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Pilot studies for personalized cancer medicine: focusing on the patient for treatment selection
Leticia De Mattos-Arruda1, Jordi Rodon
1Molecular Therapies Research Unit, Vall d'Hebron Institute of Oncology, Vall d'Hebron University Hospital, Barcelona, Spain; Universitat Autònoma de Barcelona, Barcelona, Spain.
Abstract:
Advances in diagnostics and targeted therapies during the past decade have changed how oncology is viewed. "Stratified medicine" has emerged from the accumulated evidence garnered from matching targeted therapies with tumor molecular aberrations. Concomitantly, current knowledge derived from large-scale, massively parallel sequencing technologies and global research initiatives such as the international 1000 Genomes Project, the Cancer Genome Atlas, the International Cancer Genome Consortium, and publicly available catalogs such as the Catalogue of Somatic Mutations in Cancer and Genomics of Drug Sensitivity in Cancer have illuminated the utility of understanding the molecular basis of cancer through genome analysis. In addition, multiple collaborative efforts are widening the possibility of universally personalizing cancer care. Although several key challenges of personalized cancer medicine (PCM) need to be addressed, some pilot studies are transforming the way we analyze tumor tissue molecular aberrations, design clinical trials, and measure treatment efficacy. Taken together, these pilot studies are paving the way for clinical trials that are designed to empirically test the concept of PCM. In this paper, we describe lessons learned from the first pilot initiatives of PCM and how this knowledge is being used to design novel clinical trials.
Insights
Personalized cancer medicine (PCM) uses tumor molecular analysis to match targeted therapies. Lessons from early PCM pilot studies are guiding the design of new clinical trials for tailored cancer care.
Area of Science:
- Oncology
- Genomics
- Translational Medicine
Background:
- Advances in diagnostics and targeted therapies have revolutionized oncology.
- Stratified medicine has emerged, linking targeted therapies to specific tumor molecular aberrations.
- Large-scale sequencing initiatives (e.g., Cancer Genome Atlas) highlight the importance of genome analysis in understanding cancer.
Purpose of the Study:
- To describe lessons learned from initial personalized cancer medicine (PCM) pilot initiatives.
- To explain how this knowledge is informing the design of novel clinical trials.
- To discuss the transformation of cancer care through molecular analysis and collaborative efforts.
Main Methods:
- Review of findings from early personalized cancer medicine pilot studies.
- Analysis of data from large-scale genomic projects and cancer mutation catalogs.
- Examination of collaborative efforts in cancer research and treatment.
Main Results:
- Pilot studies are transforming the analysis of tumor molecular aberrations.
- Clinical trial design and efficacy measurement are being redefined by PCM principles.
- Early initiatives provide valuable insights for future PCM research and application.
Conclusions:
- Personalized cancer medicine (PCM) is advancing through integrated genomic analysis and targeted therapies.
- Lessons from pilot studies are crucial for developing effective, personalized cancer treatment strategies.
- Collaborative research and data sharing are essential for the widespread adoption of PCM.
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