Apr 04 2014

Advocates Shaky That NIH Funding Remains Steady

NIH fundingAdvocates for biomedical research expressed their disappointment in President Barack Obama’s proposed budget for fiscal year 2015, which includes a slight rise in funding for the National Institutes of Health. This modest boost is expected to support 9,326 new and competing grants, 329 more than this year.

The FY15 budget in­cludes $30.2 billion for NIH to back research at institutions across the U.S. NIH Director Francis Collins, MD, PhD, told the Wall Street Journal that his agency is “grateful that in a tough budget year, the NIH was able to get an increase.” However, he noted that NIH is about $500 million behind where it was in the Obama administration’s proposed fiscal-year 2012 budget.

The research community had hoped that the 2015 budget would help to make up for lost ground following the devastating sequestration last year, which resulted in a 5 percent cut to NIH’s budget. Instead, they continue to worry that keeping funding levels at status quo will not sustain the fast pace of scientific innovation. Several research advocacy groups are calling on the administration and Congress to make a significant investment in NIH funding during their budget negotiations.

“The United States once stood firmly at the forefront of the research revolution, but after a decade of budgets that have not kept pace with inflation and last year’s across-the-board sequestration cuts, NIH has seen a more than 20 percent decline in its purchasing power and can only fund one in every seven research grants it receives,” wrote the United for Medical Research in a statement. “As such, the U.S. is slipping in its position as the global leader in the life sciences.”

The $30.2 billion proposed in the budget, adjusted for inflation, would be $100 million lower than the 2002 funding level for the NIH. Mary Woolley, president and CEO of Research!America, reiterated that insufficient funding levels for federal research and health agencies jeopardize scientific discovery and its capacity to improve health outcomes and stem the explosion of chronic diseases.

“We simply cannot sustain our nation’s research ecosystem, combat costly and deadly diseases like Alzheimer’s and cancer, and create quality jobs with anemic funding levels that threaten the health and prosperity of Americans,” Woolley wrote in a statement.

In his budget message, President Obama emphasized the importance of biomedical research and proposed an Opportunity, Growth, and Security Initiative that would provide an additional $970 million for NIH, which would support about 650 additional new grants. But an analysis of federal policy reported in Inside Higher Ed described this potential windfall as “almost fanciful” because the new initiative would depend on Congress reaching consensus on major changes in the tax code.

While The Science Coalition, a nonprofit, nonpartisan organization of the nation’s leading public and private research universities, appreciated the signs of President Obama’s “commitment to world-class science and research,” it remains wary about the long-term funding trends.

“The result is that we now face an innovation deficit — the gap between needed and actual federal investments in research and higher education,” stated Jon Pyatt, 2014 president of The Science Coalition. “We urge Congress to make investments in research and education a national priority. Only by doing so can we close the innovation deficit and truly put our nation back on a track for long-term economic health, well-being, and security.”

Phil

Philip R. Johnson, MD
Director, The Children’s Hospital of Philadelphia Research Institute
Chief Scientific Officer and Executive Vice President

Permanent link to this article: http://www.research.chop.edu/blog/advocates-shaky-nih-funding-remains-steady/

Apr 02 2014

1 in 68: An Interview with CHOP Experts on the CDC’s New Autism Rate

autismThe Centers for Disease Control and Prevention (CDC) recently announced a new prevalence estimate for Autism Spectrum Disorder: 1 in 68 individuals. This is 29 percent higher than the estimate released in 2012 (1 in 88), 64 percent higher than the estimate from 2009 (1 in 110), and a 124 percent increase from the report from 2007 (1 in 150).

Philip R. Johnson, MD, director of The Children’s Hospital of Philadelphia Research Institute, recently interviewed Center for Autism Research experts to get their take on the new CDC numbers.

 

Dr. Johnson: What could account for such a dramatic rise from the initial reports?

Judith Miller, PhD: There are a few reasons that we can hypothesize that can account for this rise. For one, with each surveillance study, the participating sites get better and better at gathering records from their clinical and educational sources. Secondly, autism spectrum disorder (ASD) awareness has increased dramatically over the last 15 years, so with each year increasing numbers of parents, schools, and clinicians are considering whether a child’s difficulties might be due to autism. Third, the true prevalence may be rising due to real changes in the factors that cause autism.


