Bench to Bedside

June 2013

CHOP Earns #1 Ranking on U.S. News & World Report’s Best Children’s Hospital List


We’re pleased to announce that The Children’s Hospital of Philadelphia earned the number one ranking in the recently released 2013-14 U.S.News & World Report Honor Roll of Best Children’s Hospitals. In addition to the Honor Roll ranking, CHOP programs ranked in the top four in each of the ten specialty areas evaluated in the U.S. News survey.

The annual rankings highlight the top 50 U.S. hospitals in a number of pediatric specialties: cancer, cardiology & heart surgery, diabetes & endocrinology, gastroenterology & GI surgery, neonatology, nephrology, neurology & neurosurgery, orthopedics, pulmonology, and urology.

“We are humbled to be recognized again by U.S. News and World Report with a number one ranking among the nation’s children’s hospitals. This recognition is a tribute to the exceptional work of our staff and their tireless dedication to our patients,” said Steven M. Altschuler, MD, chief executive officer of CHOP.

Children’s Hospital was ranked first in pulmonology and urology, and second in cardiology & heart surgery, diabetes & endocrinology, gastroenterology, and orthopedics. The Hospital ranked third in cancer, nephrology, and neurology & neurosurgery, and was fourth in neonatology.

“As we mark this important milestone, it’s an opportunity to renew our commitment to improve the health of the children who enter our doors, as well as those across the country or the world whom we may never meet,” Dr. Altschuler said.

“The Children’s Hospital of Philadelphia deserves high praise,” said U.S.News & World Report Health Rankings Editor Avery Comarow. “This ranking shows the dedication and expertise that CHOP brings to the care of children who need those qualities the most. We think it is important to identify and call attention to pediatric centers like this one.”

The U.S.News survey methodology combines clinical data points from 179 pediatric centers with a separate reputational score derived from a survey in which 1,500 pediatric specialists — 150 in each specialty — were asked to which hospital they would send the sickest children. More information about CHOP’s rankings is available here.

The full rankings and methodology are available here. The rankings will also be published in the U.S.News Best Hospitals 2014 guidebook, which will be available in August.

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Experts Call for “Human Vaccines Project” to Accelerate Vaccine Development


A group of prominent vaccine researchers, including the Research Institute’s chief scientific officer, Philip R. Johnson, MD, and the University of Pennsylvania’s Stanley Plotkin, MD, recently called for a “human vaccines project” to accelerate the development of vaccines to prevent “major global killers such as AIDS, tuberculosis, malaria, and other infectious diseases.”

In a recently published Science article, the authors point out that though vaccines have been extremely effective in the past, “past strategies … are unlikely to succeed in the future,” against diseases for which there remain no vaccines. And while advances into vaccine discovery have been made, translation of those advances into successful vaccines “remains impeded by lack of understanding of key vaccinology principles in humans.” New clinical research initiatives should be established to accelerate vaccines development, the authors argue.

Wayne C. Koff, PhD, senior vice president and chief scientific officer of the International Aids Vaccine Initiative (IAVI), was the article’s first author. Other contributors include the Scripps Research Institute’s Dennis Burton, PhD, Sanofi’s senior vice president and chief scientific officer Gary J. Nabel, MD, PhD, and the Emory Vaccine Center’s Rafi Ahmed, PhD.

Past Performance Does Not Guarantee Future Results

Vaccines have “led to the eradication of smallpox, near eradication of polio, and prevention of untold millions of deaths from infectious diseases each year, and are one of the most effective public health measures available,” the authors note.

Vaccines have been responsible for marked improvements in health, quality of life, and life expectancy, but there remain many debilitating diseases for which vaccines do not exist. Chlamydia, dengue, Epstein-Barr virus, hepatitis C, herpes simplex, HIV/AIDS, malaria, rhinovirus, and tuberculosis are just some diseases that cannot be inoculated against. Many of these “cause considerable global morbidity and mortality,” the researchers point out.

For example, approximately 50-100 million people worldwide are infected with the dengue virus every year, according to the World Health Organization. Of those, 500,000 progress to the dangerous dengue hemorrhagic fever and roughly 22,000, mostly children, die. Meanwhile, malaria infects more than 200 million people worldwide, and causes approximately 660,000 deaths annually, mostly in Africa. And tuberculosis causes 8.7 million illnesses and 1.4 million deaths a year around the world.

