Epilepsy that first occurs in adults and older children is diagnosable and generally responds well to medication. Unfortunately, when epilepsy begins in infancy or in young children, it represents a set of complex disorders without specific etiologies, resulting in perplexing conditions causing a stressful life for the affected children and their parents. The etiology of infantile epilepsy is multifactorial, including genetics, metabolic, and structural brain issues. This research project, in partnership with Rady Children's Genomic Institute, will explore both the basic science and clinical aspects of epilepsy with an emphasis on children diagnosed as infants or at an early age. The Rady team will provide whole genome sequencing for children, and the Steele team will investigate genetic mutations and determine their functionality and how to treat them. The goal is to develop a new paradigm for treatment of these children through research including whole genome sequencing, and examination of structural brain disorders, and metabolic issues.
The Steele Center has become a leader in pediatric research across the globe. PANDA is proud to have funded internationally recognized physician-scientists to investigate a range of pediatric illnesses including those involving cancer, autoimmune disorders, genetics, gastroenterology, and developmental and behavioral pediatrics. This year, PANDA will focus our fundraising efforts on the Children’s Autoimmunity and Allergies Project, which seeks to help unravel the mystery of common autoimmune issues among children– type 1 diabetes, juvenile arthritis, Crohn’s disease, colitis, eosinophilic esophagitis, celiac disease and postinfectious autoimmune encephalopathy.
Children suffering from complex diseases often face a trial and error approach to treatment that leaves them susceptible to adverse reactions and side effects that may affect them for the rest of their lives. A "personalized" approach hopes to enable physicians to predict a child’s response, resistance, or adverse reaction to particular treatment options. The objective of the PANDA Personalized Pediatric Medicine Project is to conduct pilot studies and establish an integrated system for personalized medical approaches to treat children suffering from complex diseases including cancer. This individualized system will enable physicians to accurately predict a child’s response, resistance, or adverse reaction to particular treatment options.
Instead of relying on statistics from large clinical studies, personalized medicine allows for physicians to focus on an individual child’s response to therapy. This customized treatment approach will enable researchers to identify markers in a child’s genetic sequence that influence why certain treatments work and others fail. Identifying precise genomic signatures will help provide a more direct route from diagnosis to successful treatment for each patient. Tailoring treatments for each individual child is the ultimate goal.
The traditional field of cancer treatment focuses on eliminating malignant cells. While traditional chemotherapy directly kills cancer cells it also has devastating side effects. In addition, drug-resistant tumor cell clones may emerge, which lead to a relapse of the cancer and sometimes death. Over the last decade, with funding support from PANDA, The Steele Children’s Research Center has pioneered the use of chemotherapeutic drugs at non-toxic levels in combination with immunotherapy. The results have been primary tumor elimination, prevention of cancer metastasis and improved survival for our children.
Our objective is to create an interface between traditional cancer chemotherapies and immune-based strategies, focusing on pediatric cancer patients. The PANDA Children’s Anti-Tumor Immunity Program, integrated within the Steele Children’s Research Center, will identify new generation anti-cancer drugs with the ability to contain the cancer, develop natural immunity to the cells and reduce the emergence of tumor cell clones. We will then translate the most promising of these approaches to the children’s bedside.
The 2013 and 2014 proceeds funded the PANDA Children's Autoimmune Disorders Project and endowment. Help and answers are desperately needed for families facing the challenges of autoimmune diseases like type 1 diabetes, juvenile arthritis, Crohn's/colitis, severe food allergies, eosinophilic esophagitis, and celiac disease.
The human immune system is designed to recognize its own cells and to fight off germs carrying disease. Autoimmune diseases result when the bodies' fighter cells turn against its own healthy cells, and destroy them. Current estimates suggest that as many as 9.4% of the world's population suffer from an autoimmune disease, and in the US it is estimated to be as many as 50 million people, costing nearly $100 billion per year in treatment and care. These diseases are progressive, debilitating, and are one of the leading causes of death and disability. Children are being diagnosed at ever-increasing rates, and we must ensure that research is rapid and able to improve and affect care for our children. The PANDA Children's Autoimmune Disorders Project will continue to build on discoveries being made at the Steele Children's Research Center.
