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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

Assorted News from the Last Week: St. Jude survivorship portal and its ability to facilitate breakthroughs in pediatric cancer survivorship research were published recently in Cancer Discovery. The portal integrates data involving whole genomic sequencing, treatment exposure and outcomes — creating an unprecedented research system that houses 400 million genetic variants from over 7,700 childhood cancer survivors. In an effort to raise awareness about pediatric brain cancer, encourage research on diffuse intrinsic pontine glioma (DIPG) tumors and other pediatric cancers, and honor patients and families who have faced this disease, the U.S. Senate passed a bipartisan resolution (S.Res. 691) designating May [...] Read more

Assorted News from the Last Week: To understand how CNS tumors impact the AYA population, NCI-CONNECT (Comprehensive Oncology Network Evaluating Rare CNS Tumors), the Central Brain Tumor Registry of the United States (CBTRUS), and the American Brain Tumor Association (ABTA) have partnered on a new statistical report that explores statistical trends in AYAs with primary CNS tumors in the United States between 2016 and 2020. (NCI) Among adults previously treated for a childhood cancer who attained full-time employment 25 years following successful treatment, nearly 30% to 40% either died, became unemployed, or moved to part-time employment or left the labor force [...] Read more

Assorted News from the Last Week: Brain cancer in children is notoriously hard to treat – a new mRNA cancer vaccine triggers an attack from within. In an analysis from the Children’s Oncology Group phase III AALL1131 study reported in the Journal of Clinical Oncology, Alexander et al found that exposure to propofol was associated with an increased risk of impairment in reaction time/processing speed at 1 year after treatment in children with high-risk B-cell acute lymphoblastic leukemia (B-ALL). While overall cancer risk was no different for children born after fertility treatments compared with those naturally conceived, frozen or fresh [...] Read more

Assorted News from the Last Week: Congratulations to CAC2 Supporting Organization Day One Biopharmaceuticals.  The FDA has granted accelerated approval to tovorafenib (Ojemda) for the treatment of pediatric patients 6 months of age and older with relapsed or refractory low-grade glioma harboring a BRAF fusion or rearrangement, or a BRAF V600 mutation and marks the first systemic therapy indicated for pediatric patients with low-grade glioma harboring BRAF rearrangements, including fusions.  There are a ton of articles about this approval.  This OncLive piece was the first in. The FDA approved lutetium Lu 177 dotatate for the treatment of pediatric patients aged 12 [...] Read more

Assorted News from the Last Week: To achieve successful socioeconomic reintegration in the long term, it is crucial to implement a childhood cancer survivorship program that addresses the unique needs of individuals based on their disease and cultural background throughout their entire lives. Data sharing platforms for precision care can revolutionize the way we treat patients and advance treatments and cures for pediatric brain cancer. Following a progressive expansion, the ZERO Childhood Cancer program is now available to all children with cancer in Australia. This means that every child, regardless of their cancer type or risk profile, will now have [...] Read more

Assorted News from the Last Week: The UK's National Institute for Health and Care Excellence (NICE) recommended Novartis' autologous CAR T-cell therapy Kymriah (tisagenlecleucel) for children and young adults with previously treated B-cell acute lymphoblastic leukemia (ALL). CAC2 Supporting Member Oncoheroes Biosciences has been chosen as one of the eleven finalists for the inaugural J.P. Morgan Asset Management: Life Sciences Innovation Summit. Scheduled for May 14th–15th, 2024, at the Cleveland Clinic Abu Dhabi, this event aims to cultivate a thriving ecosystem for early-stage entrepreneurs in the life sciences sector. Researchers have identified new variations in neuroblastoma that could lead to [...] Read more