New vaccines are clearly needed. But the development of new vaccines can be difficult, to say the least. The success rates of new vaccines are “not optimal and are even worse for the subset of complex pathogens for which viability and immune-evasion mechanisms present additional challenges,” the researchers note.

In one survey of projects cited in the Science paper, biopharmaceuticals have a 40 percent success rate compared to 22 percent for vaccines. And clinical trials of vaccines can be extremely costly — coming in between $50 and $100 million — and can take years to complete. Trials “often are undertaken with low probabilities of success,” the researchers say.

Better Vaccine Research Through Technology

But the emergence of new technologies — such as whole-genome sequencing— is “fueling a revolution in vaccine discovery,” the researchers say. Antigen discovery technologies, adjuvants (agents added to drugs to increase their effects) and delivery technologies, and next-generation sequencing technologies that will allow researchers to better decipher human immune responses offer a “tremendous opportunity for accelerating vaccine development.”

For example, antigen discovery or “reverse vaccinology” technologies allow researchers to discover antigens by sequencing whole genomes of microorganisms and using bioinformatics to design vaccines, the authors say. Likewise, researchers’ ability to “analyze immune responses at both the single cell and systems level” offers the potential to better understand how the human body responds to vaccines and to assess how protective current vaccines are.

Overall, the successful application of information made available by such new technologies could “shorten the time required for successful development of new and improved vaccines,” the researchers say. To best make use of these technological advances, and to speed the development of vaccines for diseases like dengue and malaria, they call for a “new human immunology-based clinical research initiatives” to be established.

“Collectively, such a “Human Vaccines Project” holds the potential to greatly accelerate the development of next-generation vaccines against major global killers such as AIDS, tuberculosis, malaria, and other infectious diseases; enable more successful vaccine development against allergies, autoimmune diseases, and cancers; and provide a foundation for vaccine development against new and emerging diseases,” the researchers write.

“Vaccines are the ultimate form of preventative medicine,” says CHOP’s Chief Scientific Officer, Dr. Phil Johnson.  “We would much rather prevent diseases than treat them.”

“Among the most effective of public health interventions, vaccines collectively prevent the death of 2-3 million people every year. Yet researchers have not been able to devise broadly effective vaccines against a number of deadly or debilitating infectious agents that have evolved sophisticated mechanisms to evade the immune system. New approaches are needed to prevent infection by such pathogens,” said the IAVI in a statement.

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Antibiotic Usage Education, Interventions Improve Prescription Practices


As disease-causing microbes continue their worrisome trend of developing resistance to commonly used antibiotics, public health experts have called for more selective use of those medicines. A new study suggests that educating pediatricians in their offices, and auditing their prescription patterns, encourages them to choose more appropriate antibiotics for children with common respiratory infections.

“Although much research has focused on improving how hospitals use antibiotics, there have been few studies of interventions in outpatient settings, where the vast majority of antibiotic use occurs,” said study leader Jeffrey S. Gerber, MD, PhD, an infectious diseases specialist at Children’s Hospital. “We focused on increasing appropriate antibiotic prescribing in primary care practices.”

Dr. Gerber and colleagues recently published their study of an “antimicrobial stewardship” program in Journal of the American Medical Association. Last year Dr. Gerber and his team presented their research at the IDWeek conference — a joint meeting of the Infectious Diseases Society of America, the Society for Healthcare Epidemiology of America, HIV Medicine Association, and the Pediatric Infectious Diseases Society.

Antimicrobial stewardship, pointed out Dr. Gerber, usually involves prospective audits of prescription patterns — comparing the prescription for a given diagnosis to the current recommendations of professional organizations (in the current study, as issued by the American Academy of Pediatrics). For this study, stewardship also included personalized, private feedback reports to practitioners, advising them of whether their prescriptions followed current recommendations.

The study concentrated on whether the pediatricians prescribed narrow-spectrum antibiotics, as recommended, or broad-spectrum antibiotics for acute bacterial respiratory infections such as pneumonia, acute sinusitis, and streptococcal pharyngitis (or “strep throat”). All are common conditions for which children receive antibiotics.