The 2012 proceeds funded the Steele Children’s Research Center – Phoenix Translational Center. Historically, the University of Arizona College of Medicine has been located in Tucson, nurturing our state’s future physicians and conducting academic research in one location. With Arizona’s tremendous growth has come the need for additional physicians and scientists to serve our communities. Recently, a second College of Medicine campus was created and is located in the heart of downtown Phoenix.
Developing a physical presence in Maricopa County will allow the Steele Center to lead the state’s pediatric basic science research, with bench-to-bedside discoveries at its laboratory center in Tucson, while partnering with clinical enterprises in Maricopa County to fully expand its translational reach in downtown Phoenix. The Phoenix Translational Center will help to provide the resources for physician-scientists to carry out safe, efficient and ethical clinical research involving children, train and develop pediatric investigators for the future and educate the public on children’s health and advances in the treatment of pediatric diseases.
The 2011 project helped fund efforts to help some of the most vulnerable patients, babies in the neonatal intensive care unit (NICU). More than a half million babies in the United States and nearly 10,000 in Arizona are born prematurely each year. Preterm birth is the leading cause of infant illness and accounts for more than two thirds of all infant deaths. The PANDA Healthy Babies Project is working to increase the likelihood that babies can leave the NICU healthy and in the shortest amount of time. Of the myriad of health issues that premature babies face, Necrotizing Enterocolitis (NEC) is the most common gastrointestinal disease that affects them. NEC is an inflammatory disease that can cause destruction of the intestine and in its severest form can result in surgical removal of portions of the affected intestine. Sadly, 20% to 40% of babies affected with NEC will die from the disease.
Currently, the University of Arizona’s Steele Children’s Research Center is the only facility in the state of Arizona that combines the highest level clinical services for preterm infants along with cutting edge basic science research. The Steele Center boasts several of the best known scientists and physicians in this field, including Drs. Alan Bedrick, MD, Bohuslav Dvorak, PhD and Melissa Halpern, PhD. In addition to their current work, the team is also taking steps to build collaborative research partnerships with NICUs throughout Arizona, including the Phoenix area.
With PANDA’s help and your generous support, the Steele Center is:
- Advancing the field of neonatology through a better understanding of why certain premature infants get NEC
- Working to discover ways to prevent babies from developing NEC
- Finding the means to predict NEC before it becomes a medical or surgical emergency
- Funding training for new scientists with interest in neonatology, building on the Steele Center’s strong foundation in neonatology research
Project 2010 funded the PANDA Children’s Cancer Immunology Program that is on the cutting-edge of offering a promising option to fight cancer. Children fighting cancer face tremendous odds. Despite advances in survival rates, chemotherapy and radiation are toxic, causing short-term suffering and long-term complications.
The PANDA Children’s Cancer Immunology Program funded research for a new option of treatment—immunotherapy. Immunotherapy re-programs a child’s own disease-fighting cells to recognize his or her own individual cancer cells and prevent them from multiplying. Scientists believe that a child’s own immune system may become an effective way to treat cancer or to prevent relapses.
Dr. Emmanuel Katsanis at the Steele Children’s Research Center heads a team of physician-scientists conducting research in pediatric immunotherapy. Dr. Katsanis and the Steele Center are one of the few research centers in the country conducting pediatric cancer immunology research and developing novel immunotherapy treatments against cancer.
The PANDA Children’s Cancer Immunology Program fund the:
- Development of promising, individualized anti-cancer vaccines to stimulate the immune system to fight cancer
- Advancement of cancer research—transitioning successful laboratory research to human clinical trials
- Recruitment of scientists to the Steele Center to further the expansion of pediatric cancer immunology research
With your generous support, the PANDA Children’s Cancer Immunology Program can potentially change the way we treat pediatric cancer in the future!
