subpopulations in between. Users can construct queries to slice and dice data — grouping subjects, for instance, by age or ethnicity, calling up individual blood test results or MRIs, or including or excluding specific diagnoses.

One advantage of Harvest is that it provides transparency and visibility to data in a manner that is familiar to a researcher who is invested in a particular disease or project.

“Harvest adopts convenient and clear interfaces to view and explore data that are increasingly used in other industries, such as social media,” said the paper’s senior author and CBMi director Peter S. White, Ph.D. “We have found that this often helps users to quickly familiarize themselves with data they are seeing, which increases the likelihood that they will trust the resource, and even be incentivized to contribute to the project as it develops.”

The Harvest developers say their tool reflects the growing complexity of research in the Big Data era of electronic health records and genomic technology. In the 1980s and 1990s, much federal research followed a hypothesis-driven model, focusing on predefined measurements within a patient population. Many current databases collect vast amounts of many data types with fewer preconceived notions of what is significant. “Harvest allows users to formulate and refine questions and even explore a different view of data that wasn’t apparent or important to them a few minutes before,” said Italia.

The CBMi team originally designed Harvest to manage data from AudGenDB, an audiology database funded by the National Institute on Deafness and Other Communications Disorders. In the current paper, the study team evaluated Harvest by running it through its paces with data from two other collections: CardioDB, which stores clinical data from 47,000 of CHOP’s pediatric cardiology patients, and OpenMRS, a public dataset holding lab results, infection status and other clinical results from electronic health records.

CBMi is now applying Harvest to more collections, including the Longitudinal Pediatric Data Resource, a long-term informatics system being created to store clinical data derived from thousands of children with conditions detected in national newborn screening programs. “Although these collections contain very different types of data, the Harvest toolkit is flexible enough to adapt to these different scenarios, while at the same time maintaining a consistent set of basic components,” added Ruth.

Harvest has been focused primarily on the end-user up to this point. Future work includes a bigger focus on software developers and applications support personnel who are responsible for setting up and maintaining Harvest applications. “However, as we think about the life cycle of a Harvest application, we want it to be as easy as possible for administrators and software developers to get Harvest into the hands of researchers,” Italia said.

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Offering Basic Scientists an Introduction to Translational Research


In an example of CHOP Research’s commitment to educating the next generation of scientists, a recent workshop held at the Institute introduced young investigators to the principles of translational research. Led by a committee of Children’s Hospital faculty and organized by the CHOP Research Office of Postdoctoral Affairs, the 2013 Pediatric Translational Research Workshop for Basic Scientists brought together a group of graduate students, postdocs, and early-career researchers for a week of presentations, clinical visits, and discussions.

“The workshop was designed to provide participants with the fundamental tools and knowledge to bring their basic research discoveries to the clinic,” said Deputy Scientific Director of CHOP Research Tom Curran, PhD, FRS, who was the workshop’s facilitator and the lead faculty organizer. “In addition to cutting-edge scientific presentations from top CHOP and UPenn researchers, the rigorous agenda included lectures and discussions focused on basic/clinical research collaborations, navigating an Institutional Review Board, and evaluating the ethics of translational research,” he added.

Workshop attendees came from a number of Philadelphia-area universities. In addition to The Children’s Hospital of Philadelphia, institutions represented included the University of Pennsylvania, Temple University, Thomas Jefferson University, the University of Medicine and Dentistry of New Jersey (now Rutgers Biomedical and Health Sciences), and the Monell Chemical Senses Center.

In his welcome address on the workshop’s opening day, Dr. Curran acknowledged there was “no simple answer” to pursuing a career in translational research. However, in order to give the participants “a real feel for translational research,” the workshop would offer “specific, in-depth experiences,” he said. Dr. Curran also noted how important collaboration with other researchers — particularly cross-disciplinary collaboration — is to successful translational research.

“Working as part of a team can be one of the most rewarding experiences you’ll have,” Dr. Curran said.

The weeklong workshop featured speakers from a variety of disciplines from Children’s Hospital and the University of Pennsylvania. Among the leading CHOP researchers who presented were Philip R. Johnson, MD, director of CHOP Research, Louis Bell, MD, chief of the division of General Pediatrics, Peter Adamson, MD, chief of the division of Clinical Pharmacology and Therapeutics, and fetal surgeon Alan Flake, MD, director of the Center for Fetal Research. The sessions spanned the research spectrum, from those on cystic fibrosis therapies to mitochondrial disease to several talks on translational research in neurodevelopmental disorders.

Translational Research’s Impact

For his part, CHOP Research’s Dr. Johnson presented the workshop participants with three examples of how translational research has affected Children’s Hospital patients. Saying that his job was to make translational research “real” for the attendees, Dr. Johnson discussed three case studies: the work done by Stephan Grupp, MD, PhD, to treat acute lymphoblastic leukemia with T cell therapy; fetal surgery to correct myelomeningocele, a devastating form of spina bifida, which is led by CHOP’s N. Scott Adzick, MD; and the investigation by the University of Pennsylvania’s Albert M. Maguire, MD, and Jean Bennett, MD, PhD, into gene therapy to treat eye disease.

Translational research is not about “instant gratification,” Dr. Johnson said, noting that it can take decades for work to go from the bench to the bedside. But translational research offers investigators the opportunity to see their work pay off and make an impact on patients, he said.

To give attendees a sense of the effect translational research can have, the workshop also included visits from patients and patient families — including those with cystic fibrosis, the developmental disorder Cornelia de Lange Syndrome, the rare genetic disease Fibrodysplasia ossificans progressiva, and mitochondrial disease — and a visit to the Hospital’s pediatric intensive care unit (PICU). The PICU visit, and a discussion of the attendees’ impressions, was moderated by Athena Zuppa, MD, MSCE, an attending physician in Pediatric Critical Care medicine.

Several outside speakers gave talks during the week, including former FDA official Stephen Spielberg, MD, PhD, current editor-in-chief of Therapeutic Innovation and Regulatory Science, and Richard Woodward, PhD, chief executive officer of Vascular Magnetics, Inc. Dr. Spielberg discussed how industry, academics, and the FDA view translational medicine, while Dr. Woodward’s talk was focused on setting up a biotechnology business. Workshop participants were also given the chance to network with each other and CHOP and UPenn faculty at events both on and off campus.

And last but not least, Joseph W. St. Geme, III, MD, Children’s Hospital’s physician-in-chief and chair of the Department of Pediatrics at the University of Pennsylvania, delivered the workshop’s closing address. A noted microbiologist, Dr. St. Geme recently joined CHOP after spending a number of years at Duke University, where he served as chairman of the Department of Pediatrics and chief medical officer.

“The Pediatric Translational Research Workshop for Basic Scientists reflects the CHOP Research Institute’s commitment to the next generation of leaders in translational science,” said Dr. Curran.

To read more about the 2013 Pediatric Translational Research Workshop for Basic Scientists, click here. And to learn more about The Children’s Hospital of Philadelphia’s educational resources, see the CHOP Research site.

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