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Assorted news from the last two weeks: The FDA has granted rare pediatric disease designation to IMX-110 for the treatment of rhabdomyosarcoma, a life-threatening cancer found in children. This designation makes IMX-110 eligible for a fast track designation and a priority review designation in the future, according to a press release by Immix Biopharma, Inc. FDA-approved TRK inhibitors entrectinib and larotrectinib have demonstrated safety and efficacy in treating a variety of solid tumors in pediatric and adult cancer patients. The FDA granted orphan drug designation to silmitasertib for treatment of medulloblastoma. A molecular building block of many animal proteins, the amino […]

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Assorted news from last week: From the Lancet: Adolescent and young adult cancers contributed substantially to the overall adolescent and young adult disease burden globally in 2019. These results provide new insights into the distribution and magnitude of the adolescent and young adult cancer burden around the world.  An immunotherapy harnessing the immune system’s “natural killer” cells has proven effective in treating acute myeloid leukemia (AML) in some adults whose cancers return. Now, researchers at Washington University School of Medicine in St. Louis have shown, in a small clinical trial, that the same natural killer cells also can help some […]

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Assorted news from last week: Adding a novel anti-GD2 antibody (hu14.18K322A) to induction chemotherapy for children with newly diagnosed high-risk neuroblastoma yielded impressive survival outcomes and improved early responses versus historical benchmarks, investigators reported. Researchers have developed a test that detects specific changes in DNA fragments, or cell-free DNA, shed from medulloblastoma tumor cells into the fluid surrounding the brain and spinal cord, known as the cerebrospinal fluid.  Based on their findings from a recent study, the researchers believe this test could potentially be used to identify children who, shortly after completing treatment, still have evidence of cancer—known as residual [...] Read more

Assorted news from last week: According to Jim Morris, investigative reporter for Public Health Watch, “for the foreseeable future, thousands of children will continue to be diagnosed with cancer each year and require treatment. But unless the drug-development process undergoes a significant transformation, they will remain a lower priority than adults.” The FDA approves Rituximab Plus chemotherapy for pediatric patients (≥6 months to <18 years) with previously untreated, advanced stage, CD20-positive diffuse large B-cell lymphoma (DLBCL), Burkitt lymphoma (BL), Burkitt-like lymphoma (BLL), or mature B-cell acute leukemia (B-AL). “Mariah Forster Olson’s treatment for childhood cancer left her with a range […]

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Assorted news from last week: A primer on where the Centers for Medicare and Medicaid Services see Medicaid and the Children’s Health Insurance Program (CHIP) heading in the future. Based on a Health Affairs commentary from Brooks-LaSure and Tsai, the three key priority areas are (1) coverage and access, (2) equity, and (3) innovation and whole-person care. CAC2 Supporting Organization Oncoheroes Biosciences has been awarded to be part of the 1st generation of companies accelerated in the #BCNHealthBooster, an initiative promoted by the Parc Científic de Barcelona (PCB) of Universitat de Barcelona and BarcelonaCityCouncil, with the collaboration of Barcelona Activa […]

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Assorted news from last week: Provocative article written by pediatric cancer advocates, including several CAC2 members — innovation can be crippled by commercialization — meaning next better treatments are completely at the mercy of companies who now have income from drug sales from approved drugs and may not agree to provide the drug for further testing in combinations, etc. To encourage drug companies (sponsors) to develop effective treatments for childhood cancer, authorities in the United States (US) and Europe have made new rules for drug development, and this team from Pfizer has some recommendations to help sponsors maximize their chances of […]

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Assorted news from last week: Save the Date:  CureFest 2022 will be held Sep. 24 and 25 in Washington, DC. Second Paediatric Strategy Forum for anaplastic lymphoma kinase (ALK) inhibition in paediatric malignancies reported their work in the most recent edition  of European Journal of Cancer.  The Forum was sponsored by ACCELERATE in collaboration with the European Medicines Agency with the participation of the Food and Drug Administration. CAC2 Member Oncoheroes Biosciences Inc., a biotech focused on advancing new therapies for childhood cancer, has out-licensed worldwide the adult rights to Notable Labs, Inc. (“Notable”), a clinical-stage predictive precision medicine company.  Under the terms of this […]

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Assorted news from last week: Save the Date:  CureFest 2022 will be held Sep. 24 and 25 in Washington, DC. The Children’s Brain Tumor Network (CBTN) has collected tumor samples from patients at 26 member institutions all over the world. Through funding from the Childhood Cancer Data Initiative of the National Cancer Institute (NCI), part of the National Institutes of Health (NIH).  CBTN will procure molecular characterization for thousands of these brain tumor samples providing an unprecedented level of insight into devastating cancers and paving the way for future therapeutic interventions. Compassionate use case of DAY101 demonstrates a complete response in a pediatric patient with a recurrent spindle cell sarcoma harboring a BRAF gene fusion. Despite the relative rarity […]

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