Lonafarnib

Lonafarnib is a farnesyltransferase inhibitor (FTI) that is being investigated in a human clinical trial as a potential treatment for progeria.

Lonafarnib is a synthetic tricyclic derivative of carboxamide with antineoplastic properties. As such, it is used primarily for cancer treatment. For those with progeria, research has shown that the drug reduces the prevalence of stroke and transient ischemic attack, and the prevalence and frequency of headaches while taking the medication. A phase II clinical trial was completed in 2012, which showed that a cocktail of drugs that included lonafarnib and two other drugs, met clinical efficacy endpoints that improved the height and diminished the rigidity of the bones of progeria patients.