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Assorted News from the Last Week: CAC2 Supporting Member Day One Biopharmaceutical and Ipsen unveiled an ex-U.S. licensing agreement for tovorafenib (approved as Ojemda in the U.S.) in pediatric low-grade glioma (pLGG) and "any future indications developed by Day One" outside the U.S. Several CAC2 Members contributed to the recently published manuscript that was produced by the ACCELERATE Paediatric Strategy Forum for Medicinal Product Development of PI3 K-inhibitors. The FDA has accepted the resubmission of a biologics license application (BLA) seeking the approval of remestemcel-L (Ryoncil) for the treatment of pediatric patients with steroid-refractory acute graft-vs-host-disease (SR-aGVHD). It Mattered Later: [...] Read more

Assorted News from the Last Week: Congratulations to CAC2 Supporting Organization Day One Biopharmaceuticals and CAC2 member authors and reviewers who contributed to the white paper “Advancing Pediatric Cancer Research and Drug Development through Multi-Stakeholder Collaboration: A Framework Based on the Tovorafenib Journey.” Read about the collaborative effort that made this paper possible on the CAC2 Collaborative Achievement Blog. More frequent use of genetic testing in pediatric oncology, along with the application of advanced testing methods, holds great promise for identifying patients with CPS as well as detecting somatic variants that inform diagnosis, prognosis, and treatment. The FDA has granted [...] Read more

Assorted News from the Last Week: Psychological and ethical issues raised by genomic testing in paediatric care pathway, a qualitative analysis with parents and childhood cancer patients. Immuno-oncologic profiling of pediatric brain tumors reveals major clinical significance of the tumor immune microenvironment. CAR-T therapy, which harnesses a person’s own immune cells, racks up some astonishing success stories against deadly brain tumours in children. Researchers has identified and located a population of stem-like cells that initiates and maintains Group 3 medulloblastoma (Gr3-MB) in the developing brain. They showed that eliminating the small population of stem-like cells present in Gr3-MB tumors led [...] Read more

Assorted News from the Last Week: Building a case for incorporating patient advocates throughout the research continuum in pediatric oncology. The NCI Council of Research Advocates met virtually on June 26 and heard the latest legislative report, with updates on appropriations, cancer-related legislation, and other ways that NCI is engaging and advocating. An article in support of the Creating Hope Reauthorization Act: "The rare pediatric disease voucher program creates new treatments. I have new data to prove it." Benefits for children with suspected cancer from routine whole-genome sequencing. Upcoming Webinars, Online Opportunities, and Meetings: 56th Congress of the International Society [...] Read more

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