By Matt Jones
NEW YORK (GenomeWeb News) – The National Cancer Institute plans to fund investigators who will form strategic partnerships in multidisciplinary efforts to conduct molecular analysis on cancer tumors in order to advance and improve patient care and outcomes.
The new program will provide up to $680,000 per year for up to five years to support science that builds on a new understanding about tumor annotation, specifically related to molecular alterations at the DNA, RNA, and protein levels, and how it can be used to develop new clinical tools.
The Strategic Partnering to Evaluate Cancer Signatures (SPECS) II Program will continue efforts started in the SPECS program, which showed that building a research team with all of the necessary expertise for translating a potential diagnostic signature to a clinical assay speeds up the movement of these tools toward the clinic.
Molecular analysis of tumors has already been used in retrospective studies to identify subgroups of patients who have the same diagnosis but different clinical outcomes, according to NCI, but the challenge now is to translate that knowledge into assays for clinical practice.
To move the science to that next level, molecular signatures need to be confirmed in independent studies and robust assays that can be used in the clinical setting need to be developed and validated, NCI said.
"NCI is supporting a number of discovery efforts that are generating huge amounts of information about the biology of tumors," Tracy Lively, associate chief of the Diagnostics Evaluation Branch in NCI's Division of Cancer Treatment and Diagnosis, told GWDN in an e-mail.
"The SPECS program is intended to support and accelerate the effort needed to take a discovery from the research lab and develop it into an assay that could be used to guide therapy for an individual patient and that would be performed by a pathologist in a clinical laboratory," she added.
The SPECS II phase of the program aims to generate publications and data sets that will be accessible to the scientific community for use in developing, designing, and conducting clinical trials and large clinical validation studies that incorporate molecular signatures, and for spurring commercialization of the resulting technologies.
"We are most interested in developing assays providing information to the doctor and patient that is useful in the course of planning therapy for cancer," she noted, adding that these assays could be diagnostic, prognostic, pharmacogenomic, or predictive in nature.
The program's goal is to move molecular analysis of tumors beyond the discovery phase of useful profiles and toward more knowledge about which subsets of elements in profiles need to be measured, which profiles can be reproducibly measured, and showing the clinical utility of the profiles in clinical trials or other large data sets.
NCI designed the program to support multidisciplinary groups of researchers who could pool talent, know-how, and resources with the aim of moving cancer signatures toward the clinic.
"We have seen that it is most cost effective to assemble consortia, usually multi-institutional consortia, focused on one particular clinical question, because rarely can a single institution provide all of the resources required," Lively told GWDN.
She noted that NCI used the SPECS grant mechanism to fund this program because "working with biomarkers that consist of high-dimensional data requires more in the way of technical resources, bioinformatics, and large case sets of specimens" than are available through investigator-initiated grants.
The SPECS II grants will support a range of research approaches involved in advancing molecular signatures, including but not limited to studies related to prognosis for the patient at the time of diagnosis; risk of progression in the early stages of the disease; identification of subsets within a tumor classification, stage, or grade where there is known heterogeneity in clinical behavior, such as in response to treatment with standard drugs or radiation therapy; and in studies predicting patient response to targeted therapies.
"We expect to see multi-analyte biomarkers develop into clinical laboratory assays. We expect that in some cases the assay developed with SPECS support will then go into a prospective clinical trial," Lively said.
"In some cases the researchers will be ready to submit an application to the FDA for clearance or pre-market approval of a new in vitro diagnostic device. In some cases there will be a commercial partner ready to take a test to market. Any or all of these would be desirable outcomes, depending on the use that the new assay is intended to serve," she said.
Ongoing SPECS projects are studying molecular signatures of lung cancer, lymphoma, prostate cancer, sarcoma, leukemia, and breast cancer, but Lively said NCI has "no preference for any particular form of cancer" in this effort.
"We are just as interested in rare cancers, pediatric cancers, and hematologic cancers as we are in the solid tumors prevalent in adults," she said.
NCI has placed the focus for this study on multi-analyte signatures because tumors, not unlike cancer patients, can present layers of biological complexity.
"We expect that as we discover more biology and as we gain more experience in the clinic with targeted agents … we will find that we need to interrogate more than one aspect of the tumor in a given patient to get a picture that is complete enough to be useful," Lively said.