Home Medizin Studie analysiert den Einsatz gewebeunabhängiger Therapeutika bei Patienten mit primären Hirntumoren

Studie analysiert den Einsatz gewebeunabhängiger Therapeutika bei Patienten mit primären Hirntumoren

von NFI Redaktion

A study was published today in Trends in Cancer by researchers at the Miami Cancer Institute, part of Baptist Health South Florida, analyzing the use of tissue-agnostic therapies in patients with primary brain tumors (PBTs). The publication describes the current and potential impact of tissue-agnostic therapies on the treatment of PBTs. Within the publication, researchers discuss data from clinical trials on tissue-agnostic targets for PBTs in the context of the challenges in treating these tumors. They also describe additional tissue-agnostic targets that could benefit patients with PBTs.

Novel tissue-agnostic therapeutics targeting driver mutations in tumor cells have recently been approved by the FDA and other regulatory agencies around the world based on studies demonstrating their effectiveness and safety across various tumor histologies. However, the relative rarity of primary brain tumors has limited their representation in early studies of tissue-agnostic drugs.

Manmeet S. Ahluwalia, MD, MBA, FASCO, Chief of Medical Oncology, Scientific Director, Deputy Director, and the Fernandez Family Foundation Endowed Chair of Cancer Research at the Miami Cancer Institute and lead author of the study

Basket studies – which test targeted therapy based on the molecular profiles of a wide range of cancers – have recently led to the next frontier of precision medicine, that is, tissue-agnostic approvals. These enable the use of targeted therapies based on molecular alterations that are independent of the tumor’s tissue of origin. There is significant genetic, epigenetic, and immunological heterogeneity of PBTs, increasing the complexity of tissue-agnostic therapies. Inherent differences between different types of PBTs must be carefully considered, as even within the same tumor, multiple subtypes within the same spectrum can exist, further amplifying histological heterogeneity. However, the concept of tissue-agnostic therapies emerged primarily from the argument that common molecular profiles can limit the impacts of these inter- and intratumoral heterogeneities from a therapeutic standpoint by targeting common molecular alterations.

Over the past decade, several therapeutics with broad efficacy in various cancer types have been developed and approved due to a better understanding of tumor biology. The increasing use of next-generation sequencing and biomarker-driven basket studies has helped identify several actionable genetic alterations and their clinical utility. As a result, this has led to approvals for tissue-agnostic malignancies by the FDA and others, many of which have already proven useful in PBTs.

“However, the limited number of patients with different PBTs in published studies impedes their widespread acceptance,” added Ahluwalia. “Therefore, it is increasingly important for individuals with primary brain tumors to undergo molecular profiling to maximize therapeutic options based on individual characteristics and to capture these data through real-world evidence analyses. These tissue-agnostic approvals represent a new frontier, promising better treatment outcomes for gliomas in precision oncology.”


Baptist Health South Florida

Journal reference:

Ahluwalia, MS, et al. (2024) Impact of Tissue-Agnostic Approvals on the Treatment of Primary Brain Tumors. Trends in Cancer. doi.org/10.1016/j.trecan.2023.11.005.

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