In the News: For the Love of Grace Foundation, Hemophilia Research in the NYT, Microbiome Symposium, Baby Monitors, 2018 Parkway Run

Aug 24 2018

In the News: For the Love of Grace Foundation, Hemophilia Research in the NYT, Microbiome Symposium, Baby Monitors, 2018 Parkway Run

Microbes, monitors, and miraculous medicine converge in this week’s roundup of research-related headlines at Children’s Hospital of Philadelphia. Keep reading to learn more about new concerns for commercially available baby monitors reported by our researchers, one family’s inspirational story of resilience and recovery after their baby daughter battled a brain tumor, and exciting upcoming events in the CHOP community, including the fifth annual symposium for microbiome research.

For the Love of Grace Donates $50,000 to CHOP

A rare disease foundation created by a family whose baby daughter defied cancer presented a $50,000 donation toward brain tumor research at CHOP last week. For the Love of Grace was founded by Kristen and Joey Masciantonio after their now 3-year-old daughter, Gianna, battled a tumor on her brain stem — a fight that she and her family have struggled with since birth. When Gianna was born, the Masciantonios were told that the size and location of her tumor indicated an aggressive cancer with no surgical options, leaving their daughter only weeks to live. But at five months, Gianna received a new diagnosis of Systematic Juvenile Xanthogranuloma, a rare blood disease caused by an excess of white blood cells that cluster and damage major organs. Gianna began chemotherapy and rounds of surgeries with the team at CHOP, including Amish Shah, MD, PhD, neuro-oncologist, and Phillip “Jay” Storm, MD, chief of the Division of Neurosurgery. Today, Gianna is preparing to start preschool.

The Masciantonio’s story has made headlines for Gianna’s recovery, the foundation’s generous donation to CHOP, and the family’s remarkable story of faith: Near the beginning of their struggle three years ago, Pope Francis kissed 1-year-old Gianna on the head during his Philadelphia visit. In CBS Philly coverage of this story, Kristen and Joey say that “the doctors at CHOP, with a touch of faith from Pope Francis three years ago, saved Gianna’s life.

CBS Philly and learn Gianna’s storyon the foundation’s website.

New York Times Features Breakthrough Hemophilia B Research

The New York Times recently featured a novel treatment for hemophilia B developed by researchers in our Division of Hematology in conjunction with the biotech company, Spark Therapeutics, and published in the New England Journal of Medicine. Alongside her team, Lindsey George, MD, attending physician at CHOP and first author of the paper, is leading phase III trials on the innovative one-time therapy, currently named SPK-9001.

SPK-9001 utilizes gene therapy to deliver the factor IX gene — the missing clotting factor in hemophilia B — so that a patient’s body can safely produce sustained levels of FIX rather than receive repeated infusions of the clotting factor, and prevent debilitating bleeding episodes. The therapy could be a game-changer for patients with hemophilia B if it continues to prove safe and effective in a larger population: Patients would have the option of a one-time treatment, instead of repeated infusions of FIX, to either prevent or control bleeding episodes.

Read the full NYT article online and learn more about the therapy in the CHOP press release.

Fifth-Annual Penn-CHOP Microbiome Symposium Features Exciting Speaker Lineup

Save the date: This fall on Nov. 7 and 8, the Penn-CHOP Microbiome Program and the Penn-Vet Center for Host-Microbial Interactions will host the fifth annual Microbiome Symposium, titled “Microbes, Metabolomics, and Modern Diseases.” The event will feature a talk by Carl Zimmer, the award-winning New York Times columnist, science writer, and author of 13 books about science, followed by an exciting speaker lineup of microbiome researchers from CHOP, Penn, the University of California at San Diego, and Woods Hole. To get excited for the upcoming symposium, we invite you to check out some of the Penn-CHOP Microbiome Program’s most recent research endeavors, from leveraging the immune system to mine for microbes that prevent autoimmune disease, to studying the role of host-microbiota interactions in inflammation, on Cornerstone!

Learn more here.

Researchers Report Concerns About Physiological Baby Monitors

Two commercially available baby monitors designed to measure vital signs like oxygen levels and pulse rates showed inconsistent and worrisome results, according to new research from researchers at CHOP and published in the Journal of the American Medical Association (JAMA). Chris Bonafide, MD, MSCE, attending physician at CHOP, led the study with colleagues from CHOP as well as the ECRI Institute, a nonprofit research organization that evaluates medical devices and practices. In their study of 30 infants using the Owlet Smart Sock2 and Baby Vida monitors, the team reported that one monitor detected low oxygen levels in an inconsistent fashion while the other did not detect them at all. The FDA does not regulate these monitors, and the researchers believe their use may result in undue anxiety for parents, no medical health benefits, and unintended consequences.

Last January, researchers described this concern about consumer use of physiological baby monitors in a JAMAeditorial. “We previously discussed the consensus in the pediatric community that there is no medical reason to electronically monitor vital signs in healthy babies at home,” said Elizabeth Foglia, MD, MSCE, co-author of the paper and attending neonatologist at CHOP. “Our new study adds serious concerns about the accuracy of these consumer monitors, when we compared them to a standardized hospital monitor in a cohort of sick infants.”

Read more in the press release.

Help Fuel Childhood Cancer Research at CHOP at Parkway Run

Lace up your sneakers because it’s that time again: The Annual Parkway Run and Walk to raise money for pediatric cancer research and treatment will be held Sept. 30 this year! One hundred percent of every donation received will go toward breakthrough research and care for children treated at CHOP. Last year, the fun-filled event raised over $1.1 million, helping to accelerate the development of new therapies for pediatric cancer and address the long-term effects of the disease. You can register for the run or donate online today.

ICYMI

Recently on Cornerstone, we took a quick snapshot of new injury research on how parents are using cell phones while driving in cars with their children. We also covered a recent study from CHOP and Penn researchers into local workplace regulations to protect breastfeeding across the U.S.

Catch up on our headlines from our Aug. 10 edition of In the News:

  • 2018 Foerderer Grants Drive Novel CHOP Research
  • CHOP Immunologist and Patient Family on Megyn Kelly Today
  • Dr. Marni Falk Weighs in on New Mitochondrial Research
  • Pediatric Device Consortium Awards New Seed Grants
  • Breaking Through with Madeline Bell

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