Publications by authors named "Noah Federman"

Non-rhabdomyosarcoma soft tissue sarcoma (NRSTS) is a group of over 70 tumors that occur across the age range and account for approximately 4 % of childhood cancers. Patients with metastatic or relapsed NRSTS have a dismal prognosis. The histologic, molecular, and clinical heterogeneity of NRSTS and the rarity of individual types pose unique challenges to the study of these diseases.

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Adoptive transfer of genetically engineered T cells expressing a tumor-antigen-specific transgenic T cell receptor (TCR) can result in clinical responses in a variety of malignancies. However, these responses are frequently short-lived, and patients typically relapse within several months. This phenomenon is largely due to poor persistence of the transgenic T cells, as well as a progressive loss of their functionality and terminal differentiation in vivo.

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Background And Objectives: The combination of radiation and surgical resection represents the current standard of care for primary synovial sarcoma (SS). However, controversy remains regarding the role of chemotherapy. We sought to evaluate the impact of neoadjuvant chemotherapy on the likelihood of undergoing amputation for patients with lower extremity SS.

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Larotrectinib is a highly selective tropomyosin receptor kinase (TRK) inhibitor with efficacy in children with TRK fusion tumors. We evaluated patient outcomes after elective discontinuation of larotrectinib in the absence of disease progression in a protocol-defined wait-and-see subset analysis of eligible patients where treatment resumption with larotrectinib was allowed if disease progressed. We also assessed the safety and efficacy of larotrectinib in all pediatric patients with sarcoma.

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Introduction: Patient navigation is recommended by accrediting bodies such as the Commission on Cancer and is a key element in payment reform demonstration projects, due to the established benefits in reducing barriers to healthcare, improving care coordination and reducing healthcare utilisation. However, oncology practices are often resource constrained and lack the capacity to extend navigation services at the desired intensity for their patient population. The American Cancer Society (ACS) developed the ACS Community Access to Resources, Education, and Support (CARES) programme to expand navigation capacity through the training of students from local universities as volunteers to serve as non-clinical navigators to support cancer patients.

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Article Synopsis
  • - Sarcomas are rare tumors with over 100 subtypes, making it challenging to find effective therapies; there's a need for personalized treatment approaches to enhance patient outcomes.
  • - Patient-derived tumor organoids (PDTOs) were used to study drug resistance and sensitivity in sarcoma, analyzing 194 specimens from 126 patients across 24 subtypes.
  • - The research developed a high-throughput screening method that provided results quickly and showed that drug sensitivity linked to tumor characteristics; 59% of samples matched with at least one effective FDA-approved treatment.
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Objectives: We introduced selpercatinib prior to radioactive iodine therapy prior to radioactive iodine therapy (RAI) for pediatric papillary thyroid cancer (PTC) to enhance the tumorical effects of RAI.

Case Presentation: PTC has an excellent prognosis but is commonly associated with local and distant metastases. Successful complete response to the current standard of care, thyroidectomy with lymph node resection and RAI, is achieved in only a small minority of cases with metastases.

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Rhabdomyosarcoma (RMS) is the most common childhood soft tissue sarcoma. For the alveolar subtype (ARMS), the presence of the PAX3::FOXO1 fusion gene and/or metastases are strong predictors of poor outcome. Metastatic PAX3::FOXO1 ARMS often responds to chemotherapies initially, only to subsequently relapse and become resistant with most patients failing to survive beyond 8 years post-diagnosis.

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Purpose: Ewing Sarcoma (ES), a rare cancer with a pathognomonic translocation resulting in the Ewing sarcoma gene (EWS)::FLI1 oncoprotein, has a poor prognosis in the relapsed/refractory (R/R) setting. Tokalas (TK)216 was designed to bind EWS::FLI1 proteins directly, disrupt protein-protein interactions, and inhibit transcription factor function. TK216 plus vincristine showed synergistic activity in preclinical tumor models.

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Background: Neurotrophic tyrosine receptor kinase () gene fusions are rare oncogenic drivers prevalent in 0.3% of solid tumors. They are most common in salivary gland cancer (2.

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A "Think Tank for Osteosarcoma" medical advisory board meeting was held in Santa Monica, CA, USA on February 2-3, 2024. The goal was to develop a strategic approach to prevent recurrence of osteosarcoma. Osteosarcoma metabolism and the genomic instability of osteosarcoma, immunotherapy for osteosarcoma, CAR-T cell therapy, DeltaRex-G tumor-targeted gene therapy, repurposed drugs, alternative medicines, and personalized medicine were discussed.

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Objective: The study investigated maternal exposure to heavy metals from industrial sources during pregnancy as potential risk factors for childhood cancer.

Methods: Cases ages 0-19 were identified from California Cancer Registry. Controls (20:1 ratio) were randomly selected from California Birth Registry, frequency-matched by birth year (1998-2016).

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Background: Maternal solvent exposure has been suspected to increase offspring cancer risk. The study aimed to evaluate the associations between maternal residential exposure to solvents from industrial pollution during pregnancy and childhood cancer.

Methods: The present study included 15,744 cancer cases (aged 0-19 years at diagnosis) identified from California Cancer Registry and 283,141 controls randomly selected from California Birth Registry (20:1 frequency-matched by birth year: 1998-2016).

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Article Synopsis
  • - Nirogacestat is an FDA-approved drug for treating desmoid tumors in adults, but a phase 3 study revealed that it can cause ovarian toxicity (OT) in females capable of reproduction, which necessitated further investigation into its effects.
  • - In this study, 75% of women taking nirogacestat experienced OT, while none in the placebo group did; the average duration of OT was about 19 weeks, but most patients saw resolution of symptoms after stopping the drug or while on treatment.
  • - The findings suggest that although ovarian toxicity is common among those treated with nirogacestat, it tends to be temporary, with many patients recovering their menstrual cycles and hormonal levels after either stopping the medication or
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Background: Childhood cancers are associated with high mortality and morbidity, and some maternal prescription drug use during pregnancy has been implicated in cancer risk. There are few studies on the effects of hypertension, preeclampsia, and the use of antihypertensives in pregnancy on children's cancer risks.

Objective: This population-based cohort study analyzed the relationship between hypertension, preeclampsia, and antihypertensives taken during pregnancy and the risks of childhood cancers in the offspring.

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An 8-year-old female presented to the oculoplastics clinic with 3 months of left upper eyelid fullness and edema. Examination showed a mass in the left anterior superior orbit with erythema. Imaging demonstrated a well-circumscribed superolateral orbital mass that was T1 hypointense and T2 hypo-to-iso intense with contrast enhancement.

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Background: Lynch syndrome (LS) is an autosomal dominant genetic predisposition to multiple malignancies and is characterized by deficient DNA mismatch repair. Increased incidence of sarcomas is not formally ascribed to LS; however, increasing evidence suggests a preponderance of these malignancies in affected families. Sarcomas typically possess a low tumor mutational burden and incite a poor immune infiltrate, thereby rendering them poorly responsive to immunotherapy.

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The Research to Accelerate Cures and Equity (RACE) for Children Act mandates that newly developed targeted oncology drugs be tested in children when molecular targets are relevant to pediatric cancers. In its first year, the RACE for Children Act was effective in creating novel drug development opportunities for children with cancer; however, significant barriers to clinical trial enrollment persist. Pediatric cancer clinical trials are impacted by challenges surrounding logistics, complexity, and access.

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