The study team randomized 18 pediatricians’ practices in CHOP’s primary care network in New Jersey and Pennsylvania into two groups — one that received the intervention (an hour-long clinician-education session at the practice office, followed by audit and feedback of antibiotic prescribing) and a control group that did not receive the educational session, audit and feedback.

The study encompassed nearly 1.3 million office visits by some 185,000 patients to 162 clinicians over a study period of 32 months, from 2008 to 2011.

Among the intervention practices, broad-spectrum antibiotic prescribing decreased from 26.8 percent to 14.3 percent, or nearly half, compared to a decrease from 28.4 percent to 22.6 percent in the control group. For children with pneumonia, the inappropriate broad-spectrum prescriptions declined by 75 percent among practices receiving the intervention.

The researchers followed the effects of the intervention program for only 12 months, said Gerber, so it is unknown how long the benefits persist. In addition, the study team did not examine whether there were differences between the intervention and control groups in the outcomes of their patients’ infections.

Among other questions, said Dr. Gerber, future studies should investigate how generalizable these findings are to other health systems. Still, he added, “Our findings suggest that extending this type of intervention to the outpatient setting may have important health benefits in children.”

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Study Finds Lowering BMI Improves Insulin Sensitivity in Teens


A new study involving Children’s Hospital investigators found obese teenagers who reduced their body mass index (BMI) by 8 percent or more had improvements in insulin sensitivity, an important metabolic factor related to the later development of type 2 diabetes. To achieve the reduction in BMI, the teens followed a family-based, lifestyle-modification weight loss program that offers the potential to become a broader model.

“This threshold effect that occurs at 8 percent suggests that obese adolescents don’t need to lose enormous amounts of weight to achieve improvements,” said pediatric endocrinologist Lorraine Levitt Katz, MD, of Children’s Hospital’s Diabetes Center for Children.

According to the CDC, obesity has doubled in children and tripled in adolescents in the past 30 years, with 18 percent of those aged 12-19 classified as obese in 2010. Obese children and adolescents are at risk for developing health problems later in life, including diabetes.

The findings were published recently in The Journal of Pediatrics. The article’s first author is Pamela Abrams, MD, a member of the study team while at Children’s Hospital, and currently at St. Luke’s Center for Diabetes and Endocrinology. Children’s Hospital psychiatrist Robert I. Berkowitz, MD was the principal investigator of a clinical trial of weight loss treatments from which the researchers analyzed data for the current study, also contributed to the study.

The investigators analyzed results in 113 primarily urban adolescents, aged 13 to 17, of whom 81 percent were female and 62 percent were African American. At the start of the study, their mean BMI was 37.1, placing them in the severely obese range. None had type 2 diabetes, but their obesity placed them at high risk for developing the disease in the future.

An important goal of the study was to determine the threshold of weight loss that significantly impacted insulin sensitivity, glucose tolerance, and the presence of metabolic syndrome (MS). MS, as well as abnormal values in insulin sensitivity and glucose tolerance, is associated with the development of type 2 diabetes. In type 2 diabetes, the body is unable to produce enough or properly use insulin. Improved insulin sensitivity reflects a better ability to process insulin.

The main finding of the current study was a significant improvement in all measures of insulin sensitivity. There was also a trend toward improvement in metabolic syndrome.

The threshold of 8 percent — the level at which a decreased BMI showed improved insulin sensitivity — was consistent with results found previously in adults. Importantly, said Dr. Abrams, “while the ideal goal is to achieve normal weight levels, you don’t need to be skinny to see improvements. This 8 percent reduction in BMI is achievable, and BMI is easy for primary care physicians to track.”

The weight loss program, explained Dr. Berkowitz, used family-based lifestyle modification. Clinicians taught the teens and adults about healthful eating habits and encouraged them to increase physical activities, such as walking, climbing stairs, and engaging in sports. Some of the teens used portion-controlled, prepackaged foods, while others ate regular food but followed a calorie-restricted, nutritionally-balanced diet.

