Lonafarnib

Lonafarnib is a FTI and a synthetic tricyclic derivative of carboxamide 4-(2-{4-[(11R)-3,10-dibromo-8-chloro-6,11-dihydro-5H-benzo[5,6]cyclohepta[1,2-b]pyridin-11-yl]piperidin-1-yl}-2-oxoethyl)piperidine-1-carboxamide with antineoplastic properties. For those with progeria the drug reduces the prevalence of stroke and transient ischemic attack.

Its inhibits farnesyl transferase able to inhibit RAS signaling pathway through an inbition this enzyme, in patiets with mds or secondary secondary acute mieloid leukaemia.

Part of progerin’s toxic effect comes from attachment of farnesyl-group to the protein so the blocking of this path may show usability in progeria

Association of Lonafarnib Treatment vs No Treatment With Mortality Rate in Patients With Hutchinson-Gilford Progeria Syndrome