CHRI sponsored the 2nd annual UNMC Pediatric Cancer Research Symposium at the Truhlsen Campus Events Center on August 26-27. This event is a showcase for multidisciplinary research studies in laboratories affiliated with the Pediatric Cancer Research Group (PCRG), which is directed by Don Coulter, MD Among attendees at the symposium were representatives of several Nebraska-based philanthropies, the University of Nebraska
Foundation, the Children’s Hospital & Medical Center Foundation, and the Nebraska State Legislature, which comprise a coalition of generous support for pediatric cancer research through the PCRG. “The PCRG represents a unique group of over 30 members conducting team science with resources obtained from both public and private donors,” noted Dr. Coulter. “With the current low funding lines through the NIH, the PCRG represents a true success in outside the box thinking when it comes to completing research for kids.”
In eight plenary presentations and a poster session with nearly two dozen posters, UNMC scientists presented their work on a spectrum of topics ranging from cancer epidemiology, to mechanisms of oncogenesis and metastasis, to drug delivery. Keynote speaker, Brenda Weigel, MD, integrated many of these themes and more in her presentation, “Pediatric Drug Development: The Changing Landscape.”
Dr. Weigel, who is Professor and Director of Pediatric Hematology and Oncology at the University of Minnesota, named several major challenges that continue to impede progress on pediatric cancer treatment. Most fundamental is the fact that pediatric cancers are relatively rare; even though thousands of US children are diagnosed per year, cancer incidence in children is less than 1% of the incidence in adults. This means the incentive for research and development investment in pediatric cancer treatments continually lags behind. Additionally, Dr. Weigel stressed that the pediatric cancers are largely different diseases than adult cancers, with different underlying mechanisms, and it often turns out that children have far different reactions and sensitivities to therapeutic drugs. Furthermore, risk-benefit ratios of treatment options are different. Months or years of extended life expectancy that may be satisfactory for an older adult population often pose an unacceptable outlook for pediatric patients.
Dr. Weigel also discussed evidence of notable innovation and progress for pediatric cancer, in the realms of both science and policy-making. Thanks in part to international collaborative efforts like the Children’s Oncology Group (COG), resources are being pooled to design and support studies that examine critical issues for pediatric patients, including drug bioavailability and dose formulation, developing validated in vivo models, and quality-of-life issues for survivors. In the early days of “precision medicine,” drugs such as crizotinib are being applied for tailored use against childhood tumors with specific gene mutations. Tumor microenvironment has been recognized as a pivotal factor in cancer development and control, but models to understand and manipulate it in the pediatric patients have only started emerging. The federal 2017 Research to Accelerate Cures and Equity for Children (RACE) Act, was catalyzed by the voices of advocacy groups. RACE mandates that a company developing a cancer drug study its safety and efficacy in children, if the drug’s molecular target was deemed relevant to a pediatric cancer on a list issued by the FDA and NCI.
UNMC investigators’ presentations throughout the day provided closer snapshots of many of the themes laid out in the keynote presentation, demonstrating the depth and breadth of laboratory and clinical expertise engaged in research at UNMC and CHMC on behalf of children with cancer. Dr. Coulter affirmed, “Research is one of the facets that makes the relationship between UNMC and CHMC so successful, and our group completes research in multiple different avenues, including basic, translational and population health. Today was an excellent opportunity to share the hard work of dedicated researchers focused on the health and well-being of pediatric cancer patents.”
The UNMC speakers included D.J. Murry, PharMD, UNMC Department of Pharmacy Practice and Science; Shannon Buckley, PhD, UNMC Department of Genetics, Cell Biology and Anatomy; Gargi Ghosal, PhD, UNMC Department of Genetics, Cell Biology and Anatomy; Joe Vetro, PhD, UNMC Department of Pharmaceutical Science; Ram Mahato, PhD, UNMC Department of Pharmaceutical Science; and Nina Baranowska-Kortylewicz, PhD
by Matthew Sandbulte, CHRI Grant Writer | September 18, 2019