The 2009 "Children Helping Children" Fashion Show and Golf Tournament proceeds established the "PANDA Children's Neurological Center," a new comprehensive care facility to help children and families receive state-of-the-art specialized care in a single location. The facility is focused on improving the lives of thousands of families in Arizona whose children suffer from neurological disorders -encompassing traumatic brain injury, stroke, autism, near-drownings, meningitis, brain tumors, cerebral palsy and other causes.
These families face splintered care that is hard to find, hard to get, and incredibly frustrating for parents already overwhelmed by the challenges, fear and frustrations that come from raising a child with neurological insult. Staffed with the neurologists, behavioral pediatricians, therapists and specialists these children need, the PANDA Children's Neurological Center has multidisciplinary teams which work together to evaluate, diagnose and develop treatment plans for each patient.
The finest physicians and specialists who treat children with brain injury are hard to find and expensive to recruit. PANDA has made a two-year commitment to raise more than $1 million to create the physical space for the Center, to recruit a top-notch team, to provide seed money to ensure the Center grows and flourishes, and to promise that every child in Arizona who needs its services can receive them, regardless of their ability to pay.
The 2008 “Children Helping Children” Fashion Show proceeds funded the “Women in Science” program being developed at the Steele Children’s Research Center. While more and more women enter the medical field every year, currently only 25% of physicians are women and even fewer practice medical research. The Steele Center’s “Women in Science” program supports promising female physician-scientists as they build their clinical and research careers.
The program is currently supporting:
Dr. Hillary Franke is a pediatric intensivist who takes care of critically ill children. Her research focuses on quality of patient care in pediatrics and pediatric critical care. More specifically, Dr. Franke is interested in medication safety and high-alert medications (medications with the highest risk of causing injury when misused) in the Pediatric Intensive Care Unit (PICU). Dr. Franke has developed a list of PICU-specific high-alert medications, and will be conducting a quantitative analysis of medication harm versus clinician perceptions. Their perceptions, whether correct or not, strongly influence behavior and shape the work culture.
Dr. Sydney Rice is a developmental and behavioral pediatrician and a leader in the areas of traumatic brain injury (TBI) and autism. Dr. Rice is currently involved in an epidemiologic study of autism, and is collaborating on an ADHD research project examining the use of medications in preschoolers. She is also interested in how traumatic brain injury affects the interactions between the nervous and endocrine systems (neuroendocrine).
The PANDA Children’s Aerodigestive Disorders Center is both a clinical and research facility. It is the only one of its kind in the entire state of Arizona. The center serves children who are suffering from eosinophilic gastrointestinal disorders – disorders of the digestive tract caused by allergies to food and airborne allergens. These disorders are known as “EE” for eosinophilic esophagitis, and “EG” for eosinophilic gastroenteritis (a more debilitating form of EE). Children with EE or EG often fail to grow, suffer from nausea, vomiting, stomach pain, choking, cramping, and diarrhea. Arizona has far more cases of EE/ EG than the national average, yet there was no place in the western U.S. where children can receive dedicated care for this painful disease. In response, the PANDA Center now serves Arizona and the Southwest, employing a team approach to treat children with aerodigestive disorders.
The aerodigestive team includes Fayez K. Ghishan, MD, director of the Steele Center and a pediatric gastroenterologist; Michael Daines, MD, pediatric allergist/immunologist; Cori Daines, MD, pediatric pulmonologist; and Lindsay Brown, MS, RD, nutritionist.
“Aerodigestive disorders span the sub-specialties of gastroenterology/nutrition, pulmonary and allergy/immunology, so a team approach is the most effective way to treat these children,” Dr. Ghishan explains.
“This center has been made possible because of the generosity of the PANDAs, and we are grateful for their fundraising efforts that have made the PANDA Center a reality,” he adds.
We are grateful for the ongoing support from the community and look forward to a continued partnership that improves the lives of children not only in Arizona, but across the United States and beyond.