Dr. Johnson: How are these studies conducted? 

Susan E. Levy, MD, MPH: These studies rely on record reviews rather than in-person evaluations. Records of any child who meets the age criteria, who lives in the specified geographic region, and who has come to the attention of a healthcare or special education agency are included. An anonymous file is compiled of that child’s comprehensive health and education evaluations, which is then reviewed by clinicians using a standard rating procedure.

The studies looking at prevalence of developmental disorders such as ASD are carried out in multiple locations in the United States, funded by U.S. Department of Health and Human Services through the CDC. Sites are awarded the opportunity to participate based on their ability to access the necessary records and conduct the necessary work, and each site works closely with their local healthcare and education agencies to collect the data.

 

Dr. Johnson: Do these numbers seem to fit with clinicians’ and researchers’ sense of the prevalence?

Dr. Miller: It’s always better to have data than rely on anyone’s impression. I think this is the record review method is a probably the best one possible. However, record review will always have limitations, including the lack of gold standard clinical evaluations. The best approach would be to do a real epidemiological study, basically door to door with expert clinical evaluations but that is so expensive as to probably be impractical in this funding environment.

The CDC, however, did do a small study with in-person assessments, and it showed good correlation to the record-based study. But that was many years ago. I am hopeful the CDC can receive funds needed to continue doing validation studies. With increased awareness in the community, and recent changes to diagnostic criteria it’s important to understand how the diagnosis is being made in the community.

Marsha Gerdes, PhD: It’s probably most important to take a step back from the numbers.  Even if the diagnosis is not 100 percent accurate in all cases, these are children who are coming to the attention of their schools, parents, or healthcare providers. They are struggling in some way with social communication and behaviors. We as a community need to come together around how best to meet their individual needs and to support their families and educators.

Robert Schultz, PhD:  Given the CDC’s methods, there will clearly be errors of diagnosis, including misdiagnosing some children who really don’t have ASD. However, that does not mean that all or nearly all of the children identified don’t have a condition that causes them to struggle much more than a typical child.

It is also important to recognize that diagnostic categories such as ASD mask considerable heterogeneity. It is often said, “If you have seen one child with autism, you have seen one child.”  More recent research is showing that ASD presents on a continuum with typical development, but for certain skills and traits, just on the lower end of some continuous dimension. The point here is that there might not be sharp boundaries and a real category of autism.

The World Health Organization (WHO) and other organizations will report the “true” prevalence of brain based mental disorders is about 20 percent, and these often start at birth or prenatally. I would say 1.5 percent of children who present with ASD on record review very likely have something. The “essence” of what they have however is proving a bit more elusive than we use to understand, and many times cases do not fit neatly in current diagnostic groups. But that does not mean that these kids are not struggling and in need of interventions.

Dr. Gerdes: Let’s put the 1.5 percent in the context of other developmental disorders. The CDC’s report, “Trends in the prevalence of Developmental Disabilities in U.S. children, 1997-2008,” reported learning disabilities at 7.7 percent and ADHD at 6.7 percent, an increase of 33 percent. Many of the same factors of increasing awareness and accessing services may have played a role in increases of other developmental disabilities.

 

Dr. Johnson: OK, I get all that. But then, how does one define “normal?” If 20 percent of people have brain based mental disorders, then it’s no longer “atypical.”

Dr. Schultz: Yes, it is a value judgment as to where you draw the line and say this kind of problem necessitates special attention. The values most commonly invoked are economic and quality of life. The WHO discusses the impact of mental disorders in terms of both dollars and DALYS (disability-adjusted life years). Where you draw the line affects who gets treatment and ultimately can save both dollars and DALYS.

 

Dr. Johnson: Sounds reasonable. But still. Let’s say 1 in 68 children have “something.” Did they always have something?

Dr. Miller: At the Center for Autism Research, we see more and more children being referred for mild forms of autism (the group that saw the largest increase in the CDC study). We find that 20-25 percent of children who have been diagnosed with mild forms of ASD in the community don’t meet our gold standard criteria for an autism spectrum disorder. These children are struggling, but their clinical presentation is complex, and doesn’t always fit neatly under ASD.