The teens attended weekly group counseling meetings, separately from the parents, who had their own weekly group meetings. Group leaders encouraged parents to model healthful behavior for their children, and to support the teens’ behavior changes. “This study reinforces the importance of behavior change — adopting healthy eating habits and getting more physical activity — in achieving weight loss in adolescents,” added Dr. Berkowitz, the director of CHOP’s Weight and Eating Disorders Research Program.

Because this study was relatively small, future research in larger numbers of patients is needed to reveal longer-term results and to investigate further effects on adolescent metabolism and health, the authors noted. “These are preliminary results,” said Dr. Katz, “but this is the first study to identify a threshold effect for these improvements, and later studies can build on this one. Having more evidence about reasonable weight loss goals will help researchers develop more effective programs for teenage patients. ”

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Celebrating the Birth of CHOP’s 1,000th Fetal Surgery Patient


Children’s Hospital is proud to announce the birth of its 1,000th fetal surgery patient. Audrey Rose Oberio was born May 28 to Jackie and Gideon Oberio. The Oberios traveled to CHOP from Maryland so Audrey could be treated for myelomeningocele, the most severe form of spina bifida, at the Hospital’s Center for Fetal Diagnosis and Treatment. When she was born, Audrey Rose weighed 5 pounds, 8 ounces, and had only a scar where her doctors had operated.

Led by N. Scott Adzick, MD, the Center for Fetal Diagnosis and Treatment is the largest and most comprehensive fetal program in the world, with internationally renowned specialists treating the full range of fetal anomalies. In the form of spina bifida for which Audrey was treated, part of the developing spine fails to close properly.

With conventional postnatal surgery, myelomeningocele can lead to lifelong disabilities, including paralysis, bladder and bowel problems, and cognitive impairments. But in 2011, Dr. Adzick and his team published the results of more than two decades of research in the New England Journal of Medicine that showed fetal surgery can significantly improve the outcomes for children diagnosed in utero with spina bifida.

Their study, the Management of Myelomeningocele Study (MOMS) trial, demonstrated that two and a half years after fetal surgery children with spina bifida were better able to walk when compared to children who received surgery shortly after birth, and patients who received fetal surgery scored better on tests of motor function.

“It’s very gratifying to take this idea forward over 30 years, starting with a concept and now offering hope — to families, mothers, and the children themselves,” said Dr. Adzick, whose work was profiled in the 2011 Research Annual Report.

See below for a movie about the Oberio family’s story. learn more about CHOP’S groundbreaking fetal surgery and fetal care, visit the Center for Fetal Diagnosis and Treatment website.

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Examining the “Sense and Nonsense” of Alternative Therapies


Paul Offit, MD, chief of Children’s Hospital’s Division of Infectious Diseases, has long been a magnet for controversy. A co-creator of the rotavirus vaccine Rotateq, Dr. Offit’s willingness to speak his mind, question popular (and at times ill-informed) wisdom, and to defend science against its detractors has earned him many vocal critics over the years. For example, he has received hate mail and even death threats for working to debunk the now discredited connection between vaccines and autism.

In a move sure to earn him some new critics while reinforcing his reputation for outspoken honesty, Dr. Offit recently published a new book, Do You Believe in Magic?: The Sense and Nonsense of Alternative Medicine. Some of Dr. Offit’s previous books include Deadly Choices, about the anti-vaccine movement, and Autism’s False Prophets. In his latest book, Dr. Offit, who also directs CHOP’s Vaccine Education Center, examines the science and science fiction behind megavitamins, supplements, and alternative treatments like coffee enemas and laetrile.

Released Tuesday, Dr. Offit’s book has already received a great deal of attention from the media. USA Today, NBC News, and CBS This Morning have all featured stories about the book. Dr. Offit also published an opinion piece on CNN recently, and discussed his work on-air with the network’s chief medical correspondent, Sanjay Gupta, MD.

In the book, Dr. Offit acknowledges that people often turn to alternative treatments after becoming disillusioned with conventional medicine. However, while “conventional therapies can be disappointing, alternative therapies shouldn’t be given a free pass,” Dr. Offit writes.

This is because alternative medicine, as Dr. Offit points out, “can be quite harmful. Chiropractic manipulations have torn arteries, causing permanent paralysis; acupuncture needles have caused serious viral infections or ended up in lungs, livers, or hearts; dietary supplements have caused bleeding, psychosis, liver dysfunction, heart arrhythmias, seizures, and brain swelling; and some megavitamins have been found to actually increase the risk of cancer.”

