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CAR-T Cell Approval, Hope on Wheels, Task Force on Pregnant Women, Taking Flight for Autism, NFL Concussion Research, Doug Wallace

Published on September 8, 2017 in Cornerstone Blog · Last updated 4 months ago


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September marks National Childhood Cancer Awareness Month, and this year at Children’s Hospital of Philadelphia, we kick-started the commemorative period on the heels of exciting news about breakthroughs in pediatric cancer immunotherapy research. Oncology investigators at CHOP also got a big boost in research funding from Hyundai’s nonprofit organization, Hope on Wheels. And that’s only the beginning: Since September marks the return of the football season, we’re thrilled to share the latest headlines on how the National Football League (NFL) is helping to drive concussion research.

FDA Approves World’s First CAR-T Cell Therapy

In a truly historic moment of the childhood cancer research timeline, the U.S. Food and Drug Administration announced their approval of the world’s first chimeric antigen receptor (CAR)-T cell therapy last week. Targeted for pediatric and young adult patients with aggressive forms of leukemia unresponsive to traditional treatment, the cancer immunotherapy drug will be manufactured by Novartis Pharmaceuticals under the official name, Kymriah.

As most of our followers know, CAR-T cell therapy began its story for children with cancer in 2012 right here at CHOP and the University of Pennsylvania. Our investigators worked with the Perelman School of Medicine to conduct experimental studies of the drug in pediatric patients with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. In those early-stage clinical trials, more than 90 percent of patients achieved complete remission. Five years later, we’re brimming with stories of children with ALL who received the investigational immunotherapy (including the very first pediatric patient to be treated, Emily Whitehead) and now live cancer-free. We’re filled with pride for the researchers who made the approval possible, including Stephan Grupp, MD, director of our Cancer Immunotherapy Program; Shannon Maude, MD, attending physician in our Cancer Center at CHOP; Carl June, MD, director of the Center for Cellular Immunotherapies at Penn; and David Porter, MD, Jodi Fisher Horowitz Professor in Leukemia Care Excellence at Penn.

Learn more about the FDA approval in the CHOP press release.

Hyundai Gives CHOP Research Hope on Wheels

New grants from Hyundai Motor America’s nonprofit organization, Hope on Wheels, will allow oncology researchers to make even more breakthroughs in pediatric cancer. The organization announced an $8.5 million contribution toward cancer research in the form of 40 grants to honor National Childhood Cancer Awareness month. Throughout September, Hope on Wheels will present these grants to children’s hospitals across the U.S., and we are excited to share that CHOP investigators have received a Hyundai Scholar Hope grant and a Hyundai Young Investigator grant. The grants were designated to fund peer-reviewed, U.S.-based research projects led by investigators at Children’s Oncology Group institutions. The grants span two years, and projects will focus on developing new therapies and treatments or translational research for childhood cancer.

Read more about the cancer commitment from Hope on Wheels in the Hyundai press release.

Philly Eagles Take Flight for Autism Research

Autism continues to rank as the largest and fastest-growing developmental disability in the world, but autism researchers who want to change that momentum have a special member on their team: the Philadelphia Eagles. The Eagles held their second annual “Taking Flight for Autism” event Aug. 30 at Lincoln Financial Field in Philadelphia. The event raises money for autism support programs and the Center for Autism Research (CAR) at CHOP. More than 600 guests mingled with Eagles players, coaches, and executives for a cocktail hour, dinner, conversation, and remarks from Jeffrey Lurie, Eagles chairman/CEO; Doug Pederson, Eagles head coach, and more. Lurie and the Eagles Charitable Foundation announced that in total since 2016, the event had raised $1.6 million for CHOP.

With the generous funding and warm team spirit provided by the Eagles, CAR at CHOP will continue to research biomarkers for autism, develop and test new therapies, and improve the quality of life for children, adolescents, and adults with autism.

Dr. Arbogast Leads NFL Engineering Roadmap for Concussion Research

Last year, the NFL pledged $100 million toward concussion research in what Roger Goodell, NFL Commissioner, remarked as a mission to “look at anything and everything to protect our players and make the game safer.” In a news brief last week, the Associated Press (AP) reported on the progress of that pledge, including developments in one of the NFL’s biggest initiatives co-led by Kristy Arbogast, PhD, co-scientific director of the Center for Injury Research and Prevention at CHOP.

