Assorted News from the Last Week: AWS is providing $10 million to accelerate research for pediatric health care and to better understand rare diseases, including $1 million to the Children's Brain Tumor Network. Novartis announced that the U.S. Food and Drug Administration (FDA) approved Lutathera for the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs) for pediatric patients. JAMA Pediatrics explores the important role that patient advocates play in pediatric oncology. Abstract (will post full article when available). A Children's Oncology Group study concluded that adding the mTOR inhibitor temsirolimus (Torisel) to chemotherapy in the first-line setting failed to improve event-free survival [...] Read more

Assorted News from the Last Week: CAC2 Supporting Organization Member Day One Biopharmaceuticals announced it has entered into an exclusive licensing agreement with MabCare Therapeutics for MTX-13, a novel antibody drug conjugate (ADC) targeting protein-tyrosine kinase 7 (PTK7) which is overexpressed in in neuroblastoma, rhabdomyosarcoma and osteosarcoma in children, as well as esophageal, ovarian, lung and endometrial cancers among adults. Blinatumomab (Blincyto; Amgen) received FDA approval for the treatment of adult and pediatric patients with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (B-ALL). FDA grants rare paediatric disease designation to Race Oncology's RC220 bisantrene for the treatment of pediatric [...] Read more

Assorted News from the Last Week: Bristol Myers Squibb's Augtyro (repotrectinib) has secured an accelerated approval from the FDA for the treatment of adult and paediatric patients aged 12 years and older with solid tumours harbouring NTRK gene fusions. Researchers found that DIPG tumors' metabolism differs from adult brain tumors, a key to understanding treatment resistance in this aggressive form of pediatric brain cancer. A discussion of the question: "Is it appropriate to enroll children and adolescents in end-of-life clinical studies?" A virtual peer support program created at the Hospital for Sick Children (SickKids) has shown significant potential in helping [...] Read more

Assorted News from the Last Week: Research from the University of Birmingham could pave the way for using MRI scanning combined with machine learning to assess medulloblastomas for their 'signature' metabolic profiles without the need for invasive biopsy and could rapidly reduce the current 3-4 week wait from presentation to full diagnosis. CAC2 Student Member Joshua Omale blog post: Empowering Communities: Driving Childhood Cancer Awareness from the Grassroots Update on Cancer Predisposition Syndromes and surveillance guidelines for childhood brain tumors Results from the first 384 participants of the Australian Zero Childhood Cancer national precision medicine program (ZERO) show a precision [...] Read more

Assorted News from the Last Week: The FDA granted accelerated approval to Eli Lilly And Co’s selpercatinib (Retevmo) for pediatric patients two years of age and older with the following: Advanced or metastatic medullary thyroid cancer (MTC) with a RETmutation, who require systemic therapy. Advanced or metastatic thyroid cancer with a RET gene fusion, who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). Locally advanced or metastatic solid tumors with a RET gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options. Three blog posts by CAC2 Members: Perhaps one [...] Read more

Assorted News from the Last Week: The FDA Pediatric ODAC met to "discuss perspectives on the impact of FDARA on the field of pediatric oncology, considerations related to implementation of FDARA for new molecularly targeted drugs and biological products, and the role of proof-of-concept studies using relevant pediatric preclinical models." There was also a discussion of the role of international collaboration and how coordinated approaches to the design and conduct of molecularly targeted pediatric cancer investigations can be best achieved in light of RACE Act regulations. Click here to view all the meeting information and links to key documents and [...] Read more