Penn State advances pediatric cancer research through new targeted therapy collaboration
Penn State College of Medicine is advancing its commitment to cutting-edge cancer research and early-phase clinical trials through a new collaboration aimed at accelerating a promising targeted therapy for pediatric brain[...]
Penn State’s Beat Childhood Cancer Research Consortium welcomes SJD Pediatric Cancer Center Barcelona, strengthening global leadership in pediatric cancer research
The Pediatric Cancer Center Barcelona (PCCB) at Hospital Sant Joan de Déu has become the first center outside North America to join the Beat Childhood Cancer (BCC) Research Consortium, an international pediatric cancer research[...]
Penn State College of Medicine, Beat Childhood Cancer Research Consortium and Four Diamonds launch clinical trial that aims to combat solid tumors
In a step toward fighting solid tumors in children and young adults, Penn State College of Medicine, the Beat Childhood Cancer Research Consortium and Four Diamonds, along with Senhwa Biosciences, Inc. are launching a pioneering clinical[...]
Q&A: Finding novel therapies for childhood cancer
Since the first time she treated a child with neuroblastoma, Giselle Saulnier Sholler wanted to do the impossible for her patients. Neuroblastoma is the most common extracranial solid tumor in children with[...]
FDA approves pediatric neuroblastoma drug based on Penn State professor’s work
Giselle Saulnier Sholler, professor of pediatrics and of pharmacology, reflects on 20 years working to change cancer outcomes for children HERSHEY, Pa. — In 2003, the first year of her[...]
US WorldMeds Announces FDA Approval of IWILFIN™ (eflornithine) to Strengthen Fight Against Aggressive Childhood Cancer
LOUISVILLE, Ky.--(BUSINESS WIRE)--USWM, LLC (US WorldMeds) today announced that the U.S. Food and Drug Administration (FDA) has approved IWILFIN™ (eflornithine) 192 mg tablets, a groundbreaking oral maintenance therapy for high-risk[...]
Penn State Health recruits leading researcher to fight pediatric cancer
Cris Collingwood//August 2, 2023 An internationally known physician-scientist and her research consortium have joined Penn State Health to continue efforts to eradicate pediatric cancer. Penn State Health and Penn State College of Medicine[...]
The Power of Will
Will Lacey was just a baby when doctors diagnosed a rare form of cancer and told his family there was only one end. Nobody then could imagine the journey ahead,[...]
Michigan Child Trials Target Genetic Roots of Cancers
Thank you Detroit News for featuring a story about the work we are doing. It wouldn’t be possible without the support of Dell, TGen Foundation and our generous community. Together,[...]
Research: Beat Childhood Cancer
The Beat Childhood Cancer Research Consortium is a group of 54+ universities and children's hospitals
Every day, they remind us why we do this work. Their laughter, their courage, their imaginations, their futures...this is what drives the Beat Childhood Cancer Research Consortium.
We fight for more moments, more milestones, and more tomorrows for every child and family facing a childhood cancer diagnosis.
#beatchildhoodcancer #ourwhy #pediatriccancerresearch #clinicaltrialssavelives #PennStateHealth
A new study demonstrating the potential of GB13 as an effective treatment strategy for diffuse intrinsic pontine glioma (DIPG) has been published in Nature Communications Biology.
In this study, GB13 was evaluated across multiple animal models of DIPG. When administered directly into tumors via convection-enhanced delivery (CED), GB13 significantly reduced tumor volume and improved overall survival. Importantly, the findings support a clinical strategy to enhance standard-of-care radiation therapy. Animals treated with GB13 prior to radiation exhibited smaller tumors and longer survival compared with radiation or GB13 treatment alone. Notably, CURATIVE outcomes were observed in a human-derived DIPG model.
We are excited to build upon this data to develop our BCC024 Clinical Trial focused on targeting GB13 in pediatric DIPG patients! We are working with both the FDA and the company, Targepeutics, to make this treatment a reality for our patients.
See the full article here: www.nature.com/articles/s42003-025-09155-9
If you missed it, check out the Little Warriors Foundation Webinar with Dr. David Loeb, the Study Chair of our upcoming BCC023 trial: A Phase II Open Label Basket Trial Study Using Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma. There are 5 separate cohorts covered under this master protocol to use DFMO in combination or in a maintenance setting.
We expect this trial to be available to sites across our BCC Consortium very soon! Check out more information here:https://clinicaltrials.gov/study/NCT07321912
www.littlewarrior.org/blog/dr-david-loeb-a-phase-ii-open-label-basket-trial-study-using-eflornith...
www.littlewarrior.org
Dr. David Loeb (Children's Hospital at Montefiore) makes a repeat guest appearance to present and discuss an upcoming Phase II Clinical Trial centered around DFMO for Ewing sarcoma and Osteosarcoma.
NEW PUBLICATION ALERT: On December 17, 2025, In vitro and in vivo efficacy of romidepsin alone and in addition to standard of care for treatment of Ewing sarcoma was published in Cancers.
HDAC inhibitors were tested on Ewing sarcoma cells in the lab and a drug called romidepsin was found to be most effective. In animal studies, the combination of romidepsin and ifosfamide/etoposide (IE) led to a significant decrease in tumor volume compared to that of IE alone. Thank you to our collaborators at Atrium Health in Charlotte, NC and the patients who dedicated both their samples and clinical data to make this work possible!
Read the full article: pubmed.ncbi.nlm.nih.gov/41463266/
Today we announced a new collaboration aimed at accelerating a promising targeted therapy for pediatric diffuse intrinsic pontine glioma (DIPG).
The collaboration will complete the final preclinical studies required to initiate a Phase I/II clinical trial of GB13, an IL13Rα2-directed immunotoxin designed to target tumor cells while sparing healthy brain tissue selectively. Unlike many first-in-human studies that begin with adult populations, the planned trial is designed to enroll children and adolescents, reflecting both the urgency of the disease and the absence of effective treatments.
“This work represents what is possible when academic medicine, philanthropy and industry align around a shared mission,” said Sil Lutkewitte, CEO of Targepeutics.
Read the full press release here: pennstatehealthnews.org/topics/penn-state-advances-pediatric-cancer-research-through-new-targeted...
Yesterday, a new Case Report was published in Frontiers Pharmacology by two of our summer interns (Elizabeth Wert and Leah Menachery), Dr. Smita Dandekar, Dr. Giselle Saulnier Sholler, BCC, Penn State College of Medicine and Penn State Health Children's Hospital teams titled: "Molecular Guided Therapy Leading to Exceptional Response in Relapsed Osteosarcoma".
In this publication we took a closer look at a 9-year-old girl with refractory metastatic osteoblastic osteosarcoma with disease progression treated on the Molecularly Guided Therapy Clinical Trial, NMTRC009 (NCT02162732), utilizing genomic analysis to identify novel treatment options. The report details the rationale behind the tumor board recommendations that resulted in a novel therapeutic approach which led to long term response and survival for this patient.
For more details on this patient, check out the publication at: doi.org/10.3389/fphar.2025.1719832
We continue to use this approach today for clinical decision making through our Molecular Tumor Boards (MTBs) for other patients through our Precision Medicine Program. We currently host 2 patients at this MTBs per week to try to identify novel therapies for other patients with relapsed or refractory solid tumors.