Researchers from the Children’s Hospital of Philadelphia (CHOP) in collaboration with the School of Veterinary Medicine at the University of Pennsylvania, Perelman School of Medicine at the University of Pennsylvania and Vanderbilt University Medical Center found that venous endothelial cells (VECs), a specific type of lung blood vessel cell, can help fix damaged blood vessels in the lungs following lung injury or disease. Their findings were recently published in Nature Cardiovascular Research.

Researchers at Children’s Hospital of Philadelphia (CHOP) and the Children’s Oncology Group (COG) found that larotrectinib, an oral drug that stops cancer-cell growth, was highly effective in pediatric patients with newly diagnosed infantile fibrosarcoma (IFS) or other solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation. The study, published today in the Journal of Clinical Oncology, is the first COG trial to test precision medicine in a front-line setting across all different types of solid tumors based on a genetic biomarker rather than histology. It is poised to redefine the approach to treatment for newly diagnosed fibrosarcoma and other solid tumors with the NTRK gene fusion, potentially reducing or eliminating the need for these patients to receive chemotherapy.

Researchers at Children’s Hospital of Philadelphia (CHOP) and the Children’s Oncology Group (COG) announced the results of a Phase 3 study that demonstrated adding the bi-specific T-cell engager, blinatumomab, to chemotherapy for newly diagnosed National Cancer Institute (NCI) standard risk (SR) B-cell acute lymphoblastic leukemia (B-ALL) pediatric patients significantly improves survival outcomes. The results were published today in the New England Journal of Medicine and will be presented at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition on December 8.

Study identified a drug for potential use in future precision medicine clinical trials

Researchers at Children’s Hospital of Philadelphia (CHOP) found that most patients and their families who reported experiencing food insecurity benefitted from consistent delivery of fresh produce and prepared healthy meals post-hospitalization. The study, recently published in Pediatrics, underscores the need for hospital systems to partner with community-based organizations to address food insecurity throughout the United States.

Researchers at Children’s Hospital of Philadelphia (CHOP) announced the creation of a new AI technology called CelloType, a comprehensive model designed to more accurately identify and classify cells in high-content tissue images. The findings were published today in the journal Nature Methods.

Researchers at Children’s Hospital of Philadelphia (CHOP) announced the launch of the initial phase of DISCOVERY of Risk Factors for Type 2 Diabetes in Youth, a nationwide National Institute of Health (NIH) study to address the surge in youth diagnosed with type 2 diabetes over the past two decades.

Researchers at Children’s Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy trial for Danon disease (DD), a rare, X-linked heart condition caused by a single gene mutation. The data on the results of the RP-A501 Phase 1 trial, presented at a late breaking session today at the American Heart Association Scientific Sessions 2024 in Chicago, were also published in the New England Journal of Medicine.

Researchers at Children’s Hospital of Philadelphia (CHOP), St. Jude Children’s Research Hospital (St. Jude) and the Children’s Oncology Group (COG) today announced a significant paradigm shift in the understanding of T-lineage acute lymphoblastic leukemia (T-ALL), an aggressive and high-risk form of cancer, to one frequently driven by genetic changes in non-coding portions of our DNA. The collaborative study, supported by the Gabriella Miller Kids First Pediatric Research Program (Kids First) and National Institutes of Health (NIH) Common Fund, was published today in the journal Nature.

Improvements needed to better guide policy and best practice recommendations.