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Historic Moment in Premature Child Aging - Progeria Clinical Trial

Date: 2009-07-23 12:51:44 Member: Richard Persen Category: Research Discipline: Clinical / Health / Medicine Link: http://www.progeriaresearch.org/clinical_trial.html

The Progeria clinical drug trial began May 7, 2007 with two children arriving in Boston, MA for their first of seven visits over a two-year period. During the first visit, they were given extensive tests, and their first doses of the FTI drug. Approximately two families have been flying to Boston each week since May, and in October 2007, the trial became fully enrolled. The trial is expected to end in October 2009, with results published in 2010.

The drugs investigated are farnesyltransferase inhibitors, or FTIs. These are a class of drugs that can reverse an abnormality in Progeria cells in the laboratory. The nucleus (plural nuclei) is the structure at the center of each cell that contains DNA. Unlike the round nuclei from normal cells, Progeria cells have abnormally shaped nuclei. These abnormally shaped nuclei with multiple “lobes” can even look like a cluster of grapes or bubbles. In the laboratory, treating Progeria cells with FTIs restored their nuclei to a normal appearance

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