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Sharon J. Diskin, PhD

Fri, 03/08/2019 - 11:20
Sharon J. Diskin, PhD

Assistant Professor of Pediatrics 

My research uses integrative quantitative computational methods and rigorous experimental approaches to understand the genetic basis of childhood cancers and identify new therapeutic targets. My long term goal is to improve outcomes for those diagnosed with cancer or to prevent cancer from initiating. I was drawn to the field of cancer research after a loved one was diagnosed with colon cancer over 20 years ago, prompting a career conversion from the aerospace industry. I remain driven by the amazing young children fighting cancer yet still smiling, and their families.

I would give two words of advice to young women interested in pursuing science – perseverance and balance. Perseverance is an essential quality of all successful scientists. There may be times when it seems that nothing is working, your experiments aren’t working, the road seems too long, you may even think you aren’t cut out for a career in science. Don’t quit, believe in yourself, have perseverance. Equally important, especially for woman, is balance. There will be competing priorities – career, family, friends, and self. Be sure to make time for all of these. Balance will make all the highs and lows of your scientific career better.

Rachel K. Myers, PhD

Fri, 03/08/2019 - 09:37
Rachel K. Myers, PhD

Research Scientist, Violence Prevention Initiative, Center for Injury Research and Prevention

My research focuses on recovery after violence-related injury and developing innovative ways to address the burden of pediatric injuries. I am interested in how we can use technology to reduce the burden on patient families and improve the accuracy of our data. My interest grew from work in adolescent health, where disparities remain in which youth are most affected by violence. Working with a multidisciplinary team, I see firsthand the outcomes our programs help youth achieve, and I am driven to understand what works and how, so we can disseminate our models of care to other institutions.

I encourage young women to seek out mentors and colleagues who help build their confidence, provide critical but supportive feedback, and value their contributions. Science is a team sport, and who we work with can be as important, if not more so, than what the work is. During my first mentored research opportunity, my female mentor suggested to me that a male peer carry out more complicated calculations. While this is not the introduction to the world of science that any young woman should have, it was foundational in helping me to understand the importance of colleagues who respect your skills, provide opportunities for growth, and celebrate successes.

Yael Mossé, MD

Fri, 03/08/2019 - 09:36
Yael Mossé, MD

Attending Physician, Division of Oncology

I am a physician-scientist committed to defining and exploiting oncogenic pathways in pediatric cancers – with a focus on neuroblastoma – and translating my basic science discoveries to the clinic. Despite major enhancements in the intensity of therapy over the past several decades, the cure rate for patients with high-risk neuroblastoma lags significantly behind that of other childhood cancers. My lab has unwaveringly focused on the hypothesis that discovery of the genetic basis of this disease will provide insights that are clinically actionable and improve patient outcomes. We have harnessed our discovery of germline and somatic mutations in the Anaplastic Lymphoma Kinase gene as an opportunity to make a big difference for a small group of patients battling this often lethal disease. 

Focus. Perseverance. Ruthless prioritization. [These are] distinguishing characteristics that have inspired and motivated me and that I hope to impart to young women in science. Perhaps with these attributes always front and center, one has the opportunity to really do something great.

Sarah Tasian, MD

Fri, 03/08/2019 - 09:34
Sarah Tasian, MD

Attending Physician, Division of Oncology

I am a pediatric oncologist and physician-scientist with a research focus on precision medicine therapeutics for high-risk childhood leukemias. My translational lab has two major interests: preclinical testing of kinase inhibitors in genetic subtypes of acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) and preclinical testing of CAR T cell immunotherapies in AML and ALL subtypes. I also lead or co-lead early phase clinical trials in these areas through the Children’s Oncology Group and other consortia. All of this work is truly inspired by our young patients. They quietly show us the real problems in childhood cancer and highlight the gaps in our knowledge as physicians and scientists. We must listen to our patients carefully and deeply, then investigate diligently in the lab how to solve these problems with a goal of bringing better therapies to the clinic as quickly as possible.

Persist! Maintain a laser focus on your goals, and don’t be discouraged if a first (or second or third) attempt doesn’t work. Be open to new ideas and circumstances that may arise along the way, as these opportunities can sometimes be real silver linings.