Dr. Gerdes: Services funded by the community (e.g., Early Intervention, Special Education, Mental Health, etc.) are often, but not always, tied to both a diagnosis and a level of impairment.  This can make it difficult to help those individuals with milder symptoms or a less common presentation of symptoms. Obtaining services for those children and adults can be challenging.

Dr. Schultz: The field has really begun to move away from studying categories of mental disorders, as they don’t map onto to the brain or to the genome in a clean fashion. Tom Insel, MD, Director of the National Institute of Mental Health, has lead the way here, and is pushing for grants to propose studies that cut across diagnostic boundaries, that focus on processes rather than diagnostic categories (e.g., processes such as sociability — which can be linked to reward circuits in the brain, or executive functioning — which can be linked to functions of the frontal lobes). Deficient executive functioning and sociability are features shared by many different disorders. Ultimately profiles of functioning across these and other dimensions will likely prove both more accurate and more helpful in guiding treatment.


Dr. Johnson: Given the efforts to encourage early screening, were you surprised that the average age of diagnosis remained the same?

Dr. Levy: Not at all. The AAP’s recommendations for early screening came out in 2007. The CDC’s data is on children born in 2002 (turned 8 years old in 2010). Pediatricians have been working hard on early identification, but still confront barriers of the “perfect” tool for a busy primary care office and access to appropriate resources for treatment in the community. But early identification (by age alone) is not the only hurdle. Access to care by underserved groups of children is still a major problem. White children were more likely to be identified with autism than black or Hispanic children. High-risk groups such as former premature infants, children with acquired brain injury, genetic disorders and others need to be identified and screened or evaluated more consistently. Environmental exposures and factors need to be investigated carefully, in the context of well-designed studies as potential causes of ASD.

That said, 1 in 68 children probably do have something that deserves our clinical and research attention. Thus, the CDC work always cuts both ways — it is good to have the public’s attention on the welfare of these kids. We agree that more attention can be focused on earlier identification, and also on appropriate evidence based services that can reduce symptoms and the impact of symptoms on functioning at home and school.

To learn more about autism spectrum disorders and ASD research at The Children’s Hospital of Philadelphia, see the Center for Autism Research website.

Permanent link to this article: http://www.research.chop.edu/blog/1-68-interview-chop-experts-cdcs-new-autism-rate/

Apr 02 2014

Prominent Gene Therapy Expert Joins CHOP

gene therapyBeverly L. Davidson, PhD, a nationally prominent expert in gene therapy, joined The Children’s Hospital of Philadelphia on April 1. A member of the Hospital’s Department of Pathology and Laboratory Medicine, Dr. Davidson will serve as the new director of the Center for Cellular and Molecular Therapeutics.

Prior to coming to CHOP, Dr. Davidson — who investigates gene therapy for neurodegenerative diseases — was associate director of the Center for Gene Therapy at the University of Iowa. Dr. Davidson “will greatly enhance our abilities to translate important biological discoveries into pioneering treatments for deadly diseases,” said Robert W. Doms, MD, PhD, CHOP’s pathologist-in-chief.

Dr. Davidson has also assumed the role of director of the Center for Cellular and Molecular Therapeutics (CCMT), succeeding the Center’s inaugural director, Katherine A. High, MD. The CCMT is “dedicated to the understanding, development, and application of gene and related cell and nucleic acid therapies and the promotion of professional public education,” according to its website.

In addition to her roles at CHOP Dr. Davidson is a member of the Scientific Advisory Board of the Hereditary Disease Foundation, and is a scientific co-founder and advisor at the gene therapy company Spark Therapeutics, which launched in late 2013 with a $50 million capital commitment from CHOP.

Research Interests

 Dr. Davidson’s research has been concentrated on inherited genetic diseases that attack the central nervous system, with a particular focus on childhood-onset neurodegenerative diseases such as Batten disease and similar disorders.  According to the NIH, Batten disease and related disorders affect “an estimated 2 to 4 of every 100,000 live births in the United States.”

In these disorders, the lack of an enzyme impairs lysosomes, proteins that perform crucial roles in removing unwanted by-products of cellular metabolism. Toxic waste products then accumulate in the brain and cause progressively severe brain damage. Dr. Davidson has developed novel methods to deliver therapeutic genes to the central nervous system, and her team has succeeded in reversing neurological deficits in animal models of disease, and is working to advance this approach in humans.