As an NFL Players Association engineering consultant, Dr. Arbogast manages the “Engineering Roadmap,” a $60 million program designed to improve head protection equipment, alongside Barry Myers, MD, director of innovation at Duke University’s Clinical and Translational Science Institute. We spoke with Dr. Arbogast on the details of her research game plan in our April/May issue of Bench to Bedside, including the development of a “sensor package” that would allow researchers to measure directly and accurately what a player’s head is doing during reconstructions of concussions cases that have occurred in the NFL.

“A key component of the engineering roadmap is to accurately measure the motion and acceleration the head experiences during play in the NFL by player position, to give design direction for protective equipment,” remarked Dr. Arbogast in the AP report. “To date, we have been doing that via video reconstructions and injury event recreations using crash test dummies. These approaches are incredibly time intensive and, by design, focus on ‘events’ that must be subjectively selected from game film or injury reports.”

Learn more about how Dr. Arbogast and her colleagues are working toward making football safer on Bench to Bedside.

Diane Spatz Joins Congressional Task Force

CHOP is home to many brilliant nurse researchers who lead their respective fields in improving care for our patients and their families. One of those nurse researchers, Diane Spatz, PhD, RN-BC, FAAN, also director of the Lactation Program at CHOP, has been selected to join the Congressional Task Force on Research Specific to Pregnant and Lactating Women. As one of the few nurses selected to join the Task Force, Dr. Spatz will help to fill the gaps in knowledge and research about safe therapies for pregnant and breastfeeding mothers. The Task Force is part of the 21st Century Cures Act and includes leaders from the National Institutes of Health, the Office of Women’s Health, and the Centers for Disease Control and Prevention.

At the first task force meeting held at the NIH Aug. 21 and 22, Dr. Spatz highlighted the need for all women to have access to evidence-based information about medications during breastfeeding and pregnancy, as well as the important role nurses play in delivering accurate information to mothers.

“The lack of consistent evidence-based information on the safety of medications for pregnant women and mothers who are breastfeeding negatively impacts mothers every day,” Dr. Spatz said. “This congressional task force is a historic opportunity to improve the care of women and infants in the United States as well as globally.”

Learn more in the press release.

Doug Wallace Receives Dr. Paul Janssen Award at Symposium

In June, we received the wonderful news that Douglas Wallace, PhD, director of the Center for Mitochondrial and Epigenomic Medicine at CHOP, would receive the Dr. Paul Janssen Award for Biomedical Research – a prestigious title that honors the legacy of Dr. Paul Janssen, one of the most impactful pharmaceutical scientists of the 20th century and the founder of Janssen Pharmaceuticals (now part of the Johnson & Johnson Family of Companies). Dr. Wallace is set to receive the high-profile award Sept. 13 at the 2017 Dr. Paul Janssen Award Symposium to be held at the New York Academy of Sciences in downtown New York City. The award honors Dr. Wallace’s groundbreaking work that established the field of mitochondrial medicine. He has focused his scientific career on the role that mitochondria, our body’s tiniest batteries, play in human evolution and disease. In a Cornerstone article about the award published in June, Dr. Wallace discussed the significance of the honor, as it recognizes the importance of bioenergetics in health and disease.

“It is my hope that this validation will encourage innovative young physicians and scientists to apply the principles of mitochondrial genetics and bioenergetics to the pressing clinical problems that threaten global health,” Dr. Wallace said in the story. “Such efforts promise to have a profound effect on the well-being of all peoples.”

The 2017 Dr. Paul Janssen Symposium has another special CHOP guest making headlines at the event: Our own Chief Scientific Strategy Officer Beverly Davidson, PhD, who also is director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at CHOP, will speak on “Emerging Gene Therapies for Inherited Disorders.”

Read about Dr. Wallace’s latest research findings about the role of mitochondria in autism on Cornerstone.


Recently on Cornerstone, we met the three hemophilia researchers at CHOP who received grants as part of the Bayer Pharmaceuticals Awards Program, heard important words from Bryan Wolf, MD, PhD, our Chief Scientific Officer, about the FDA’s approval of the world’s first cancer immunotherapy, took a quick snapshot of the latest findings about autism and challenging behaviors from CAR at CHOP, and learned what the Spring 2017 recipients of Clinical Futures Pilot Grants are doing with their funding.

Catch up on our headlines from our Aug. 25 edition of In the News:

  • d3b Leads Data Collaboration for Cancer and Structural Birth Defects
  • Be Well Philly Names Dr. Senbagam Virudachalam a Semifinalist in ‘Health Hero’ Challenge
  • CHOP Researchers Talk Sports Concussion Prevention and Research on Air
  • Do Coping Interventions for Parents and Hospitalized Children Improve Outcomes?
  • Madeline Bell Named One of Modern Healthcare’s ‘100 Most Influential’

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