"BATTLE is an important step toward personalized medicine and marks a paradigm shift for clinical trials by demonstrating the feasibility of a biopsy-based, hypothesis-driven biomarker trial," said Roy Herbst, M.D., Ph.D., professor in the Department of Thoracic/Head and Neck Medical Oncology and co-principal investigator on the BATTLE clinical trials.
Patients agreed to have a new biopsy for the trial, Kim said, which was crucial to the study design because it provided fresh information on the tumor's molecular status that may have been altered by treatment since the patient's previous biopsy.
"BATTLE employed an adaptive randomization approach that allowed the statistical model to learn as the clinical trial progressed," said J. Jack Lee, Ph.D., professor in M. D. Anderson's Department of Biostatistics.
The first 97 patients were equally randomized to BATTLE's four arms. As the study progressed, information from patients' biopsies and outcomes was employed by the model to guide assignment of drugs to new patients, who became more likely to receive a drug that had worked for earlier patients with the same tumor biomarkers.
The model leads to greater use of successful drugs and minimization or dropping of those less successful. While vandetanib helped those with VEGFR overexpression, it was dropped for patients with the KRAS mutation, Lee said.
By identifying biomarkers, and thus a potential patient population for a drug at Phase II, a follow-up Phase III will require smaller sample sizes and proceed more quickly than an all-comers trial with thousands of patients, Lee said.
Kim said future BATTLE trials will test combinations of therapies as well as single agents and will concentrate on the entire range of staging fo
|Contact: Scott Merville|
University of Texas M. D. Anderson Cancer Center