National Cancer Institute
When its Chemical Biology Consortium (CBC) is up and running, NCI will have the capacity to bring the skills of hundreds of synthetic and medicinal chemists to bear on a singularly challenging problem. Designed to accelerate the discovery and development of effective, first-in-class targeted therapies, the CBC will choose high-risk targets that are of low interest to the pharmaceutical industry. To take a hypothetical example, a natural product might, theoretically, show effect in targeting a gene revealed by a characterization study to be defective in esophageal cancer. The CBC could rapidly deploy resources in order to synthesize that natural product. CBC chemists might also work on ways to make new compounds water soluble, re-engineer investigators’ assays into high-throughput screens, review data and design optimized analogs, select promising candidates based on established development milestones, and promote candidates with targeted activity to the clinic.
The process for accomplishing CBC goals is largely based on the drug discovery strategy used by the pharmaceutical industry, but dedicated to studies of those new targets that might otherwise not be developed.
“There have been some dramatic changes in the past five to seven years in the way the chemistry and the screening is done, to optimally define and develop probes and then drugs from probes when one discovers a new target,” said James H. Doroshow, M.D., director of NCI’s Division of Cancer Treatment and Diagnosis. “We believe there is this need for us to help facilitate the drug discovery process by providing resources to chemists and biologists who can help work with their own molecules or take molecules in from other academic investigators and help speed the development of not just probes, but eventually drugs that can move along through the pipeline,” he said.
The long-term vision of the CBC is to bridge the gap between basic scientific investigation and clinical research supported by the NCI, as a first step in reinforcing the NCI as a world leader in the area of innovative cancer therapeutics discovery.
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