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Updated: Jul 29, 2026
10:49
Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
Historical and methodological developments in clinical trials at the National Cancer Institute.
1Department of Biomathematics, University of Texas M. D. Anderson Cancer Center, Houston 77030.
Statistics in Medicine
|August 1, 1990
Summary
The National Cancer Institute (NCI) pioneered randomized clinical trials for acute leukaemia in 1955. Early advancements included combination therapies and statistical methods to improve cancer treatment outcomes.
Area of Science:
- Oncology
- Clinical Trials Methodology
- Biostatistics
Background:
- The National Cancer Institute (NCI) initiated its first randomized clinical trial for acute leukaemia in 1955.
- The program was significantly influenced by C. Gordon Zubrod, who established randomized trials and cooperative clinical trials at NCI.
- Marvin Schneiderman played a key role in recruiting biostatisticians and establishing statistical centers for early trials.
Purpose of the Study:
- To outline the foundational principles and early development of randomized clinical trials at the NCI.
- To highlight key methodological innovations in clinical trial design and statistical analysis during the period 1955-1965.
- To describe the evolution of treatment concepts, including combination therapy and maintenance of remission.
Main Methods:
- The study reviews the historical progression of clinical trials at NCI, from planning in 1954 to the mid-1960s.
- It details the standard sequence of clinical trials: Phase I (dosage), Phase II (efficacy), and Phase III (treatment comparison).
- Key methodological developments are identified, including specific statistical tests and models relevant to survival data analysis.
Main Results:
- The early adoption of randomization and statistical analysis principles facilitated rigorous clinical research.
- Innovative treatment strategies emerged, such as combining therapies and administering treatment during remission.
- Significant methodological advancements were made, including the Gehan plan for Phase II trials and the Mantel-Haenszel test for survival data.
Conclusions:
- The early randomized clinical trials program at NCI laid the groundwork for modern cancer research.
- The integration of biostatistics and innovative treatment strategies significantly advanced cancer patient care.
- Methodological contributions from this era continue to influence clinical trial design and analysis today.
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