There is much excitement in the cancer research community about the rapid discovery of biomarkers. These discoveries range from protein signatures that may predict recurrence or response to therapy, to new imaging technologies that measure the extent of drug-target interactions. The combination of these types of biomarker discoveries with studies that are demonstrating the predictive prowess of gene-expression profiles, like the one highlighted in this week’s lead story, is generating tremendous optimism about the future of personalized oncology care.

Of course, advanced technologies have significantly improved our ability to discover potential biomarkers. But biomarker discovery is only the beginning. The harder part is to qualify or validate the biomarker - that is, conduct the clinical studies and develop the correlative data that definitively describe the precise clinical meaning of a given biomarker in a specific situation.

It is for these very reasons - the clinical promise that biomarkers represent and the importance of validating the biomarkers - that, earlier this year, NCI joined with two other Department of Health and Human Services (HHS) agencies, the Food and Drug Administration (FDA) and the Centers for Medicare & Medicaid Services (CMS), to launch the Oncology Biomarker Qualification Initiative (OBQI).

Broadly speaking, OBQI is intended to speed the development and evaluation of new cancer therapies. By bringing together public-private consortia to work on specific projects, OBQI represents the only nationally coordinated effort to validate oncology biomarkers for use in clinical trials.

One specific area of focus is the use of biomarkers as “surrogate endpoints” in clinical trials, meaning that assessments about an intervention’s success or failure could be made after several months of treatment, instead of having to wait years to look at more traditional clinical measures. Of course, patients would still be followed to track longer term outcomes and ensure safety, but validated biomarkers that are proven to correlate with treatment response stand to greatly speed the translation of new therapies and technologies from the lab to patients.

An article in last week’s issue of the NCI Cancer Bulletin explained how NCI is leading an effort to standardize studies to assess whether the results of FDG-PET imaging can be a biomarker of patient response to treatment. Interestingly, one of the first OBQI-supported projects, just recently approved, is a phase III clinical trial that, among several aims, will determine whether FDG-PET can help refine some already established indications of response to treatment for diffuse large B-cell lymphoma (DLBCL).

The trial, being led by CALGB, is important because it is attempting to definitively establish the best treatment protocol for DLBCL, the most common lymphoma type in the United States. Incorporating an FDG-PET biomarker correlative study into this trial (CALGB 50303) represents an important step toward establishing FDG-PET as a valuable component of clinical trials and improved patient care.

A second project that will be supported under OBQI is a phase II trial to assess whether FDG-PET can predict treatment response in patients with advanced NSCLC.

OBQI’s importance is multifold. A truly collaborative effort, it not only will speed drug development, but also will allow FDA to make quicker, more informed decisions about drug approvals, and will enable CMS to make rational coverage decisions for new technologies and interventions. It also will hasten the development of standards, nomenclature, and tools for use in qualifying biomarkers of interest.

Collaborations such as these are no-lose propositions. I expect we will see more like them, and that is good news for cancer researchers and cancer patients.

Dr. John E. Niederhuber
Acting Director
National Cancer Institute