PuSH - Publication Server of Helmholtz Zentrum München

Mechahougui, H.* ; Friedlaender, A.* ; Görgülü, K. ; Tsantoulis, P.* ; Illert, A.L.* ; Subbiah, V.* ; Wahida, A. ; Kurzrock, R.*

Precision oncology in rare tumors: Have the orphans been adopted?

Med. 7:100958 (2026)
Publ. Version/Full Text DOI PMC
Open Access Hybrid
Creative Commons Lizenzvertrag
Oncology has shifted from organ- or histology-based therapy toward precision medicine, guided by molecular and immune targets. This paradigm change is especially impactful in rare cancers, which may be defined by unusual histology, anatomical site, or uncommon molecular subtypes of common tumors. The emergence of tumor-agnostic, histology-independent classifications has added new molecularly defined rare tumor groups. Biomarker-based therapies mark a turning point in personalized medicine, with several gene- and immune-targeted drugs now approved by the US Food and Drug Administration (FDA). Tissue-agnostic approvals typically rely on trials showing high response rates and durable benefit across diverse cancers driven by rare molecular alterations. This approach is crucial for the ∼200 types of histologically rare cancers, where trials in tiny patient subsets are unfeasible. Finally, the complexity and variability of tumor genomics between patients highlight the need for individualized, n-of-1 therapeutic combinations as the next step in optimizing cancer treatment.
Altmetric
Additional Metrics?
[➜Log in]
Edit extra informations Login
Publication type Article: Journal article
Document type Review
Keywords Agnostic Therapy ; Molecular Tumor Boards ; Orphan Disease ; Precision Oncology ; Rare Tumors ; Targeted Therapy; Inflammatory Myofibroblastic Tumor; Biliary-tract Cancer; Sporadic Desmoid Tumors; Erdheim-chester Disease; Giant-cell Tumors; Open-label; Clinical Activity; Targeted Therapy; Single-arm; Metastatic Cholangiocarcinoma
ISSN (print) / ISBN 2666-6359
e-ISSN 2666-6340
Journal Med (N Y)
Quellenangaben Volume: 7, Issue: 2, Pages: , Article Number: 100958 Supplement: ,
Publisher Elsevier
Publishing Place 50 Hampshire St, Floor 5, Cambridge, Ma 02139 Usa
Reviewing status Peer reviewed
Institute(s) Institute of Metabolism and Cell Death (MCD)