Elizabeth Walshe, PhD

Fri, 03/08/2019 - 09:29
Elizabeth Walshe, PhD

Research Postdoctoral Fellow, Center for Injury Research and Prevention

My research brings together the latest neuroimaging methods with virtual driving technology, establishing the Neuroscience of Driving Research Program. This program bridges basic neuroscience with applied driving research to tackle the public health problem of motor vehicle crashes. My research focuses on understanding how ongoing development of the brain and cognitive function during adolescence may impact the ability of teen drivers, alongside developing simulated driving as an ecologically relevant probe of brain function so that we can investigate typical and atypical neural and cognitive development in adolescents.

Persevere. We use the scientific method as a tool to generate new knowledge and understanding of the world around us: to investigate the unknown. So, by definition, the answer, the solution, and the path ahead will almost never be clear. To be successful in your scientific inquiry, and in a career in science, you must persevere. Keep alive your passion for the scientific method and for using it to create change, inform healthcare, and better our society. This will help you to keep going when you are unsure, get back up when you get set back, and carry on when you learn that it’s not how you thought.

Valentina Graci, PhD

Fri, 03/08/2019 - 09:21
Valentina Graci, PhD

Research Scientist, Center for Injury Research and Prevention

My research focuses on examining how children and adults move when exposed to crash-avoidance maneuvers in vehicles, and how to improve reaction times in autonomous driving scenarios that require manual take over. I conducted biomechanics and human movement research for 10 years before CHOP. Here, I had the opportunity to come face to face with automotive industries and understand their needs for improving occupant safety. This drives my motivation in understanding how to use concepts that are well known in the human movement science world to develop effective novel warning systems for driverless technology. 

Be daring and adventurous. There is not just one recipe for success! In a male-dominated world, young female investigators may feel more inclined to follow traditional stepping stones in their career. But we need to be creative in finding opportunities. Before CHOP, I was often told that I did not follow the traditional path and I had changed fields too many times. However, I would not have been able to create my own research questions and feel excited about them if I had not done that. I feel fortunate about the creativity that my eclectic background has given me.

Denise Sabatino, PhD

Fri, 03/08/2019 - 09:19
Denise Sabatino, PhD

Research Assistant Professor of Pediatrics, Division of Hematology

My research focuses on the inherited bleeding disorder hemophilia and the development of gene therapy to treat this disorder. The current treatment for hemophilia is protein replacement therapy that requires frequent clotting factor infusions to stop bleeding episodes. Using a gene-based approach, our goal is to achieve sustained therapeutic levels of the clotting factor so that patients no longer require frequent protein treatments and to prevent bleeding episodes. We focus on characterizing novel clotting factor protein variants that have enhanced function and other strategies to optimize a gene therapy approach to treat hemophilia. Our work has supported a current clinical trial for gene therapy for hemophilia A. It has been inspiring to see our findings in the laboratory translated into a new treatment for hemophilia.  

As young women pursue a career in science, it is important to understand that life will sometimes get in the way and impact your path. Learning to navigate these unanticipated twists and turns with your career goals can be an arduous journey. Embrace the collaboration and support of colleagues, but above all, face these challenges with courage and determination.

Building a Solid Foundation

Fri, 12/07/2018 - 09:56
Building a Solid Foundation

The Center for Autism Research was founded in 2008 as a joint center spanning between CHOP and the University of Pennsylvania. It grew quickly into one of the largest and most comprehensive autism research centers in the world. Scientific director Robert Schultz, PhD, a neuropsychologist pioneering autism brain imaging research, was recruited from Yale University to build the new center from the ground up with developmental pediatrician Susan E. Levy, MD, MPH, as medical director. “Dr. Schultz’s studies of the social brain were the perfect complement to the clinical research taking place at the Regional Autism Center at that time and strengthened our ability to improve early diagnosis and develop treatments for children with autism spectrum disorder,” Dr. Levy said.

In addition to advancing the science and improving clinical care for patients with ASD, it’s a central part of CAR’s mission to support families currently navigating an autism diagnosis. CAR’s outreach team hosts regular talks and workshops, and the CAR Autism Roadmap ™ is a 24/7 reference and resource directory for parents and clinicians.

Infant Brain Imaging Study Begins

Fri, 12/07/2018 - 09:54
Infant Brain Imaging Study Begins

Within the first year, under Dr. Schultz’ leadership, CAR initiated the Infant Brain Imaging Study (IBIS) funded by the Autism Centers of Excellence grant program of the National Institute of Mental Health. The long-term study focuses on infants with an older sibling already diagnosed with ASD, since these infants are at a higher risk for developing autism by age 2. Researchers use magnetic resonance imaging (MRI) to look for changes in the brains of infants who go on to develop ASD, compared with those who do not receive a diagnosis. The IBIS study, which is still active today, has produced several groundbreaking research findings, including a recent study which applied machine learning techniques to brain imaging data, to identify with more than 96 percent accuracy which children would go on to be diagnosed with autism by 2 years of age, and which children would not. “This is an astounding observation that could fundamentally change clinical practice,” said Dr. Schultz, who leads the CHOP research site along with Juhi Pandey, PhD.