She has also studied other inherited neurological diseases such as Huntington’s disease and spino-cerebellar ataxia. In these studies, she has delivered forms of RNA to the brains of animals to silence the activity of disease-causing genes. Although much of Dr. Davidson’s work has centered on delivering beneficial genes to the central nervous system, the viral vectors that she has developed are applicable to other organs and tissues — for example, in gene therapy directed to the lungs or the liver.

For more information about Dr. Davidson and her research, see the Hospital’s press release about her appointment. To learn more about the pioneering gene therapy research being performed at Children’s Hospital, see the CCMT site.

 

 

 

Permanent link to this article: http://www.research.chop.edu/blog/prominent-gene-therapy-expert-joins-chop/

Mar 31 2014

Debaters Weigh in on Mandatory Vaccination

VaccinationVaccines are a routine part of healthcare for most Americans, but some parents remain passionately opposed to childhood immunizations, despite reassurance from public health experts about their safety and value.

In the opinion pages of The New York Times, editors posed this question to a panel of experts including Kristen A. Feemster, MD, MPH, MSHPR, a pediatric infectious diseases physician and director of research at the Children’s Hospital of Philadelphia’s Vaccine Education Center: “Should parents no longer be allowed to get religious or philosophical exemptions from having their children immunized?”

The newspaper published the debate following a rare outbreak of measles in New York City that began in February.

All 50 states have school immunization requirements, but most grant religious exemptions, and 19 states allow philosophical exemptions for those who object to immunizations because of personal, moral, or other beliefs. Dr. Feemster supports the curtailment of such exemptions because some people, whether because of age or compromised immune systems, cannot receive vaccines.

“They depend on those around them to be protected,” Dr. Feemster wrote. “Vaccines aren’t the only situation in which we are asked to care about our neighbors. Following traffic laws, drug tests at work, paying taxes — these may go against our beliefs and make us bristle, but we ascribe to them because without this shared responsibility, civil society doesn’t work.”

Dr. Feemster emphasized that vaccines are safe and effective, but she recognized that a “vaccine confidence gap” persists that must be addressed consistently by the scientific and public health community. The Vaccine Education Center has multiple resources, including a vaccine mobile app, for parents and healthcare providers to develop a dialogue and stay up-to-date on vaccine news, research, and developments.

Permanent link to this article: http://www.research.chop.edu/blog/debaters-weigh-mandatory-vaccination/

Mar 28 2014

CHOP Business, Research Innovations Honored at PA Bio Dinner

research

Left to right, Joseph Reagan, Wexford Science & Technology; Spark president and CEO Jeffrey Marrazzo; Dr. Johnson; and Christopher Molineaux, president and CEO of Pennsylvania Bio.

At the recent 2014 Pennsylvania Bio Annual Dinner & Awards Celebration, The Children’s Hospital of Philadelphia’s reputation as an international leader in pediatric medicine was further cemented, as the Hospital was honored with two awards celebrating its business and research innovations. CHOP was handed the “Deal of the Year” award for its successful spinout of gene company Spark Therapeutics, while the University of Pennsylvania and CHOP were jointly given the “Patient Impact Award” for their groundbreaking immune therapy research.

An organization devoted to ensuring Pennsylvania “is the global leader in the biosciences by creating a cohesive community,” Pennsylvania Bio represents the “entire life sciences industry” in Pennsylvania. Each year the organization holds a dinner and awards ceremony to celebrate the previous year’s successes.

Based in part on the innovative work of Children’s Hospital’s Katherine A. High, MD, gene therapy company Spark Therapeutics was launched in October 2013 with a $50 million capital commitment from CHOP. According to the company’s website, Spark has “established human proof of concept in two organ systems — the eye and the liver — and are advancing a Phase 3 program in blindness caused by mutations of the RPE65 gene; a Phase 1/2 program in hemophilia B; and preclinical programs in neurodegenerative diseases and other hematologic disorders and forms of inherited blindness.”

The PA Bio “Deal of the Year” award is intended for “a company that has promoted the growth of Pennsylvania’s bioscience industry by way of a substantial deal or strategic partnership,” closed in 2013. Other nominees for the 2013 award were Invisible Sentinel and pharmaceutical company Tetralogic.

resea

Stephen A. Grupp, MD, PhD speaking at the Pennsylvania Bio Annual Dinner.

