In vitro assays as a tool to personalize treatment in central nervous system tumors: a systematic literature review

Area of Science:

• Neuro-oncology
• Translational Cancer Research
• Genomics and Precision Medicine

Background:

• Personalized neuro-oncology treatments traditionally depend on molecular profiling, but clinical benefits have been limited.
• In vitro drug sensitivity testing with patient-derived models (organoids, cell lines) is an emerging strategy.
• Molecular profiling faces challenges in glioblastoma due to absent driver mutations and evolving profiles at recurrence.

Purpose of the Study:

• To systematically review the evidence on the efficacy of in vitro drug screening in predicting treatment outcomes for brain tumors.
• To assess the potential of patient-derived models to enhance personalized neuro-oncology.
• To evaluate the quality of evidence supporting in vitro drug screening for brain tumor treatment prediction.

Main Methods:

• Systematic literature review following PRISMA guidelines.
• Inclusion of 15 studies encompassing 300 patients, predominantly cohort studies.
• Analysis of evidence from in vitro assays utilizing patient-derived stem cells, standardized assays (e.g., ChemoID), and tumor-derived organoids.

Main Results:

• In vitro assays using patient-derived models demonstrated reliable prediction of treatment outcomes.
• Patient-derived models may offer advantages over molecular profiling, particularly for glioblastoma.
• The overall quality of evidence supporting these methods was limited, with only one randomized clinical trial identified.

Conclusions:

• In vitro drug screening with patient-derived models is a promising strategy for personalized neuro-oncology.
• These models show potential to overcome limitations of molecular profiling in brain tumor treatment selection.
• Further clinical validation is required before widespread implementation in patient care.