Enhancing Autism Support in Schools

Fri, 12/07/2018 - 09:52
Enhancing Autism Support in Schools

Most children with autism receive the majority of their care in school. In 2008, CAR’s Associate Director, David Mandell, ScD, who also directs Penn’s Center for Mental Health Policy & Services Research, initiated Philly AIMS, the largest-ever randomized trial of an educational intervention for children with autism, and the first to be conducted as a partnership between an academic research center and a school district. Although the study ended several years ago, the Philly AIMS team continues to provide integrated support via professional development training and classroom-based consultation to Philadelphia School District teachers in grades K‐5 autism support classrooms.

Dr. Mandell’s team also is conducting research in Philadelphia preschools to analyze the type and quality of intervention being delivered in inclusive versus non-inclusive settings. His goal is to parse out which settings are best for which children. “By comparing outcomes of different early intervention practices and investigating intervention-child fit in community-based settings, we hope to make complex early intervention placement decisions easier for both parents and providers,” Dr. Mandell said.

Genetic Discoveries Key to Unravelling Autism

Fri, 12/07/2018 - 09:51
Genetic Discoveries Key to Unravelling Autism

In collaboration with Hakon Hakonarson, MD, PhD, and the Center for Applied Genomics (CAG), CAR has led some of the most important genetic discoveries related to autism, including the 2009 discovery of the first common genetic variants of ASD, accounting for up to 15 percent of all ASD cases. CAG-CAR research also has informed the study of genetically targeted drugs that could improve social communication difficulties, a core symptom of ASD.

Approximately 75 genes are known to significantly increase the risk of autism, and there are likely hundreds more. Capitalizing on the technology and infrastructure established by the Roberts Collaborative for Genetics and Individualized Medicine and as a research site in the Simons SPARK study, CHOP is poised to unravel the complicated genetics of autism that have kept its origins a mystery for decades.

CAR continues to accelerate research along with the Department of Biomedical and Health Informatics and the Division of Developmental and Behavioral Pediatrics by establishing a biorepository that integrates genetic data with available corresponding medical, developmental, and behavioral information from consenting patients.

New Research Approach: Digital Phenotyping

Fri, 12/07/2018 - 09:49
New Research Approach: Digital Phenotyping

CAR’s pioneering research has increased understanding of the social behaviors associated with autism that may interfere with daily life. Dr. Schultz and his team were the first to show that video games can help improve the ability of individuals with autism to understand others’ facial expressions. Two studies under way use therapeutic video games to help children with ASD develop their social skills and ability to concentrate. A third study partners with the Philadelphia Police Department and uses virtual reality to help young people with ASD acclimate to appropriate and safe interactions with law enforcement.

CAR’s international leadership in “digital phenotyping” is ushering in a new era for autism research. All behaviors that an expert clinician can observe may now be captured with sophisticated technology and used in artificial intelligence analytic frameworks to produce precise, research grade information that is predictive of evaluations requiring more than 10 times the amount of time and resources. In pilot studies, this technology predicted with greater than 90 percent accuracy — after a three-minute conversation — if a person has ASD.

Using Research to Inform Clinical Care

Fri, 12/07/2018 - 09:48
Using Research to Inform Clinical Care

CHOP is working on an ambitious project to create an Autism Spectrum Disorder Learning Healthcare System (LHS) in which scientific evidence informs clinical practice, and clinical practice promotes scientific investigation by generating new data for analysis. CHOP provides care for more than 10,000 patients with ASD, which allows LHS experts to discern patterns in symptoms, behavior, genetics, and developmental factors. They can use this knowledge to tailor a patient’s clinical support programs.

“An LHS effectively turns the clinic into a research lab, where discovery is a natural outgrowth of patient care,” Dr. Schultz said.

Developmental pediatrician Amanda Bennett , MD, MPH,, who is part of the team building the LHS infrastructure, finds the new approach incredibly exciting: “Unlike a research study or clinical trial in which some children receive the investigational therapy and others do not, with the LHS we give each child the individualized care they need now. Then, we track and analyze their reactions to that treatment — adjusting as needed — and use that information to inform future care for children with autism."

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