CHOP’s second award of the night, the “Patient Impact Award,” is for “a company or organization that has made a significant contribution to the quality of healthcare or length of life of patients in 2013,” according to the PA Bio web site. The Children’s Hospital of Philadelphia and the University of Pennsylvania were honored for their joint immune therapy research. In addition to CHOP/Penn, other nominees included medical device manufacturer Actuated Medical and Bethlehem, Pa.-based Orasure Technologies, which manufactures diagnostic and oral fluid specimen collection devices.

Led at CHOP by Stephan A. Grupp, MD, PhD, and at Penn by Carl H. June, MD, the CHOP/Penn immune therapy partnership has been investigating using modified versions of patients’ own immune cells to attack — and destroy — tumors. Last year the partnership led to dramatic, extraordinary results: two children with acute lymphoblastic leukemia, the most common form of childhood leukemia, achieved a complete response after being treated with immune therapy. Since receiving the treatment one of those patients remains healthy and cancer-free.

“The Children’s Hospital of Philadelphia is thrilled to be honored with these two awards,” said Philip R. Johnson, MD, chief scientific officer of The Children’s Hospital of Philadelphia Research Institute. “They are a testament to the hard work of our investigators and staff, who work every day to improve the health of children.”

To learn more about the innovative research being performed at CHOP every day, see the CHOP Research site or the 2013 Annual Report.

Permanent link to this article: http://www.research.chop.edu/blog/chop-business-research-innovations-honored-pa-bio-dinner/

Mar 26 2014

CHOP Pediatric Psychology Experts to Shine at SPPAC

Pediatric psychologyPediatric psychology is embedded into family-centered care at The Children’s Hospital of Philadelphia. When a child has a serious or chronic illness, pediatric psychologists listen carefully and empathetically to concerns of the young patient and family while assessing their strengths, coping strategies, and goals. Pediatric psychologists also work to improve the welfare of well children in healthcare settings, and they conduct a variety of research activities, such as finding ways to increase adherence to medical treatments.

Several of CHOP’s experts in this multidisciplinary field will be sharing their insights at the Society of Pediatric Psychology Annual Conference (SPPAC) being held in Philadelphia March 27-29.

The SPPAC will introduce a fresh format for the meeting, which will attract pediatric psychologists, researchers, and other specialists who have training and experience in addressing the emotional, cognitive, and developmental needs of medically fragile children. SPPAC Chairperson Lisa Schwartz, PhD, a psychologist in CHOP’s division of oncology, has organized many of the new elements that will be integrated into the 2014 conference.

“It’s been exciting to help shape the conference and be a firsthand witness to all the innovation in the field,” said Dr. Schwartz, who will officially welcome SPPAC attendees during opening remarks March 28.

A “first” for the conference is the incorporation of a theme, “Pediatric Psychology: From Infancy to Adulthood.” Presentations will reflect a lifespan and developmental perspective that focuses on findings from early childhood to young adulthood. They also will promote the maturation of the science of pediatric psychology via technology, advanced statistical or research designs, translational science, and team science.

The conference will offer more workshops and concurrent symposia than ever before along with research blitz sessions that will cover novel and noteworthy advances, Dr. Schwartz said. Fifteen special interest group meetings will facilitate collaboration.

Dr. Schwartz is eager to hear the keynote presentation, “An Integrated Framework for Linking Exposure and Phenotypic Data in the National Children’s Study,” by Captain Steven Hirschfeld, MD, PhD, director of the National Children’s Health Study. By following 100,000 children from before birth to age 21, study researchers hope to better understand how children’s genes and their environments interact to affect their health and development. The Children’s Hospital of Philadelphia is a site of this important study.

CHOP psychologists will present talks throughout the conference, including one that Dr. Schwartz will give March 28 on young adults’ transition to adult medical care. Dr. Schwartz highlighted a few other “not-to-miss” examples of CHOP’s contribution:

Jodi Mindell, PhD, associate director of the Sleep Center at CHOP, will be a speaker at one of the six pre-conference workshops March 27. During “Pediatric Behavioral Sleep Medicine: Tips and Tools for Practitioners,” attendees will learn about intervention strategies for a variety of sleep difficulties in pediatric patients, including insomnia, circadian rhythm disturbances, childhood fears, and non-adherence to positive airway pressure therapy.

