Six-year-old ’s death leads to hope of new drug for childhood cancer

Parents of Abbie Mifsud created the charity that has funded vital research on brain stem tumoursResearchers say they are close to testing a drug that could tackle a previously untreatable childhood brain cancer. The condition is known as DIPG – diffuse intrinsic pontine glioma – and every year it affects between 30 to 40 boys and girls, aged between five and 10. All develop tumours caused by the condition and die, often within months of their diagnoses.But now scientists believe they may soon be able to tackle the condition – and one of the key organisations involved is a remarkable charity set up by Amanda and Ray Mifsud, whose daughter Abbie died of DIPG in 2011. Their charity,Abbie ’s Army, has raised money that has provided vital backing for research by Professor Chris Jones, of the Institute of Cancer Research, London.Continue reading...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Drugs Health Science Society UK news Source Type: news

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Conclusions: Brain tumors especially germinoma are associated with the development of hypothalamic–pituitary antibodies and pituitary defects. The correct interpretation of APA/AHA antibodies is essential to avoid a misdiagnosis of an autoimmune infundibulo-neurohypophysitis or pituitary hypophysitis in patients with germinoma.
Source: Frontiers in Endocrinology - Category: Endocrinology Source Type: research
(Johns Hopkins Medicine) In experiments with human cells and mice, researchers at the Johns Hopkins Kimmel Cancer Center report evidence that combining the experimental cancer medication TAK228 (also called sapanisertib) with an existing anti-cancer drug called trametinib may be more effective than either drug alone in decreasing the growth of pediatric low-grade gliomas.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
Abstract PURPOSE: Pediatric low-grade glioma (pLGG) is the most prevalent childhood brain tumor. Patients with BRAF V600 mutation-positive pLGG may benefit from treatment with dabrafenib. Part 2 of a phase I/IIa study, open-label study (NCT01677741) explores the activity and safety of dabrafenib treatment in these patients. PATIENTS AND METHODS: Patients ages 1 to
Source: Clinical Cancer Research - Category: Cancer & Oncology Authors: Tags: Clin Cancer Res Source Type: research
After using computer algorithms to sour through 9,000 possible drug combinations, Stanfors scientists found a pair with potential for treating diffuse intrinsic glioma, a rare and lethal childhood cancer.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news
(NIH/National Center for Advancing Translational Sciences (NCATS)) Researchers have devised a new, promising plan of attack against deadly childhood brain cancers called diffuse midline gliomas (DMG). NCATS and Stanford University scientists and their colleagues showed that combining two drugs killed DMG patient cells grown in the laboratory and in animal models. The drugs countered the effects of a genetic mutation that causes the diseases. Their studies also uncovered an unrecognized vulnerability in the cancer cells that scientists may be able to exploit.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
CCR Grand Rounds Dr. Jabado ’ s research focuses on elucidating genetic signatures of pediatric astrocytomas and examining how they compare to adults. These are deadly brain tumors that originate in the brain and include glioblastomas (GBM, the highest grade of astrocytomas), which are one of the deadliest cancers in humans. Her group uncovered that pediatric high-grade astrocytomas (HGA) are molecularly and genetically distinct from adult tumors. They also identified a new molecular mechanism driving pediatric HGA, namely recurrent somatic driver mutations in the tail of histone 3 variants (H3.3 and H3.1). These mut...
Source: Videocast - All Events - Category: General Medicine Tags: Upcoming Events Source Type: video
Abstract Pediatric brain tumors are the leading cause of childhood cancer-related death. Immunotherapy is a powerful new approach for treating some refractory cancers; applying this 'fourth pillar' of cancer treatment to pediatric brain tumors is an exciting but challenging prospect. This review offers new perspectives on moving towards successful immunotherapy for pediatric brain tumors, focusing on pediatric high-grade glioma (HGG), a subgroup with universally poor outcomes. We cover chimeric antigen receptor T cell (CAR-T) therapy, vaccine therapy, and checkpoint inhibition in this context, and focus on the nee...
Source: Trends in Immunology - Category: Allergy & Immunology Authors: Tags: Trends Immunol 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
Diffuse intrinsic pontine glioma (DIPG) is a highly aggressive paediatric brain tumour with fatal outcome. The Individualised Therapy For Relapsed Malignancies In Childhood (INFORM) registry study offers comprehensive molecular profiling of high-risk tumours to identify target  alterations for potential precision therapy. We analysed molecular characteristics and clinical data after brainstem biopsy of all enrolled newly diagnosed DIPGs.
Source: European Journal of Cancer - Category: Cancer & Oncology Authors: Source Type: research
In conclusion, CAR-T treatment combined with intratumoral delivery of poly I:C resulted in synergistic antitumor activity. We thus provide a rationale to translate this immunotherapeutic strategy to solid tumors. Introduction Adoptive T cell immunotherapy has been demonstrated to be a new way to fight malignancies. In particular, T lymphocytes engineered to express chimeric antigen receptor (CAR) have shown great promise in treating hematological malignancies (1). CD19-targeted CAR-T cells have been approved by FDA to treat relapsed B cell acute lymphoblastic leukemia (B-ALL) and Diffuse Large B-cell lymphoma (DLBC...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
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