Sarasar (US), SCH 66336
3D model (JSmol)
Except where otherwise noted, data are given for materials in their standard state (at 25 °C [77 °F], 100 kPa).
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Lonafarnib is a farnesyltransferase inhibitor (FTI) that has been investigated in a human clinical trial as a treatment for progeria, which is an extremely rare genetic disorder in which symptoms resembling aspects of aging are manifested at a very early age.
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.
- Liu G, Marrinan CH, Taylor SA, et al. (2007). "Enhancement of the antitumor activity of tamoxifen and anastrozole by the farnesyltransferase inhibitor lonafarnib (SCH66336)". Anticancer Drugs. 18 (8): 923–31. doi:10.1097/CAD.0b013e3280c1416e (inactive 2017-01-16). PMID 17667598.
- “The FTI Drug Lonafarnib”, Progeria Research Foundation. Accessed October 3, 2017.
- "Lonafarnib". NCI Drug Dictionary. National Cancer Institute.
- Ullrich, N. J.; Kieran, M. W.; Miller, D. T.; Gordon, L. B.; Cho, Y.-J.; Silvera, V. M.; Giobbie-Hurder, A.; Neuberg, D.; Kleinman, M. E. (2013). "Neurologic features of Hutchinson–Gilford progeria syndrome after lonafarnib treatment". Neurology. 81 (5): 427–30. doi:10.1212/WNL.0b013e31829d85c0. PMC . PMID 23897869.
- "Experimental Drug Is First To Help Kids With Premature-Aging Disease", NPR, September 24, 2012