And though many people think “excess vitamins can’t do any harm,” it turns out that “large quantities of supplemental vitamins can be quite harmful indeed,” Dr. Offit writes in The New York Times. In his recent op-ed, “Don’t Take Your Vitamins,” he notes excess amounts of vitamin E have been shown to increase the risk of heart failure and prostate cancer.

However, because megavitamins and other supplements are not regulated by the Food and Drug Administration, “consumers don’t know that taking megavitamins could increase their risk of cancer and heart disease and shorten their lives,” Dr. Offit writes. All therapies should therefore “be held to the same high standard of proof,” he notes in Do You Believe in Magic?

“Because the truth is, there’s no such thing as conventional or alternative or complementary or integrative of holistic medicine. There’s only medicine that works and medicine that doesn’t,” he says.

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Children’s Hospital Researcher Named 2013 Pew Scholar


By being named a 2013 Pew Scholar, Children’s Hospital investigator Claudio Giraudo, PhD, joins a “prestigious community of Nobel laureates, MacArthur fellows, Albert Lasker Basic Medical Research Award winners, and hundred of other pioneers who earned Pew grants at the start of their careers.” The Pew Scholars are each awarded $240,000 over four years to conduct their research, and Dr. Giraudo is one of just 22 researchers to be selected out of 134 nominated.

The Pew Scholars Program in the Biomedical Sciences “identifies and invests in talented researchers in medicine or biomedical sciences,” enabling “promising scientists to take calculated risks and follow unanticipated leads to advance human health,” according to the Pew website. Since 1985, Pew has awarded more than 500 investigators over $130 million in research funding.

After receiving his doctorate in cell biology and biochemistry from the National University of Cordoba, Argentina in 2002, Dr. Giraudo went on to do postdoctoral research at Memorial Sloan-Kettering Cancer Center, Columbia University and Yale University. In 2012 he joined the faculty of Children’s Hospital and the University of Pennsylvania as an assistant professor of pathology and laboratory medicine.

Dr. Giraudo’s lab studies “intracellular membrane trafficking and calcium-regulated exocytosis in eukaryotic cells,” researching how immune cells secrete granules that destroy infected cells. With the support of the Pew Scholar grant, Dr. Giraudo hopes “to identify the protein machinery cytotoxic T lymphocytes (CTLs) use to secrete granules that break down cells.” A type of white blood cell, CTLs target and kill other cells, including cancer cells and cells that have been infected with viruses.

“This award will give me the chance to study what happens at the cell membrane during the body’s immune response, which could be therapeutically targeted to improve prognosis and quality of life of patients,” Dr. Giraudo said.

In addition to better understanding cell-mediated killing, the Pew-supported research project could also shed light on how similar processes lead to disease, such as in diabetes and brain disorders, he added.

“The Pew Scholars Program gives innovative scientists both the freedom to take calculated risks and the resources to pursue the most promising, but untried, avenues for scientific breakthroughs,” said Pew CEO and president Rebecca W. Rimel.

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Center for Injury Research & Prevention Awarded for Teen Driving Initiative


Car crashes are the leading cause of death for teens in the United States, with teen drivers ages 16 to 19 three times more likely than drivers ages 20 and older to be in a fatal crash per mile driven. For several years, the Teen Driver Safety Research team at the Center for Injury Research & Prevention (CIRP) has strived to reduce the frequency and severity of teens’ motor vehicle crashes, injuries, and fatalities.

The team’s efforts have received numerous accolades and acknowledgements, with the most recent from the New Jersey Teen Safe Driving Coalition, which awarded CIRP the 2013 Graduated Driver License (GDL) Champion Award.

The annual GDL award program recognizes those who have advocated for New Jersey’s three-step, novice driver licensing system. Designed to ensure teens survive their most dangerous driving years, GDL helps them gain experience and build the skills necessary to become competent, skilled drivers for life.

CIRP’s support of enhancements to the state’s GDL program, coupled with guidance in developing and evaluating New Jersey’s “Share the Keys” program and research assessing the impact of the nation’s first GDL decal requirement merit this recognition.

