Optimus-era dose finding for rare cancers Journal Article
| Authors: | Murciano-Goroff, Y. R.; Devlin, S. M.; Iasonos, A.; Drilon, A. |
| Article Title: | Optimus-era dose finding for rare cancers |
| Abstract: | Advances in cancer biology and diagnostics have led to the recognition of a multitude of rare cancer subtypes, emphasizing the pressing need for strategies to accelerate drug development for patients with these cancers. This paper addresses the unique challenges of dose finding in trials that accrue small numbers of patients with rare cancers; strategies for dose optimization are proposed, in line with evolving approaches to dose determination in the age of the US Food and Drug Administration’s Project Optimus. © 2024 American Association for Cancer Research. |
| Keywords: | human tissue; protein expression; gene mutation; somatic mutation; dose response; antineoplastic agents; drug approval; united states; united states food and drug administration; cancer patient; antineoplastic agent; genetic analysis; neoplasm; neoplasms; gastrointestinal stromal tumor; imatinib; progression free survival; epidermal growth factor receptor 2; antineoplastic activity; food and drug administration; dose-response relationship, drug; drug development; cancer research; cancer therapy; histology; gene rearrangement; immunotherapy; diagnosis; tyrosine kinase receptor; blood pressure; maximum tolerated dose; drug therapy; trastuzumab; pharmacokinetics; rare disease; personalized medicine; rare diseases; central nervous system disease; non small cell lung cancer; randomized controlled trial (topic); molecularly targeted therapy; dose limiting toxicity; phase 2 clinical trial (topic); frequency analysis; phase 1 clinical trial (topic); patient-reported outcome; minimum inhibitory concentration; germline mutation; humans; human; article; ic50; osimertinib; malignant neoplasm; pharmacogenetic testing; tumor mutational burden; selpercatinib; ic90; multiomics; meningeal melanocytosis |
| Journal Title: | Cancer Discovery |
| Volume: | 14 |
| Issue: | 6 |
| ISSN: | 2159-8274 |
| Publisher: | American Association for Cancer Research |
| Date Published: | 2024-06-01 |
| Start Page: | 909 |
| End Page: | 914 |
| Language: | English |
| DOI: | 10.1158/2159-8290.Cd-24-0368 |
| PUBMED: | 38826101 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | The MSK Cancer Center Support Grant (P30 CA008748) is acknowledged in the PDF -- Corresponding authors is MSK author: Alexander Drilon -- Source: Scopus |
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