July 20, 2006: PRF-funded researchers continue to drive Progeria research to new levels of progress in our quest for the cure. Published in today's online edition of the Journal of Clinical Investigation
, the findings support the start of human clinical trials with the cancer drug, farnesyltransferase inhibitor (FTI).
PRF is funding a clinical drug trial for Progeria
, which began on May 7th. The trial is treating the children with a drug called FTI. This is a critical time to help PRF, as we are raising money to fund the trial.
Excerpts taken from the Journal of Clinical Investigation:
Cancer Drug Improves Progeria: New Mouse Model Closest to Disease in Humans
Using their new mouse model of progeria, UCLA Investigators 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. This new mouse model closely mirrors the genetic defect causing many cases of progeria in humans.
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.
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.