Over the past five years, the CIRP team has supported and assisted New Jersey teen driver safety advocates working to not only strengthen the state’s GDL program, but also to engage parents and law enforcement officers in understanding and enforcing its evidence-based provisions.

CIRP’s specific teen driver-related work has focused on testifying at committee hearings and meeting with legislators to advocate for bills calling for stricter passenger and nighttime driving provisions and a longer permit phase that includes increased parent-supervised practice driving hours and parent education.

Significantly, CIRP developed rigorous methods to evaluate the effect of New Jersey’s decal program on enforcement of GDL provisions and on probationary driver crash rates. In a study published in the American Journal of Preventive Medicine, they reported that in the first year of implementation GDL-related citations issued to probationary drivers increased 14 percent and the rate of police-reported crashes among the same group declined 9 percent.

“CIRP’s interest in evaluating the nation’s first GDL decal has been important not just to New Jersey, but also to other states considering similar public policy,” said CIRP co-scientific Director Flaura K Winston, MD, PhD. “Additionally, despite other countries requiring teen drivers to display decals for many decades, this is the first scientific look at the effect of decals on crash rates.”

CIRP staff also regularly provide guidance to the New Jersey Division of Highway Traffic Safety and other groups to develop research-based, data-driven programs that includes clear behavioral objectives that are helping parents recognize the critical role they play in helping their teens navigate their most dangerous driving years.

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Designing a More Accessible, Easier to Install Safety Seat


According to the National Highway Transportation Safety Administration (NHTSA), a properly installed child safety seat (CSS) shouldn’t be able to move more than an inch from front to back or from side to side. But, as anyone who has ever installed a CSS knows, this can be much more difficult to achieve than it sounds.

In addition to the physical demands of installing a CSS, many are accompanied by byzantine, labyrinthine instruction manuals that can be more confusing than helpful. Indeed, a NHTSA 2004 survey found that a shocking 72.6 percent of car safety seats are installed incorrectly.

With the overall goal of making “safety seats that are easier to install accurately and securely,” the Center for Injury Research and Prevention at The Children’s Hospital of Philadelphia (CHOP)’s Jessica H. Mirman, PhD, recently partnered with Minnesota Health-Solutions (MHS), a Saint Paul, MN-based small business, to conduct a study of innovative car safety seat designs with funding from the Centers for Disease Control and Prevention Small Business Innovation Research (SBIR) program.

With the ultimate goal of the project to bring a product to market, MHS partnered with Britax, a leading CSS company, to develop a new “ClickTight Installation System.” According to the Britax website, the system is “a series of components engineered to simplify the process of securing” a car seat to a car. “Installing a seat that features ClickTight alleviates both the confusion and stress that may come along with installing your child’s car seat,” the company claims. The new installation system will be incorporated in several Britax products now in development.

Using a prototype of an improved seat, CHOP researchers conducted human subject testing to determine if caregivers were able to achieve more secure and accurate installations. According to Dr. Mirman, though most car seats are installed incorrectly, parents won’t seek help if they think they installed them correctly. Additionally, car seat checkpoints are underutilized, due to the overestimation of correct car seat installation by caregivers.

An applied developmental psychologist, Dr. Mirman first joined CHOP in 2007 after receiving her doctorate from Fordham University. In addition to her child passenger safety research, Dr. Mirman also investigates teen driver safety.

To learn more about car safety seat guidelines, visit the CHOP website. Click HERE for a list of participating car seat check centers in and around Philadelphia.

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Chief Scientific Officer Highlights CHOP Research Institute Achievements


The Children’s Hospital of Philadelphia Research Institute recently announced its position as the top-ranked independent pediatric hospital in the United States in funding from the National Institutes of Health. CHOP received more than $125 million in NIH funds in FY2012, placing it not only at the top of the list of pediatric hospitals but also fourth among all independent hospitals in the country.

While the #1 ranking is impressive and something to celebrate, achieving this milestone involved far more than receiving more NIH grant dollars than any other pediatric hospital. During the annual Scientific Symposium, Chief Scientific Officer Phil Johnson, MD, gave a “State of the Research Institute” address where he discussed the factors that brought the Institute to this prestigious position.

