Decision to not make a drug for childhood cancer on the NHS is 'devastating', say charities

Health watchdog Nice, which advises NHS England, stressed it would like to recommend dinutuximab beta for patients with neuroblastoma – but argued it is too expensive.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news

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In conclusion, the data suggest that A3 AR may represent a marker of NB malignancy as well as a drug target for treatment of this solid tumor.Graphical Abstract
Source: Journal of Molecular Neuroscience - Category: Neuroscience Source Type: research
nd Precision Medicine Abstract In this scientific statement from the American Heart Association, experts in the field of cardiomyopathy (heart muscle disease) in children address 2 issues: the most current understanding of the causes of cardiomyopathy in children and the optimal approaches to diagnosis cardiomyopathy in children. Cardiomyopathies result in some of the worst pediatric cardiology outcomes; nearly 40% of children who present with symptomatic cardiomyopathy undergo a heart transplantation or die within the first 2 years after diagnosis. The percentage of children with cardiomyopathy who underwent a he...
Source: Circulation - Category: Cardiology Authors: Tags: Circulation Source Type: research
Abstract Telomeres play important roles in genome stability and cell proliferation. High risk neuroblastoma (HRNB), an aggressive childhood cancer, is especially reliant on telomere maintenance. Three recurrent genetic aberrations in HRNB (MYCN amplification, TERT re-arrangements, and ATRX mutations) are mutually exclusive and each capable of promoting telomere maintenance mechanisms (i.e., through telomerase or ALT). We analyzed a panel of 5 representative HRNB cell lines and 30 HRNB surgical samples using assays that assess average telomere lengths, length distribution patterns, single-stranded DNA on the G- and...
Source: Neoplasia - Category: Cancer & Oncology Authors: Tags: Neoplasia Source Type: research
Conclusion Astroblastoma is an extremely rare CNS tumor. Morphological diagnosis is difficult, as the typical astroblastic rosettes may be present also in other CNS tumors, including some gliomas and ependymomas. In fact, AB can be considered as a morphologic pattern, which can be associated with a spectrum of molecular entities. Total resection is the best treatment; the precise role of chemotherapy and radiotherapy is still debated, particularly for high-grade tumors. We believe that DNA-methylation profiles represents an important instrument for confirming diagnosis, predicting prognosis and better defining the molec...
Source: Frontiers in Genetics - Category: Genetics & Stem Cells Source Type: research
Targeting β3-adrenergic receptor signaling inhibits neuroblastoma cell growth via suppressing the mTOR pathway. Biochem Biophys Res Commun. 2019 Apr 25;: Authors: Deng J, Jiang P, Yang T, Huang M, Qi W, Zhou T, Yang Z, Zou Y, Gao G, Yang X Abstract Neuroblastoma (NB), the most common extracranial solid tumor in childhood, always leads to an unfavorable prognosis. β3-adrenergic receptor (β3-AR) signaling plays an important role in lipid metabolism. Although previous studies have focused mainly on the role of β2-AR in tumor cells; there are few studies about the cancer-related functi...
Source: Biochemical and Biophysical Research communications - Category: Biochemistry Authors: Tags: Biochem Biophys Res Commun Source Type: research
In this study, we investigated the impact of the human ALK-F1174L mutation in vivo on sympathetic neuroblast differentiation using Sox10-Cre;LSL-ALK-F1174L mice. The aim was to improve our understanding of the implication of ALK deregulated signaling in the initial steps of NB genesis. Results ALK-F1174L Causes Embryonic Lethality When Expressed in NCCs Prior to SA Lineage Commitment To assess the impact of the human ALK-F1174L activating mutation on the development and differentiation of the SNS, Sox10-Cre;LSL-ALK-F1174L embryos were generated. This model allowed to restrict the expression of the ALK-F1174L variant to ...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
AbstractNeuroblastoma (NB) is one of the most common malignant tumors derived from pluripotent cells of the neural crest. Nodal is an important embryonic morphogen which can re ‐express in cancer cells. The roles of Nodal in the progression of NB are not illustrated. Our present study reveals that Nodal is upregulated in NB cells and tissues. Targeted inhibition of Nodal can suppress thein vitro migration and invasion of NB cells while increase its chemo ‐sensitivity to doxorubicin (Dox) treatment. Nodal positively regulates the expression of Zeb1, one well‐known transcription factors of epithelial to mesenchymal tra...
Source: BioFactors - Category: Biochemistry Authors: Tags: Research Communication Source Type: research
(Louisiana State University Health Sciences Center) Research led by Michael Lan, Ph.D., Professor of Pediatrics and Genetics at LSU Health New Orleans School of Medicine, found that a compound named 5'-iodotubercidin (5'-IT) suppresses the growth of neuroblastoma cells and identified a potential new therapeutic approach for the disease.
Source: EurekAlert! - Cancer - Category: Cancer & Oncology Source Type: news
Conclusions: CAR T cell therapies have demonstrated the clinical benefits of harnessing our body's own defenses to combat tumor cells. Similar research is being conducted on lesser known modifications and gene-modified immune cells, which we highlight in this review. Introduction Chimeric antigen receptors and engineered T cell receptors (based on previously identified high affinity T cell receptors) function by redirecting T cells to a predefined tumor-specific (or tumor-associated) target. Most of these modifications use retroviral or lentiviral vectors to integrate the construct, and most of the receptors ...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
(Children's Hospital of Philadelphia) Physician-scientists in the Cancer Center at Children's Hospital of Philadelphia have developed a preclinical, potent therapy attached to an antibody that targets a surface protein expressed in most childhood neuroblastomas, effectively killing cancer cells.
Source: EurekAlert! - Biology - Category: Biology Source Type: news
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