Paul Robins, PhD, program director of pediatric psychology at CHOP, will participate March 28 in a panel discussion, “The Maturation of Pediatric Psychology Training: 2013 Society of Pediatric Psychology Task Force Recommendations.” He also is part of a symposium March 29 and will speak about, “Translating Competencies Into Practice: Internship Training in Clinical Child Psychology.”

Meghan Marsac, PhD, a CHOP clinical psychologist and behavioral researcher, is chairing a symposium, “Utilizing Web-based Programs to Promote Child Health,” March 29. She also will give a talk on the application of an internet-based game for secondary prevention of post-traumatic stress following acute medical events. Nancy Kassam-Adams, PhD, co-director of CHOP’s Center for Pediatric Traumatic Stress, will be the discussant.

SPPAC provides an ideal forum for networking and career building. Students, trainees, and early career professionals who pre-register for a mentoring lunch March 28 will have the opportunity to consult with more than 25 established investigators and clinicians from CHOP who can help them to navigate research careers in pediatric psychology.

The three-day conference will take place at the Loews Hotel in Center City Philadelphia, and it is expected to draw more than 500 researchers and practitioners from the Delaware Valley and beyond.

Permanent link to this article: http://www.research.chop.edu/blog/chop-pediatric-psychology-experts-shine-sppac/

Mar 24 2014

CHOP Researcher Advances Adolescent Care in Africa

adolescents

Dr. Lowenthal examining an African child in Botswana.

A Children’s Hospital of Philadelphia pediatrician’s research projects span across continents to Botswana, a sub-Saharan African country with a busy clinic for 2,000 HIV-infected children and adolescents who stole her heart.

Elizabeth Lowenthal, MD, MSCE, remembers noticing a remarkable 12-year-old boy in Botswana, “a real leader” who spontaneously sat down and began translating for an American social worker who had recently arrived to organize a camp for patients. Smart and engaging, he did well on antiretroviral therapy until he became an older teenager. He struggled with his adherence, ended up failing his treatment, and became resistant to the medications.

Dr. Lowenthal brought this lesson to CHOP, along with many others that she learned while living for four years in Botswana, where she provided care as the clinical director of the Botswana-Baylor Children’s Clinical Centre of Excellence. She began practicing there in 2004, when the country was just getting antiretroviral treatment. Currently, more than 3 million children worldwide are affected by HIV, and more than 90 percent live in sub-Saharan Africa.

“Lots and lots of people in Botswana are infected, and just about everybody is affected one way or another by HIV,” Dr. Lowenthal said. “Botswana has better resources than most of the countries in sub-Saharan Africa, so figuring out what can work there hopefully can translate to where most of the kids are.”

As one of the only pediatricians at the time treating children with HIV in Botswana, Dr. Lowenthal became involved with government committees that were creating public health policies for the country. The committee members would turn to Dr. Lowenthal for recommendations for the pediatric population with HIV, but she found that there were little data in the literature to guide some of their decisions.

So while her heart was still in Botswana, she returned to the U.S. and began a research fellowship at CHOP to learn how to scientifically approach these questions. Dr. Lowenthal is now an assistant professor of pediatrics at CHOP, a staff physician at the Special Immunology Family Care Center, and lead research physician for CHOP Global Health.

In her first major research publication in the Journal of the American Medical Association, Dr. Lowenthal’s findings supported the use of efavirenz as initial antiretroviral treatment because it was associated with less virological failure when compared to another drug, nevirapine, which is used more commonly in children. The study influenced not only the treatment guidelines in resource-limited settings, such as Botswana, but it also helped to clarify U.S. guidelines.

adolescentsMost of her current work is focused on adherence to HIV treatments among perinatally HIV-infected adolescents in Africa, a fast-growing population. Good treatment outcomes have allowed more children with HIV to reached young adulthood, and now they face unique challenges. As Dr. Lowenthal described in a recent Lancet Infectious Diseases review paper, they must confront psychosocial issues, negotiate decisions about sexual relationships, and manage a chronic illness during a period of rapid physical and psychological changes. Unfortunately, this tumultuous time threatens the longevity of their treatment.