Those factors underscored the challenges inherent in the shifting landscape of research funding over the last decade. But they also highlighted the challenges that lie ahead, and the unique strengths and programs of the CHOP Research Institute that set it apart.

Despite decreasing federal dollars supporting biomedical research, the majority of the Institute’s funding comes from the NIH. Dr. Johnson explained that the Institute’s research expenditures have more than doubled over the past 10 years, an astounding growth that parallels the Hospital’s growth.

The number of CHOP investigators has surged from approximately 300 to more than 450 during this time. The number of grant applications, awards, and proposals submitted to the Hospital’s Institutional Review Board — which reviews proposals for clinical research — have all experienced enormous growth as well, Dr. Johnson said.

The influence of this increased activity is evident not only in the increasing number of investigators and grant awards, Dr. Johnson noted, but also in the impact their research has on the collective scientific knowledge.

“Over the past five years, the Research Institute more than doubled the number of publications in peer-reviewed journals,” he said. “But more importantly, our investigators are publishing more and more in the most prestigious, highest-ranking journals in the world. Our contribution to the understanding of diseases and innovative ways to treat them is nothing short of astounding.”

To highlight this, Dr. Johnson shared the amazing story of Emily Whitehead, who suffered from an aggressive form of acute lymphoblastic leukemia that relapsed after she received conventional chemotherapy. Faced with few viable treatment options, oncologist Stephan A. Grupp, MD, PhD, tried an experimental therapy using bioengineered T cells that were custom-designed to multiply rapidly and destroy leukemia cells.

Dr. Grupp achieved a complete response in Emily by using an innovative treatment approach that involved reprogramming her own immune cells to attack her cancer. More than a year after her treatment, Emily remains cancer-free.

The innovative research program at CHOP means the promise of potential new therapies for children is tremendous. However, challenges like decreased federal funding for research and healthcare reform may slow the pace of the discovery. And Dr. Johnson expressed concern that these factors may discourage the next generation of scientists.

The Research Institute, however, is uniquely poised to face these challenges, Dr. Johnson said. Programs like the CHOP Research Institute Summer Scholars Program (CRISSP) provides full-time mentored research experience to future leaders in the biomedical sciences, with a special emphasis on advancing laboratory, clinical, behavioral, and translational pediatric research.

“This program brings undergraduates into the Research Institute and shows them how great science is,” Dr. Johnson said. He also discussed the new CHOP Pediatric Translational Research Workshop for Basic Scientists, a program that introduces scientists to the principles and fundamental elements of pediatric translational research.

“These programs are the way we will rebuild the future generation of scientists,” Dr. Johnson said. “The strength of our research program and unique programs bolsters my confidence that we will weather the storm.”

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GlaxoSmithKline Launches Program to Partner with PIs in Early Drug Discovery


GlaxoSmithKline (GSK) has announced an innovative competitive program aimed at translating novel science into new medicines. The Discovery Fast Track Competition is an opportunity for investigators at academic institutions to submit innovative concepts, discoveries, and research to GSK that may lead to a partnership in early drug discovery.

An expert panel of judges at GSK will evaluate each submission, selecting up to 10 winners. Winning investigators will collaborate with a team of GSK scientists to put their innovative research concept on an accelerated drug discovery path.

GSK will provide state-of-the-art capabilities to scale biological reagents and develop assays to support high-throughput screening of millions of compounds against a specified target or pathway. The company’s scientific expertise in data analysis and screening triage, combined with its capabilities, enable accelerated identification of high-quality chemical probes.

GSK and the winners will work together to interpret the output to identify chemical probes that could further the research field and lead to high-impact publications.

PLEASE NOTE: Principal investigators interested in possibly participating in the GSK program should coordinate their efforts with Ellen Purpus in the Office of Technology Transfer to ensure the protection of intellectual property through patent filings, if required. GSK will not be under any obligation for confidentiality or non-use of your proposals.

Finalists will be notified early August 2013. Non-finalist applicants will be notified by email shortly thereafter. Winners will be selected in late October 2013.

For more information about Discovery Fast Track Competition, including what GSK is looking for and how intellectual property will be protected, visit the GSK website.

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Produced by The Children’s Hospital of Philadelphia Research Institute.

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