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Basket Trials and the MD Anderson Precision Medicine Clinical Trials Platform
Rabih Said1, Apostolia-Maria Tsimberidou
1From the Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX.
Abstract:
Precision medicine incorporates information regarding tumor biology involved in patients' carcinogenesis and individualized treatment of patients using drugs that inhibit the molecular basis of their disease. Implementation of precision medicine accelerated the drug approval process, translating discoveries in basic science and biotechnology into patient care. Clinical trials with innovative or adaptive design including "basket" and "umbrella" trials explore personalized therapies against in selected tumor types and/or across tumor types. In 2007, we started the Initiative for Molecular Profiling and Advanced Cancer Therapy, the first precision medicine program across tumor types. We demonstrated that therapy matched to patients' tumor molecular profiling is associated with improved rates of response, progression-free survival, and overall survival compared with nonmatched targeted therapy. We have now entered a new era of precision medicine that includes comprehensive tumor testing and multiple innovative modalities hoping to overcome the complexity of tumor biology to improve patient outcomes.
Insights
Precision medicine uses tumor biology for individualized cancer treatment, improving patient outcomes. This approach, including molecular profiling, enhances targeted therapy effectiveness and survival rates.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacogenomics
Background:
- Precision medicine tailors cancer treatment to individual tumor molecular profiles.
- It accelerates the translation of scientific discoveries into clinical practice.
- Innovative clinical trial designs like basket and umbrella trials are key.
Purpose of the Study:
- To evaluate the impact of a precision medicine program on patient outcomes.
- To demonstrate the efficacy of molecular profiling-matched therapy.
- To highlight advancements in precision oncology.
Main Methods:
- Initiative for Molecular Profiling and Advanced Cancer Therapy (IMPACT) program established in 2007.
- Therapeutic matching based on comprehensive tumor molecular profiling.
- Comparison of matched versus non-matched targeted therapies.
Main Results:
- Therapy matched to molecular profiling significantly improved response rates.
- Matched therapy demonstrated superior progression-free survival (PFS).
- Matched therapy was associated with improved overall survival (OS).
Conclusions:
- Precision medicine, guided by molecular profiling, enhances cancer treatment efficacy.
- This approach leads to better patient outcomes, including survival.
- Comprehensive tumor testing and innovative modalities define the future of precision oncology.
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