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MOMS2 Study Findings, Proof-of-Concept Award, Single Ventricle Heart Defects, Sarcoma Expert, Breaking Through Podcast

Published on January 31, 2020 in Cornerstone Blog · Last updated 5 months 3 weeks ago


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In the News


By Nancy McCann

Notable publications, big awards, and new faces on campus all add up to an exciting week here at Children’s Hospital of Philadelphia Research Institute. Learn about the lasting benefits of prenatal surgery, meet the CHOP doc who received the University City Science Center Proof-of-Concept Program Award, and find out which Center received a $1 million donation for innovative research. Let’s give a warm welcome to a world-renowned sarcoma expert joining our ranks, and make sure you take time to listen to CHOP President and CEO Madeline Bell’s newest podcast.

MOMS2 Research Reveals Fetal Surgery Benefits Continue to Childhood

Findings of a study coauthored by CHOP doctors will help to inform families with children who have spina bifida. Results of the “MOMS2: Follow-up of the Management of Myelomeningocele Study,” published in Pediatrics, show significant physical and emotional benefits in school-age children who received corrective surgery in the womb for MMC. 

Long-term gains included improved mobility and independent functioning in school age children. Walking unassisted, better gross and fine motor skills, improved bladder and bowel control, and fewer surgeries for shunt placement and revision were among the benefits. In addition, the children and families in the prenatal group reported less stress on the family overall, resulting in better quality of life.

“These findings add to the growing body of literature demonstrating the benefits of prenatal surgery for spina bifida,” said Dr. Adzick, a coauthor of the study and director of the Center for Fetal Diagnosis and Treatment. “Fetal surgery is a complex and serious procedure and should only be done by experienced teams. Research needs to continue to refine the technique in a way that will improve outcomes even further.”

Read this Cornerstone story to learn more about the fascinating study, and get to know, 6-year-old Audrey, who has benefited from this surgery. 

More information at CHOP News and The Philadelphia Inquirer.

CHOP Doc Receives Science Center Proof-of-Concept Program Award

The University City Science Center announced postdoctoral fellow Ian Henrich, PhD, was among three awardees in the latest round of its QED Proof-of-Concept Program. This program partners with regional academic and research institutions to prepare their most promising life science technologies for commercialization.

Dr. Henrich, lead investigator on the award, is a researcher in Margaret Chou, PhD’s, lab, where they are developing a novel therapy utilizing the USP6 protein to fire up the patient’s immune system against cancer cells.

“We will use nanoparticle technology to jumpstart the immune system,” Dr. Chou said. “Our nanoparticles are designed to specifically target acute myeloid leukemia cells, causing them to draw in and activate immune cells so that they can eradicate the cancer.” 

Not only does the research team envision that USP6 nanoparticles could act as an effective agent by itself, but they “also predict that it would greatly increase the number of patients who could respond to these other immunotherapies,” Dr. Henrich said.

For more information, see CHOP News and the Science Center press releases.

CHOP Awarded $1 Million for Single Ventricle Heart Defect Research

We are proud to announce CHOP is a recipient of the first Additional Ventures Innovation Funds, receiving $1 million to “accelerate scientific discoveries for the treatment of single ventricle heart defects (SVDs).” In collaboration with four other awardees, CHOP will focus on cutting-edge research to understand better the root causes of SVDs.

“This joint initiative will fuel breakthrough research that will meaningfully improve the lives of children born with single ventricle heart defects,” said Bryan Wolf, MD, PhD, Chief Scientific Officer at CHOP Research Institute. “We look forward to collaborating with these other institutions and continuing the cutting-edge research that has put our Cardiac Center at the forefront of pediatric cardiac care.”

Learn more at CHOP News.

CHOP Welcomes Patrick Grohar, MD, PhD, World Renowned Sarcoma Expert

A warm welcome goes out to one of the newest members of the Research Institute family: Patrick Grohar, MD, PhD. Dr. Grohar is an attending physician with the Cancer Center and the Director of Translational Research with the Center for Childhood Cancer Research at CHOP. His research focuses on developing therapies for pediatric sarcomas, particularly Ewing sarcoma, a form of cancer that is rare in adults but relatively common in the pediatric population. It most often occurs in the pelvis and less commonly in the long bones of the arms and legs.

For sarcoma patients, the current therapeutic approach of chemotherapy, radiation, and surgery is effective in many patients, but less so in those whose cancer has spread or relapsed. New therapies are needed for this patient population, and Dr. Grohar is optimistic about the potential of bench-to-bedside research conducted in synergy with Penn Medicine’s Abramson Cancer Center. 

“In today’s day and age, I don’t think that changing outcomes for patients is unrealistic,” Dr. Grohar said. “We’ve made progress. We have some exciting data in preclinical models. It’s all about serving the patients, and coming to CHOP felt like an opportunity to make an impact.”

Get to know more about Dr. Grohar at CHOP News.

Have You Heard CEO Madeline Bell’s Latest Podcast?

Well just in case you missed it, we’ve got the link right here. In this, the 17th episode of “Breaking Through,” released Jan. 24, Bell chats with Beverly Davidson, PhD, director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics and Chief Scientific Strategy Officer at CHOP Research Institute. Dr. Davidson is developing innovative gene therapies to treat inherited brain disorders, such as Alzheimer’s. Listen in, and learn about Dr. Davidson’s breakthrough moments during each decade of her career, what it’s like to be a woman in a male-dominated field, and what advice she has for aspiring scientists. 


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

  • Researchers Find Genetic Difference Helps Distinguish Diabetes Type 
  • New Study Highlights Human Factors in Self-Driving  
  • Identifying Glomerular Disease for Clinical Research With EHR-based Algorithm 
  • Philadelphia Inquirer Features Novel CHOP Ewing Sarcoma Research Genomics 

That’s it for this briefing! Keep up with our news, stories, and updates in real time by following us on Twitter, Facebook, LinkedIn, or Instagram. Or subscribe to our newsletter to get an email sent every other Friday by signing up here.