Although the year is coming to a close, the research achievements at The Children’s Hospital of Philadelphia in 2015 remain enduring contributions to pediatric health. A special year-in-review issue of the Research Institute’s Bench to Bedside online news publication looked back at 2015 and highlighted some of the scientific advancements that made the year remarkable.
A $50 million gift from Philadelphia philanthropist Raymond G. Perelman that provides direct support for an expansion of pediatric research was an inspiring start in January. A Center of Emphasis at the Research Institute took on a new name — the Raymond G. Perelman Center for Cellular & Molecular Therapeutics (CCMT) — and CCMT researchers made progress in their pursuit of new gene and cell therapies in search of cures for debilitating disorders.
Spring brought the launch of two ambitious initiatives: the Center for Perinatal and Pediatric Health Disparities Research and the Fetal Neuroprotection and Neuroplasticity Program. Also with high aspirations, the PennCHOP Microbiome Program opened its doors in late summer, welcoming investigators to augment their current research interests by exploring how the microbiome influences pediatric health and disease. Visit the program’s homepage to see vibrant pictures of the microbes — too small to be seen by the naked eye — that live in our guts and elsewhere on our bodies.
This spirit of innovation spread throughout several large-scale research collaborations in which CHOP researchers have leadership roles. Check out the projects’ catchy acronyms:
CER2: The Comparative Effectiveness Research Through Collaborative Electronic Reporting Consortium is creating a national “super network” devoted to comparative effectiveness research based on electronic health record clinical data.
PEDSnet: A National Pediatric Learning Health System is a patient-centered clinical data research network headquartered at CHOP that received renewed funding to enter its next phase.
eMERGE: The Electronic Medical Records and Genomics program encourages scientists who uncover the genetic causes of pediatric conditions to take the next step and analyze how this knowledge can be implemented in medical settings to improve care.
PEPR: The Validation of Pediatric Patient-Reported Outcomes in Chronic Diseases Consortium, which consists of CHOP and three partner institutions, will advance the science of patient-reported outcome measures.
Yet the biggest buzzwords of the year likely were “precision medicine.” President Obama launched the Precision Medicine Initiative in January, saying it offers “one of the greatest opportunities for new medical breakthroughs that we have ever seen.”
Indeed, CHOP researchers already had begun delving into ways to personalize treatments based on the specific molecular pathway or gene found to underlie the patient’s own disease. Most recently, CHOP researchers reported on how a change in the LMO1 gene results in a “super-enhancer” that causes tumors to arise and grow out of control in an aggressive subtype of neuroblastoma. Their findings may present new opportunities to develop novel treatment options.
Explore more genetic discoveries in the December issue of Bench to Bedside, along with other year-end articles about research projects that had wide-ranging impact on patient safety, policy, and technology. What a year!