“While these treatments are lifesaving and amazing, they only work if you take them consistently,” Dr. Lowenthal said. “If they miss doses for a period of time, they develop resistance to them. Many adolescents, unfortunately, are losing the limited treatment options that they have. If we can’t support adherence well enough, all of these other problems are not going to be relevant because kids aren’t going to continue to survive.”

Her research projects take many perspectives to help predict which adolescents are going to have difficulty with adhering to their medication long-term. Recognizing those children who would most likely benefit from support services could be an important first step to possibly preventing their failure, Dr. Lowenthal said.

An ongoing study is comparing methods that measure adherence to determine which are the most useful among HIV-infected adolescents in Botswana. These methods include self-report, electronic monitoring devices, pill counts, medication refill rate, and monitoring virus suppression. Dr. Lowenthal’s research team also is considering how adolescents’ autonomy over medication is associated with these measurement methods.

In a pilot study published in AIDS Care, her research team evaluated the Pediatric Symptom Checklist (PSC) as a simple screening tool to identify children and adolescents with psychosocial needs. A prospective study is under way to assess whether high scores on the PSC precede adherence problems and treatment failure. Once these psychosocial areas are identified, healthcare providers could use them as starting points for interventions.

“In a setting where you have thousands of kids, trying to figure out what are the big issues that we need to approach is really important,” Dr. Lowenthal said. “They are certainly complex problems that need complex solutions. We are starting with just trying to understand what’s at the root.”

adolescentsDr. Lowenthal’s work in Botswana has crossover to her clinical work in the Special Immunology Family Care Center, where she and her team provide treatment for about 150 HIV-infected children. For example, artists at CHOP adapted a book created for children in Botswana to help initiate discussions about their HIV status. In the version for the American children, the good guys are superheroes instead of soldiers, and the kids play basketball instead of soccer, but the message is the same: Taking your antiretroviral medications keeps your body strong.

Dr. Lowenthal keeps in touch with some of her patients in Botswana, and her research allows her to visit two or three times a year. The amazing young man she met during her early years in Africa is now in his twenties and doing better again. Yet, she continues to ask: “What could I have done to have kept him from losing options and struggling during his teenage years?”

A CDC-PEPFAR Public Health Effectiveness Grant funds Dr. Lowenthal’s work, and additional questions related to optimizing adherence measurement and defining developmental and psychosocial factors associated with nonadherence are funded by an NIH K23 Career Development award.

Permanent link to this article: http://www.research.chop.edu/blog/chop-researcher-advances-adolescent-care-africa/

Mar 21 2014

CHOP Sports Medicine, Concussion Specialist Interviewed by Fox 29

concussion

Christina L. Master, MD, was recently interviewed on Fox 29’s Good Day Philadelphia during a segment about sports injuries and concussion.

The Children’s Hospital of Philadelphia’s Christina L. Master, MD, was recently interviewed on Fox 29’s Good Day Philadelphia during a segment about sports injuries and concussion. In part a report about the recently released documentary film Head Games: The Global Concussion Crisis, Dr. Master discussed the appropriate age for children to begin playing contact sports and helmets, among other topics.

After being asked when it was safe for kids to being playing contact sports like hockey and football, Dr. Master said “we do think that contact sports ought to be held off until they’re older … probably into the middle school, the teenage years.”

Indeed, citing USA Hockey’s example, Dr. Master noted experts urge younger children to focus on fundamental skills, and not participate in contact skills like checking in hockey or heading soccer balls. As part of its Progressive Checking Skill Development Program, USA Hockey recommends that legal body checking not begin until age 13 or 14.

When asked if improved helmets might reduce concussions, Dr. Master pointed out that “when you think about the fact that a concussion is your brain shaking in your skull, there’s really no way a helmet with protect 100 percent against that.”

And when asked what advice Dr. Master might have for parents, she said, “I think it’s important to, as a parent, choose a situation that’s conducive to learning fundamental skills … the idea is to learn the skills, have fun, be fit,” she added.

For more, see the full interview below!

