ROP Screening Tool, Sickle Cell Disease Target, PolicyLab Keynote, 2018 St. Baldrick's Grants

It’s still a month before teachers and students are officially back to school, but here at Children’s Hospital of Philadelphia Research Institute, you can learn something new every day. In this edition of our biweekly news roundup, discover the latest findings from our ophthalmologists on how clinicians should choose to screen premature babies for a potentially blinding eye disorder, find out how CRISPR-based technology allowed scientists to reveal insights into sickle cell disease, and prepare for an educational and exciting speech from the recently announced keynote speaker at PolicyLab’s upcoming 10th Anniversary Forum

Ophthalmology Researchers Develop ROP Screening Tool

In a multi-hospital collaboration led by our own Gil Binenbaum, MD, MSCE, pediatric ophthalmologist, researchers have developed a novel screening tool for retinopathy of prematurity (ROP), a potentially blinding disorder that can occur in premature babies whose retinal blood vessels grow in the wrong direction.

The new tool offers clinicians a simple way of determining which babies born premature should be screened for ROP. By doing so, it can ease the burden on babies, parents, nurses, and doctors by reducing the number of ROP eye exams conducted. The tool is based on six key criteria: birth weight below 1,051 grams, gestational age younger than 28 weeks, hydrocephalus, and slow weight gain during three specific time periods between ages 10 and 40 days.

“The criteria we developed were highly sensitive; in fact, they were slightly more sensitive that the current screening guidelines, and yet they were much more accurate than the current guidelines,” Dr. Binenbaum said. “Using these modified screening criteria could potentially reduce the number of babies who need to be examined by almost a third, which would be beneficial for those babies, and allow us to focus all our efforts on treating the babies who are at high risk for retinal detachment and blindness.”

The findings were published July 12 in JAMA Ophthalmology.

Read more in the press release, and learn more about how Dr. Binenbaum and his team at CHOP are working to improve ROP diagnoses for newborns in this Bench to Bedside.

Scientists Identify Possible Sickle Cell Disease Drug Target

A team of CHOP and University of Pennsylvania scientists described another breakthrough in the research and treatment of sickle cell disease (SCD), an inherited blood disorder that affects red blood cells. Using a screening tool based on CRISPR gene editing-technology, the researchers identified a key signaling protein, known as HRI, that regulates the production of hemoglobin, the iron-carrying component of red blood cells.

In human culture cell experiments, the team found that blocking HRI reduced the characteristic sickling that distorts the shape of red blood cells in SCD. Furthermore, losing the function of HRI appeared to be well-tolerated, as the researchers were able to decrease sickling in red blood cells obtained from SCD patients without impairing either the viability or maturation of the cells. The discovery offers a potential target for innovative drugs to treat SCD.

“We have found a protein with activity specifically in red blood cells that could be a ‘druggable’ target, possibly with a small molecule — a pill that patients could take to treat sickle cell disease,” said study co-leader Gerd A. Blobel, MD, PhD, scientist at CHOP. “Our long-term goal is to carry out follow-up studies to evaluate whether this approach improves clinical outcomes in patients.”

Dr. Blobel and his study co-leader, Junwei Shi, PhD, a scientist at Penn, published their findings in the journal Science.

Learn more about the study in the press release.

U.S. Department of Defense Assistant Secretary Will Speak at PolicyLab Forum

PolicyLab, a Center of Emphasis at the Research Institute, announced that Stephanie Barna of the U.S. Department of Defense (DoD) will speak as the keynote for the center’s upcoming 10^thAnniversary Forum. Barna is Acting Assistant Secretary of Defense for Manpower and Reserve Affairs at the DoD. Held at the American Museum of National Jewish History Oct. 22, the “Charting New Frontiers in Children’s Health Policy and Practice Forum” is a full-day event. National and regional leaders will gather to discuss a variety of topics related to advancing child, adolescent, and family health and well-being, including achieving health equity for youth, transforming juvenile justice for better outcomes, and much more. (See the full agenda here.)

Learn more about the symposium and register through the PolicyLab website. Don’t miss a guest blog post about PolicyLab’s anniversary from Director David Rubin, MD, MSCE, and Deputy Director Meredith Matone, DrPH, MHS, on Cornerstone.

Cancer Researchers Awarded 2018 St. Baldrick’s Grants

St. Baldrick’s Foundation has announced a new batch of research grant recipients — including a handful of scientists from our Cancer Center — in what the organization named its “biggest grant cycle of 2018.” Within this cycle, St. Baldrick’s will award a total of $19.1 million to researchers and institutions committed to improving outcomes for children with cancer. We are pleased to announce Michael Leibowitz, MD; Jennifer Kalish, MD, PhD; Garrett Brodeur, MD; and Sogol Moustofi-Moab, MD, MSCE, of our Cancer Center, received 2018 grants:

• As a St. Baldrick’s Scholar, Dr. Leibwoitz will focus his research project on advancing cellular immunotherapy to become an effective and viable treatment option for pediatric solid tumors.
• Also as a St. Baldrick’s Scholar, Dr. Kalish will continue to study how genetic causes of Beckwith-Wiedemann Syndrome lead to cancer.
• Dr. Brodeur’s research aims to develop more effective but less toxic therapy for childhood cancer using nanomedicine-based drug delivery, a method developed by Dr. Brodeur and his team to deliver more drug to the tumor and much less to the patient’s healthy organs.
• Through a Supportive Care Research Grant, Dr. Moustofi-Moab is, for the first time, studying the effect of cancer treatment (radiation vs. chemotherapy) on making insulin and breaking down blood sugar in childhood cancer survivors. Her research addresses the fact that the majority of pediatric cancer survivors are diagnosed with chronic health problems including diabetes, hypertension, and heart disease within 30 years of cancer diagnosis and treatment.

Learn more about St. Baldrick’s Foundation 2018 grants here.

ICYMI

Recently on Cornerstone, we shared a guest blog from Lindsey Waqar, MPH, CCRC, lead clinical research coordinator for the Division of Rheumatology, wrapped up a few takeaways from the “Personalized IBD and VEO-IBD: Genomics, Microbiome, Biologics, and Beyond” symposium, and explored exciting new research from our Division of Nephrology in regards to pediatric chronic kidney disease.

Catch up on our headlines from our July 13 edition of In the News:

• Dr. Richard Aplenc Receives ALSF Epidemiology Grant
• New Software Tool May Streamline Genetic Diagnoses
• National Guidelines Recommend Honey After Swallowing Button Battery
• Save the Date: PolicyLab 10^thAnniversary Forum
• CIRP Study Offers New Insights Into Parental Cell Phone Use While Driving