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Health Agencies Update
October 26, 2005

Cancer Drug and Progeria

JAMA. 2005;294(16):2016. doi:10.1001/jama.294.16.2016-a

Researchers have found that a class of experimental anticancer drugs may hold promise for treating Hutchinson-Gilford progeria syndrome (commonly called progeria), a fatal genetic disorder that causes premature aging. The disorder has no current treatment and is estimated to affect one child in 4 million.

In laboratory studies led by the National Human Genome Research Institute, farnesyltransferase inhibitors—drugs in clinical trials for treating patients with myeloid leukemia, neurofibromatosis, and other conditions—were able to reverse the nuclear structure abnormalities that are the hallmark of cells from children with progeria (Capell et al. Proc Natl Acad Sci U S A. 2005;102:12879-12884).

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