Cancer Drug Improves Progeria: New Mouse Model Closest to Disease in Humans

FINDINGS: Newswise — UCLA investigators found that an experimental cancer drug improved signs of progeria in a new mouse model of progeria. This new mouse model closely mirrors the genetic defect causing many cases of progeria in humans. Progeria is a rare disease causing accelerated aging and cardiovascular disease in children.

IMPACT: The UCLA team has been at the forefront of progeria research. Using their new mouse model of progeria, they found that an experimental drug, called a farnesyltransferase inhibitor, improved bone density, reduced bone fractures, delayed the onset of the disease, and helped with weight gain. The effects of the drug were more dramatic than in previous studies using other animal models. These findings will help researchers target drug therapies for children with progeria and lead to a better understanding of mechanisms of disease. The UCLA findings have made it feasible to consider human clinical trials.

AUTHORS: UCLA researchers include Dr. Shao H. Yang, Loren G. Fong, Ph.D., and Dr. Stephen G. Young of the David Geffen School of Medicine at UCLA.

FUNDING:The study was funded by the National Institutes of Health, the American Heart Association, and the Progeria Research Foundation.

JOURNAL:The study appears in the July 20 online edition of The Journal of Clinical Investigation. A PDF of the full study is available.