Philadelphia News, Weather and Sports from WTXF FOX 29

Permanent link to this article: http://www.research.chop.edu/blog/chop-sports-medicine-concussion-specialist-interviewed-fox-29/

Mar 19 2014

U.S. News Ranks Department of Pediatrics No. 1 in Nation

pediatricsEach year, U.S.News & World Report ranks professional school programs in business, education, engineering, law and medicine. In its latest survey of U.S. medical schools, announced earlier today, the magazine awarded its top ranking in pediatrics, for the second year in a row, to the physicians and scientists who work at The Children’s Hospital of Philadelphia.

This marks the 11th consecutive year that the program has been ranked first or second in the nation. We want to congratulate the faculty and staff of the Department of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania, CHOP’s academic partner, for this impressive recognition.

U.S.News recognized the Perelman School of Medicine at the University of Pennsylvania as the nation’s best pediatrics department, followed by Harvard University in second place and the University of Cincinnati in third. The university’s Department of Pediatrics is located here at Children’s Hospital.

The 2015 Best Graduate Schools rankings are based on two types of data: expert opinions about program excellence and statistical indicators that measure the quality of a school’s faculty, research and students. The data come from surveys of administrators at more than 1,350 programs and more than 13,500 academics and professionals, conducted during the fall of 2013 and early 2014. The rankings also took into account an institution’s research activity, reflected in grant awards to faculty from the National Institutes of Health.

We congratulate all who have worked so hard to sustain this impressive record of medical and academic excellence and thank them for the distinction they bring to CHOP through their efforts.

Permanent link to this article: http://www.research.chop.edu/blog/u-s-news-ranks-department-pediatrics-1-nation/

Mar 17 2014

New Gene Sequencing Test to Speed Donor Matching, Research

Gene SequencingGenetics experts at The Children’s Hospital of Philadelphia have developed a unique test to characterize the genes that encode HLA molecules. Relying on gene sequencing to type human leukocyte antigens (HLAs) — complex proteins that are essential to immune function — the new test has the potential to improve transplantation outcomes through refined donor compatibility assessments, and will expedite the donor selection process from bone marrow registries.

“This new test addresses a sixty-year-old problem,” said Dimitri Monos, PhD, director of the Immunogenetics Laboratory in the Division of Genomic Diagnostics. “Since the discovery of HLAs in the early 1950s, it has been a challenge to accurately and thoroughly characterize HLA gene sequences. We have now used next-generation sequencing tools to significantly advance HLA typing.”

The test also provides an advanced tool for research in immunological diseases, infectious diseases, and pharmacogenomics — the field that studies the influence of genetic variations on drug efficacy and toxicity. Children’s Hospital is the first hospital to offer this new comprehensive HLA typing test.

“This is a new, disruptive technology, with the potential to transform research and clinical practice, in transplantation and other fields,” said Robert Doms, MD, PhD, CHOP’s pathologist-in-chief.

HLA genes are the most complex gene family known in the entire human genome, and their sequences are highly variable, to a degree not adequately captured by conventional typing tests. Current tests often provide ambiguous and limited results, by sequencing only segments of HLA genes and failing to distinguish among different alleles suggested by a given sequence. In addition, preliminary testing often must be followed by a second level testing, adding expense and time to the HLA typing process.

The new test, says Dr. Monos, is a single test that provides the highest resolution possible by covering the full HLA genomic region. It can currently distinguish among 10,500 different alleles of all known HLA types and can fully characterize new alleles yet to be discovered. The researchers validated the test by comparing its results against previously sequenced data from a collection of over 300 samples characterized at five different genes.

CHOP will be offering the HLA typing test for patient testing as a service to medical and academic centers. The test is faster, more precise, and costs less than existing testing procedures. The new test’s most significant short-range impact may be in typing donors in bone marrow/stem cell registries.

“This faster, more thorough technology allows us to better account for subtle genetic differences between individuals,” said Dr. Monos. “We expect this knowledge to yield clinical benefits, by facilitating more precise matches between transplant donors and recipients, and assessing the significance of mismatches in genomic regions of the HLAs that were previously uncharacterized.”

To learn more about Children’s Hospital’s genetic testing services, see the Division of Genomic Diagnostics. For more information about the HLA typing test, see the full press release.

Permanent link to this article: http://www.research.chop.edu/blog/new-gene-sequencing-test-speed-donor-matching-research/

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