As we look ahead to a new year and new opportunities in 2017, it’s worth first taking a look back at how much we achieved in 2016. It was a busy year, and investigators from Children’s Hospital of Philadelphia made remarkable progress and reached major milestones of discovery to aid children’s health. These achievements are too numerous to mention in a single roundup, so this review will not be comprehensive. Even so, these highlights of stories we brought you over the course of 2016 in Bench to Bedside should give a good taste of where we’ve been — and whet your appetites for what’s to come.
New Research Initiatives Launch
The past year saw the launch of several new research initiatives at CHOP. In January, the Center for Data-Driven Discovery in Biomedicine was announced, and in November we brought you the latest updates on its major data-sharing platform, CAVATICA. Two new Research Affinity Groups also began, focused on mHealth (health research using mobile and digital tools) and on global health. And in the fall, CHOP announced the new $50 million Roberts Collaborative for Genetics and Individualized Medicine.
Childhood Cancer Advances
Childhood cancers are among the most critical and devastating diseases that researchers at CHOP are working to address. CHOP was honored to host the Coalition Against Childhood Cancer for its summit and annual meeting over the summer, while CHOP researchers have worked on continuous advances against cancer at multiple levels including molecular, technological, and national leadership. Some of the highlights include:
- CHOP oncologist Peter Adamson, MD, serving as a member of the Blue Ribbon Panel for the National Cancer Moonshot
- Developing plans for an innovative clinical trial for neuroblastoma, NEPENTHE, that speeds access to new precision treatments and may be a model for how future precision medicine clinical trials are designed
- Making important preclinical discoveries, such as potential new therapies for neuroblastoma and a high-risk subset of acute lymphoblastic leukemia
- Determining whether some treatments are best delivered at home or in the hospital
- Developing ways to automate processes in cancer clinical trials to better capture data about treatment side effects
- Looking out for the needs of childhood cancer survivors including medical complications and self-management as they grow up
Molecular Secrets Unlocked
The “bench” aspect of bench-to-bedside research is critically important for understanding mechanisms of disease at a molecular level and pioneering new treatments that act on these mechanisms. CHOP had more than its fair share of exciting basic science discoveries in 2016 — and many had clear connections to future translation into therapies. CHOP researchers found an epigenetic mechanism that connects cancer cell growth with cancer cell proliferation that might apply to numerous cancer cell types. Another team discovered an RNA molecule that might play a critical role in the body’s inflammatory responses in multiple diseases. Other immunology research uncovered a mechanism by which viruses can trick the immune system and take over its machinery, which gives new insight into how that machinery works in the first place. And another team described an important molecular interaction in the braking system that the body uses to prevent an allergic or autoimmune response.
Enhancing Child, Caregiver, and Pediatrician Relationships
At the opposite end of the bench-to-bedside research spectrum, investigators at CHOP are also deeply invested in improving care for patients. Several studies in 2016 show how CHOP researchers and clinicians are doing so by enhancing relationships among pediatric providers, parents or other caregivers, and children, to support children’s health together. Some highlights:
- Launching a portfolio of activities focused on intergenerational family services, i.e. supporting children’s health by supporting the needs of parents and caregivers
- Asking parents and teens how they can better support adolescent health needs through family communication
- Empowering parents with skills to address preschoolers’ behavior problems before they become diagnosable behavioral disorders
- Developing tools to help pediatricians support parents’ desire to quit smoking
- Identifying needs for better communication between parents and primary care pediatricians about autism care
- Partnering with caregivers to develop personalized accommodations for nonverbal and minimally verbal children on the autism spectrum so they can participate in research and ultimately benefit from the findings
Research Partnerships with Education, Government, and Large Systems
To truly make an impact on children’s health, no single pediatric hospital can go it alone. Researchers at CHOP have embraced this principle and worked in partnership with schools, government agencies, and large healthcare systems to reach children wherever they may need assistance to thrive in full health.
In schools, for example, CHOP researchers are partnering to improve behavioral health services for students and to collaboratively create and test behavioral interventions to help children develop healthy relationship skills. They are also partnering with teachers and students on creative ways to improve education in cardiopulmonary resuscitation (CPR) to improve the health of entire communities.
Other CHOP research on CPR in the hospital is tied into larger healthcare systems so that innovations developed in critical care here can save lives across the country and around the world. Likewise, innovative projects in pediatric medical trauma systems and in psychological trauma-informed care are connected to opportunities for systemic improvement through the healthcare system and through policymakers. CHOP researchers also worked closely with policymakers to improve care for vulnerable children who receive prescriptions for psychotropic medications that may be unwarranted.
CHOP is a powerful hub for research on the power plants of the cell, the mitochondria. Efforts in the Center for Mitochondrial and Epigenomic Medicine, the Mitochondrial Disease Clinical Center, and across other collaborating specialties, are converging on deeper understanding of how energy production in mitochondria relates to a wide range of diseases and conditions, as well as new approaches to treating conditions known to be connected to mitochondria. Among the highlights this year:
- Discovering functions or possible impairments of mitochondria in the brain that are involved in normal brain functioning and recovery from injury and in neuropsychiatric disease
- A CHOP expert weighing in on ethical considerations for new reproductive technologies to prevent inheritance of mitochondrial diseases, and taking part in national and international efforts to speed the development of new therapies for these diseases
- A CHOP mitochondrial medicine pioneer receiving a prestigious honor shared with such luminaries as Albert Einstein, Louis Pasteur, and Benjamin Franklin
- Cancer biologists pursuing the hypothesis that mitochondria could be part of the mechanism by which cancer resists therapies
- Investigating a mystery surrounding mitochondrial proteins and cellular stress responses
The promise of new technology is often exciting, and it is certainly so in pediatric research. In addition to highlighting the launch of the CHOP mHealth Research Affinity Group, we also shared several stories about mHealth projects in a June special issue of Bench to Bedside: CHOP researchers are using social media tools to improve HIV prevention and care for teenagers, using a mobile app to collect data about babies’ sleep patterns, connecting young adult survivors of childhood cancer with continued care, and using digital tools with incentives to improve teens’ control over diabetes.
New technologies infuse many other areas of research, too. The advent of lymphatic imaging technologies is one area where CHOP and Penn researchers have made remarkable strides in both understanding and treating an underappreciated organ system. Elsewhere at CHOP, researchers are developing imaging tools to understand the placenta, testing ways to speed blood vessel repair with magnetism, developing computer-vision technologies to quantify dermatological assessments, and creating tools to link pediatric care with public health systems.
What innovations will 2017 bring? Stay with us to find out, and get a preview in our look ahead!