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This Week in NEJM: Mar 24, 2011


In NEJM this week, Bruce Chabner says the "striking" results of recent Phase I trials for targeted cancer drugs have started a discussion about shortening the process required to get approval from regulators. Normally, it takes a cancer drug about seven years to go from human trials to FDA approval, but the news of success in Phase I trials reaches physicians early and creates demand for access to the drug, Chabner says. New understanding of the hand genetics plays in cancer development has only "sharpened" the discussion by enabling rapid development of targeted therapies, he adds, and because patient subgroups with high response rates to certain drugs can now be defined, Phase I trials are becoming even more successful. The mechanism of "accelerated approval," which the FDA introduced in 1992 to quickly approve drugs meant for life-threatening diseases with no effective treatment, could be used in the case of targeted cancer drugs that show promise in Phase I trials, Chabner says. "Early approval would allow rapid general access to treatment, while further evaluation focused on defining optimal doses, schedules, and drug combinations; long-term benefits; toxic effects; and resistance mechanisms," he adds.

Published online in advance in NEJM this week, a group of clinicians ask whether the link between vitamin D and cancer prevention is ready for "prime time." Given the potential of vitamin D to prevent cancer, many were surprised that an increase intake of the vitamin didn't figure prominently in the new Dietary Reference Intakes established by the Institute of Medicine. Although IOM concluded that vitamin D plays an important role in bone health, it was determined that the evidence linking vitamin D to cancer and other diseases was "inconsistent and inconclusive as to causality," the authors write. The premise is biologically plausible, they add, but more evidence is needed.

The Scan

Germline-Targeting HIV Vaccine Shows Promise in Phase I Trial

A National Institutes of Health-led team reports in Science that a broadly neutralizing antibody HIV vaccine induced bnAb precursors in 97 percent of those given the vaccine.

Study Uncovers Genetic Mutation in Childhood Glaucoma

A study in the Journal of Clinical Investigation ties a heterozygous missense variant in thrombospondin 1 to childhood glaucoma.

Gene Co-Expression Database for Humans, Model Organisms Gets Update

GeneFriends has been updated to include gene and transcript co-expression networks based on RNA-seq data from 46,475 human and 34,322 mouse samples, a new paper in Nucleic Acids Research says.

New Study Investigates Genomics of Fanconi Anemia Repair Pathway in Cancer

A Rockefeller University team reports in Nature that FA repair deficiency leads to structural variants that can contribute to genomic instability.