Related Experiment Video
Updated: Aug 2, 2025

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
Cancer of unknown primary (CUP) through the lens of precision oncology: a single institution perspective
L Weiss1,2,3, K Heinrich1, D Zhang1,4
1Department of Medicine III, University Hospital, LMU Munich, Munich, Germany.
Purpose:
For patients with cancer of unknown primary (CUP), treatment options are limited. Precision oncology, the interplay of comprehensive genomic profiling (CGP) and targeted therapies, aims to offer additional treatment options to patients with advanced and hard-to-treat cancers. We aimed to highlight the use of a molecular tumor board (MTB) in the therapeutic management of CUP patients.
Methods:
In this single-center observational study, CUP patients, presented to the MTB of the Comprehensive Cancer Center Munich LMU, a tertiary care center, were analyzed retrospectively. Descriptive statistics were applied to describe relevant findings.
Results:
Between June 2016 and February 2022, 61 patients with unfavorable CUP were presented to the MTB, detected clinically relevant variants in 74% (45/61) of patients, of which 64% (29/45) led to therapeutic recommendation. In four out of 29 patients (14%), the treatment recommendations were implemented, unfortunately without resulting in clinical benefit. Reasons for not following the therapeutic recommendation were mainly caused by the physicians' choice of another therapy (9/25, 36%), especially in the context of worsening of general condition, lost to follow-up (7/25, 28%) and death (6/25, 24%).
Conclusion:
CGP and subsequent presentation to a molecular tumor board led to a high rate of therapeutic recommendations in patients with CUP. Recommendations were only implemented at a low rate; however, late GCP diagnostic and, respectively, MTB referral were found more frequent for the patients with implemented treatment. This contrast underscores the need for early implementation of CGP into the management of CUP patients.
Insights
Comprehensive genomic profiling (CGP) and molecular tumor board (MTB) reviews offer therapeutic recommendations for cancer of unknown primary (CUP) patients. However, low implementation rates highlight the need for earlier CGP integration in CUP management.
Area of Science:
- Oncology
- Genomics
- Precision Medicine
Background:
- Cancer of unknown primary (CUP) presents limited treatment options.
- Precision oncology, utilizing comprehensive genomic profiling (CGP) and targeted therapies, aims to improve outcomes for advanced cancers.
- Molecular tumor boards (MTBs) are emerging as key decision-making platforms in complex oncology cases.
Purpose of the Study:
- To evaluate the utility of a molecular tumor board (MTB) in guiding the therapeutic management of cancer of unknown primary (CUP) patients.
- To assess the rate of therapeutic recommendations derived from CGP and MTB review in CUP.
- To identify factors influencing the implementation of MTB-based treatment strategies for CUP.
Main Methods:
- A single-center, retrospective observational study analyzed CUP patients presented to an MTB.
- Descriptive statistics were employed to summarize patient data and MTB findings.
- The study period spanned from June 2016 to February 2022, involving a tertiary care center.
Main Results:
- Of 61 CUP patients reviewed, 74% had clinically relevant genomic variants detected via CGP.
- Therapeutic recommendations were made for 64% of patients with detected variants.
- Treatment recommendations were implemented in only 14% of cases, with limited clinical benefit observed.
Conclusions:
- CGP and MTB review yield a high rate of therapeutic recommendations for CUP patients.
- Low implementation rates of recommendations were observed, often due to physician preference or patient decline.
- Early integration of CGP and MTB referral is crucial for improving treatment outcomes in CUP.
Related Concept Videos
Cancers Originate from Somatic Mutations in a Single Cell
Cancer

