NIC researchers develop new Phase 0 cancer trial

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Researchers at the National Cancer Institute have developed a new type of cancel trial that they say may speed up discovery of new drugs. The Phase 0 trial involves giving small doses of a drug to a few patients for a short period of time. The approach tests tolerance and whether the drug can hit a target, giving researchers an idea of whether it's worth advancing the candidate to larger trials.

Phase 0 can be started much earlier in the discovery process because these studies involve smaller drug doses, fewer patients and a shorter trial period than traditional Phase I studies, thus requiring less preclinical data. "For the past several decades there has been a low success rate of new therapies for the treatment of cancer. This has necessitated re-evaluation of the standard anticancer drug development paradigm, of which phase 0 trials will be a key part of a new approach.," said trial leader Dr. Shivaani Kummar.

The method was first put to the test on Abbott Laboratories' experimental cancer med, ABT-888, an enzyme inhibitor designed to be used in conjunction with chemotherapy drugs to make them more effective. The Phase 0 trial demonstrated ABT-888 was safe and had the potential to work against a number of advanced cancers. Data from the Phase 0 trial is now being used to design the Phase I study. "Most importantly, this trial showed that it is possible to enroll a small number of patients, treat them with a low dose of a new drug, identify whether the desired target of the drug was affected, and obtain all of this critical information relatively quickly," the National Cancer Institute said in a statement.

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