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Last Posted: Apr 10, 2024
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Nucleic acid-based drugs for patients with solid tumours.
Sebastian G Huayamares et al. Nat Rev Clin Oncol 2024 4

From the abstract: "The treatment of patients with advanced-stage solid tumours typically involves a multimodality approach (including surgery, chemotherapy, radiotherapy, targeted therapy and/or immunotherapy), which is often ultimately ineffective. Nucleic acid-based drugs, either as monotherapies or in combination with standard-of-care therapies, are rapidly emerging as novel treatments capable of generating responses in otherwise refractory tumours. These therapies include those using viral vectors (also referred to as gene therapies), several of which have now been approved by regulatory agencies, and nanoparticles containing mRNAs and a range of other nucleotides. "

Exploiting tumor aneuploidy as a biomarker and therapeutic target in patients treated with immune checkpoint blockade
LF Spurr et al, NPJ Precision Oncology, January 2, 2023

From the article: "Tumor mutational burden (TMB) has received considerable attention as a validated biomarker of ICB response, which resulted in the 2020 FDA approval for the use of high TMB (=10 mutations per megabase of DNA) as a tissue-agnostic biomarker for patients treated with pembrolizumab. However, the overall response rate in TMB-high tumors in the study which catalyzed FDA approval was only 29%. Thus, much debate remains regarding how to optimally utilize TMB as a biomarker of ICB response and whether other tumor or host features provide additional predictive value in this setting. Multiple complementary biomarkers have been proposed, including neoantigen load, CD8+ T cell expression signatures, PD-L1 expression, mismatch repair deficiency, and HLA genotype. "

'It's all gone': CAR-T therapy forces autoimmune diseases into remission.
Heidi Ledford et al. Nature 2023 12

From the article: "Engineered immune cells have given 15 people with once-debilitating autoimmune disorders a new lease on life, free from fresh symptoms or treatments. The results raise hopes that the approach — called CAR-T-cell therapy — might one day be extended to a variety of other conditions fuelled by rogue immune cells that produce antibodies against the body’s own tissues. "

Computational immunogenomic approaches to predict response to cancer immunotherapies.
Venkateswar Addala et al. Nat Rev Clin Oncol 2023 11

From the abstract: " Cancer immunogenomics is an emerging field that bridges genomics and immunology. The establishment of large-scale genomic collaborative efforts along with the development of new single-cell transcriptomic techniques and multi-omics approaches have enabled characterization of the mutational and transcriptional profiles of many cancer types and helped to identify clinically actionable alterations as well as predictive and prognostic biomarkers. Researchers have developed computational approaches and machine learning algorithms to accurately obtain clinically useful information from genomic and transcriptomic sequencing data. "


Disclaimer: Articles listed in the Public Health Genomics and Precision Health Knowledge Base are selected by the CDC Office of Public Health Genomics to provide current awareness of the literature and news. Inclusion in the update does not necessarily represent the views of the Centers for Disease Control and Prevention nor does it imply endorsement of the article's methods or findings. CDC and DHHS assume no responsibility for the factual accuracy of the items presented. The selection, omission, or content of items does not imply any endorsement or other position taken by CDC or DHHS. Opinion, findings and conclusions expressed by the original authors of items included in the update, or persons quoted therein, are strictly their own and are in no way meant to represent the opinion or views of CDC or DHHS. References to publications, news sources, and non-CDC Websites are provided solely for informational purposes and do not imply endorsement by CDC or DHHS.

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