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Finding a needle in a haystack: functional screening for novel targets in cancer immunology and immunotherapies.
Oncogene
January 2025
Department of Obstetrics and Gynecology, Key Laboratory of Birth Defects and Related Diseases of Women and Children of MOE, West China Second Hospital, State Key Laboratory of Biotherapy, and Department of Neurosurgery, West China Hospital, Sichuan University and Collaborative Innovation Center, Chengdu, P. R. China.
Genome-wide functional genetic screening has been widely used in the biomedicine field, which makes it possible to find a needle in a haystack at the genetic level. In cancer research, gene mutations are closely related to tumor development, metastasis, and recurrence, and the use of state-of-the-art powerful screening technologies, such as clustered regularly interspaced short palindromic repeat (CRISPR), to search for the most critical genes or coding products provides us with a new possibility to further refine the cancer mapping and provide new possibilities for the treatment of cancer patients. The use of CRISPR screening for the most critical genes or coding products has further refined the cancer atlas and provided new possibilities for the treatment of cancer patients.
View Article and Find Full Text PDFAssociation of age, race, and ethnicity with access, response, and toxicities from CAR-T therapy in children and adults with B-cell malignancies: a review.
J Immunother Cancer
January 2025
Department of Medicine and Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA
Chimeric antigen receptor T cell (CAR-T) therapies are now standard-of-care for several B-cell malignancies, and additional indications are being evaluated. In this review, we survey data on how outcomes after CAR-T therapies vary according to age, race, and ethnicity. We also review the representation of age, racial, and ethnic groups in key CAR-T clinical trials.
View Article and Find Full Text PDFPaediatric strategy forum for medicinal product development in diffuse midline gliomas in children and adolescents ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration.
Eur J Cancer
January 2025
ACCELERATE, Europe; Gustave Roussy Cancer Centre, Paris, France.
Fewer than 10 % of children with diffuse midline glioma (DMG) survive 2 years from diagnosis. Radiation therapy remains the cornerstone of treatment and there are no medicinal products with regulatory approval. Although the biology of DMG is better characterized, this has not yet translated into effective treatments.
View Article and Find Full Text PDFAccess to CARe: A Narrative of Real-World Medical Decision-Making to Access Chimeric Antigen Receptor (CAR) T-Cell Therapy in Children, Adolescents, and Young Adults.
Pediatr Blood Cancer
January 2025
Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA.
Chimeric antigen receptor (CAR) T-cell therapy is a potentially life-saving treatment for children with relapsed/refractory B-cell hematologic malignancies, and remains an important investigational therapy for other childhood cancers. Yet, access to this class of therapies remains suboptimal through both commercial use and clinical trials, especially in children, adolescents, and young adults. Using a series of case-based discussions, we outline guidance on real-world medical decision-making, and offer potential solutions to enhancing access to CAR T-cell therapy as a treatment modality.
View Article and Find Full Text PDFCardiovascular Complications of Immune Effector Cell Therapies in Pediatric Hematological and Solid Tumors.
Pediatr Blood Cancer
January 2025
Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston, Texas, USA.
Background: Immune effector cell (IEC) therapies, including chimeric antigen receptor (CAR)-modified T-cell therapy, have shown efficacy in pediatric B-cell acute lymphoblastic leukemia (B-ALL) and are being investigated for other malignancies. A common toxicity associated with IEC therapy is cytokine release syndrome (CRS), which can lead to cardiovascular decompensation due to systemic inflammation. Data are limited regarding cardiovascular adverse effects in children.
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