The FDA and ASCO, along with the American Society of Hematology (ASH) and the American Association for Cancer Research (AACR), convened April 18, 2012, for the “Minimal Residual Disease (MRD) as a Surrogate Endpoint in Acute Lymphoblastic Leukemia (ALL) Workshop”—the first in a series of workshops intended to bring together researchers, advocates, and pharmaceutical and diagnostic device industries to help develop procedures for qualifying MRD as a biomarker of efficacy or response to treatment.

ASCO 2011-2012 President Michael P. Link, MD, served as a panelist and members Stephen Hunger, MD, and Gregory Reaman, MD, served as Co-Chairs. “In most cases of ALL treated with current, state-of-the-science, multi-agent chemotherapy regimens, early MRD has surpassed, in importance, other widely used and accepted prognostic indicators,” according to a workshop briefing document. “As a prognostic biomarker, assessment of MRD has had a profound impact worldwide on the design and conduct of clinical trials in ALL for all known risk groups of patients, currently defined by longstanding, accepted clinical and biological prognostic factors.”

Workshop objectives were to:

• Establish qualification of early MRD as an efficacy/response biomarker in ALL
• Reach a consensus position on ZZEarly MRD (critical threshold value) as a surrogate endpoint
• Preferred technology platform for MRD determination with defined performance characteristics
• Standardized methodology and need for centralized performance
• Determine the need for FDAapproved in vitro diagnostics

The next step on the ALL forefront is for the FDA to bring discussion questions to its Oncologic Drugs Advisory Committee. From there, the FDA will create draft guidance, which ASCO will respond to with comments. Concurrently, ASCO will be helping to organize upcoming FDA leukemia workshops, tentatively slated for February 2013.