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In the News: FDA Approval, Opioid-related Poisoning, Science Slam, PCORI Funding
In this week's news roundup, the U.S. FDA approved the first drug to treat Friedreich's ataxia, a study finds a drastic increase in opioid-related poisonings in children, CHOP celebrates its first annual Science Slam winners, and Clinical Futures, a Children's Hospital of Philadelphia Center of Emphasis, receives PCORI funding to accelerate the journey of research findings into clinical practice.
U.S. FDA Approves First Friedreich's Ataxia Treatment
Friedreich's ataxia (FA) is a rare autosomal recessive neuromuscular disorder that causes progressive loss of muscle strength and coordination leading to an inability to walk or speak, and it affects approximately 5,000 patients in the United States. The U.S. Food and Drug Administration approved omaveloxolone (Skyclarys™), the first drug to treat FA. David Lynch, MD, PhD, director of the Friedreich's Ataxia Program and attending neurologist in the Division of Neurology at CHOP, has led several studies demonstrating the drug's effectiveness in improving neurological symptoms and slowing overall disease progression for individuals 16 to 40 years of age.
Mutations in the frataxin gene cause FA, which leads to dysfunction in the mitochondria that results in degeneration of several areas in the brain and spinal cord. The disease also impairs Nrf2 signaling, which regulates cellular defense against oxidative stress. Nrf2 signals, which are activated by omaveloxolone, are a key area of study for Dr. Lynch.
"We are grateful for the support of the Friedreich's Ataxia Research Alliance, whose generous funding supported the background research that paved the way for this development, and to the patients who participated in the trials that led to this approval," Dr. Lynch said.
Learn more in this CHOP press release.
Study Finds Concerning Increase in Opioid-related Poisonings in Children
Christopher Gaw, MD, MBE, is first author of study that found a significant increase in the number of young children who have died from opioid-related poisonings over a three-year period.
Scientists analyzed data from 40 U.S. states registered in the National Center for Fatality Review and Prevention, which provides resources for child death reviews and maintains a centralized reporting system that collects data.
"By comprehensively assessing fatal poisonings among children at a national level, we were able to better understand the scale of this tragic and preventable public health issue," said Dr. Gaw, pediatric emergency medicine fellow with CHOP's Poison Control Center and the Center for Injury Research and Prevention at CHOP. "We were also able to specifically characterize the proportion of poisoning deaths that could be attributed to opioids each year."
Opioids contributed to 24.1% of deaths in 2005, rising to 52.2% by 2018. More than half of reported poisonings affected children 5 years old and younger, and more than 65% of the fatalities occurred at home. Despite initiatives to reduce opioid prescriptions and utilize single-dose packaging, these safeguards do not apply to illicit sources of opioids such as heroin and fentanyl.
"It's clear from these findings that preventing fatal pediatric poisonings requires a multifaceted approach involving caregiver education and community-level interventions," said Daniel J. Corwin, MD, MSCE, senior author on the study and associate director of research in the Division of Emergency Medicine at CHOP. "One such intervention is improving the availability of naloxone for the public, which can rapidly reverse opioid overdose and is safe and effective for use in children."
Congratulations to Our Inaugural Science Slam Winners
CHOP and the University of Pennsylvania hosted the first annual Science Slam Competition to celebrate the 100th Anniversary of CHOP Research Institute. Winners received a monetary prize and were invited to share in person at the Science Slam Awards Ceremony and 100th Anniversary Celebration Lunch March 7. Michael Lauer, MD, deputy director for Extramural Research at the National Institutes of Health, was the keynote speaker for the event.
Congratulations to the inaugural Science Slam winners in their respective categories:
- Research Faculty/Postdoctoral Fellow Award: Jelte Kelchtermans, MD, pulmonology fellow at CHOP
- Predoctoral Graduate Student Award: Rebecca Somach, PhD candidate at Penn
- Research Staff Award: Michael McCoy, laboratory caretaker at CHOP.
Participants submitted a video that followed guidelines established by Three Minute Thesis (3MT®), designed to help researchers communicate the significance of their research clearly and concisely in a short time. Presenters could rely on a single, static slide with basic text to accompany a three-minute oral explanation of their research. Volunteer judges made their evaluations based on how well the projects addressed their theses while avoiding jargon, and how well the presenters conveyed their enthusiasm and significance of the research in the allotted time.
See a photo of the winners on @CHOP_Research. Learn more about CHOP's Science Slam and the Three Minute Thesis concept.
PCORI Funding Accelerates Research Into Clinical Practice
Clinical Futures, a CHOP Center of Emphasis, will lead the newly launched Health Systems Implementation Initiative (HSII) to encourage a more effective, direct transition of new research into healthcare practices and cut back on the estimated 17-year lag between publication of study results and their implementation. The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors committed $50 million to support the initiative, and CHOP is one of 42 participating health institutions selected to receive funding. Clinical Futures Director Alexander Fiks, MD, MSCE will head CHOP's participation in HSII.
"A main focus of Clinical Futures is in translating the best medical evidence into practice," Dr. Fiks said. "The PCORI Health Systems Implementation Initiative will provide support to realize this goal for the benefit of patients and families. This new initiative builds on prior successes we have achieved with PCORI funding in the area of antimicrobial stewardship, as well as ongoing work in the treatment of kidney stones and rheumatologic conditions in pediatric patients."
Investigators in Clinical Futures use innovative approaches to conduct prospective and retrospective clinical effectiveness research. This funding will help to develop applicable translation from research evidence to improved clinical outcomes for children.
Catch up on our headlines from our March 3 In the News:
- Sandra Amaral Joins the American Society of Clinical Investigation
- Vote for CHOP Biomedicine Studies in STAT Madness
- Jakob Seidlitz and Brain Chart Team Win Grand DataWorks! Prize
- Jolley and Pei Receive Single Ventricle Research Fund Grants
- Nurse Scientist Calls for Development of Early Interventions for Infants With Congenital Heart Disease
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