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UnknownClinical trial of a farnesyltransferase inhibitor in children with Hutchinson-Gilford progeria syndrome.
Hutchinson-Gilford progeria syndrome (HGPS) is an extremely rare, fatal, segmental premature aging syndrome caused by a mutation in LMNA that produces the farnesylated aberrant lamin A protein, progerin. This multisystem disorder causes failure to thrive and accelerated atherosclerosis leading to ea...
S-EPMC3478615 | biostudies-literature
UnknownGenomic characterization of colorectal tumors: insights into significantly mutated genes, pathways, and survival outcomes.

Background

Identifying significantly mutated genes in tumors aids in understanding disease etiology and survival and may aid in the discovery of new drug targets. We aimed to detect and characterize mutated genes from a large, well-characterized group of colorectal cancers.

Methods

In...
S-EPMC12831426 | biostudies-literature
UnknownA distinct Fusobacterium nucleatum clade dominates the colorectal cancer niche.
Fusobacterium nucleatum (Fn), a bacterium present in the human oral cavity and rarely found in the lower gastrointestinal tract of healthy individuals1, is enriched in human colorectal cancer (CRC) tumours2-5. High intratumoural Fn loads are associated with recurrence, metastas...
S-EPMC11006615 | biostudies-literature
UnknownCirculating tumor DNA level is associated with time to clinical recurrence in Merkel cell carcinoma: Implications for patient management.

Background

Merkel cell carcinoma (MCC) recurs in 40% of patients. Circulating tumor DNA (ctDNA) is an emerging blood-based biomarker for early MCC recurrence detection.

Objective

To evaluate the timing and prognostic significance of ctDNA levels relative to clinical recurrence.

Met...

S-EPMC12596136 | biostudies-literature
UnknownThe use of neoadjuvant larotrectinib in the management of children with locally advanced TRK fusion sarcomas.

Background

The highly selective oral tropomyosin receptor kinase (TRK) inhibitor larotrectinib has demonstrated significant activity in adult and pediatric TRK fusion cancers. In the current study, the authors describe the clinical course of children with locally advanced TRK fusion sarcoma ...
S-EPMC6263791 | biostudies-literature
UnknownDecitabine and vorinostat with FLAG chemotherapy in pediatric relapsed/refractory AML: Report from the therapeutic advances in childhood leukemia and lymphoma (TACL) consortium.
Survival outcomes for relapsed/refractory pediatric acute myeloid leukemia (R/R AML) remain dismal. Epigenetic changes can result in gene expression alterations which are thought to contribute to both leukemogenesis and chemotherapy resistance. We report results from a phase I trial with a dose expa...
S-EPMC8986610 | biostudies-literature
UnknownLarotrectinib for paediatric solid tumours harbouring NTRK gene fusions: phase 1 results from a multicentre, open-label, phase 1/2 study.
Not available
S-EPMC5949072 | biostudies-literature
UnknownGenetic Mechanisms of Immune Evasion in Colorectal Cancer.
To understand the genetic drivers of immune recognition and evasion in colorectal cancer, we analyzed 1,211 colorectal cancer primary tumor samples, including 179 classified as microsatellite instability-high (MSI-high). This set includes The Cancer Genome Atlas colorectal cancer cohort of 592 sampl...
S-EPMC5984687 | biostudies-literature
UnknownOutcome of Patients With Recurrent Osteosarcoma Enrolled in Seven Phase II Trials Through Children's Cancer Group, Pediatric Oncology Group, and Children's Oncology Group: Learning From the Past to Move Forward.

Purpose

The use of radiographic response as the primary end point in phase II osteosarcoma trials may limit optimal detection of treatment response because of the calcified tumor matrix. We performed this study to determine if time to progression could be used as an end point for subsequent ...
S-EPMC5012712 | biostudies-literature
UnknownCurrent state of pediatric sarcoma biology and opportunities for future discovery: A report from the sarcoma translational research workshop.
Sarcomas are a rare subgroup of pediatric cancers comprised of a variety of bone and soft-tissue tumors. While significant advances have been made in improving outcomes of patients with localized pediatric sarcomas since the addition of systemic chemotherapy to local control many decades ago, outcom...
S-EPMC5497490 | biostudies-literature
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