Lonafarnib clinical trial for progeria has scientific roots in basic biology discoveries

Published on September 26, 2012 at 5:12 AM · No Comments

The good news widely reported this morning of positive results from a clinical drug trial at Boston Children's Hospital for the previously "untreatable" rapid aging disorder in children known as progeria has its scientific roots in basic biology discoveries made in recent years.

A paper published Monday in the Proceedings of the National Academy of Sciences (PNAS) reports that the use of farnesyl transferase inhibitors (FTI) significantly slows the progress of progeria, a rare and until now "untreatable" lethal genetic disorder. Also known as Hutchinson-Gilford Progeria Syndrome (HGPS), progeria has been described as out-of-control rapid aging in children. A ""normal"" baby born with HGPS will stop growing by 16-18 months and quickly develop signs of old age including hair loss, thin skin, osteoporosis and, most dangerously, progressive arteriosclerosis. By 10 years of age progeria children appear to be 80. The PNAS paper apparently shows a significant slowing of bone loss and blood vessel blockage.

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