News & AnalysisMedicine

Drug Trial Offers Uncertain Start in Race to Save Children With Progeria

See allHide authors and affiliations

Science  28 Sep 2012:
Vol. 337, Issue 6102, pp. 1594-1595
DOI: 10.1126/science.337.6102.1594

You are currently viewing the summary.

View Full Text

Log in to view the full text

Log in through your institution

Log in through your institution

Summary

This week marks the publication of a paper describing the first clinical trial of a drug for progeria, an ultrarare syndrome that seemingly ages children at warp speed. More than anything, the trial highlights just how difficult it can be to test a therapy in a terminal disease with so few patients. Twenty-five children, 75% of those known to have the disease worldwide when the trial began, received lonafarnib, a failed cancer drug that lab studies had indicated might help. The authors are upbeat about the trial's results, but they are in the minority.