PRF-funded, UCLA researchers have taken a Progeria-like mouse model and tested a potential drug treatment for children with Progeria. Their study released in Science Feb.16th has found that this FTI drug improves some signs of the disease.
In September The Progeria Research Foundation was pleased to announce that PRF-funded researchers had published studies supporting a potential drug treatment for children with Progeria – Progeria cells became normalized (in a dish) when given this drug. The next phase of testing has been in animal models, and we are excited to report that a study released in Science Feb.16th has found that this FTI drug improves some signs of disease in a progeria-like mouse model. PRF helped fund UCLA researchers Dr. Loren Fong and Dr. Stephen Young for this latest, vital step towards clinical treatment trials with the children. Click here for more details on this exciting news.
And the scientific publications continue! PRF funded a study by Dr. Karima Djabali, along with Dr. Dayle McClintock and PRF Medical Director Dr. Leslie B. Gordon, which was just published in this week’s PNAS (Proceeding of the National Academy of Sciences). The study concludes that the defective protein produced by the Progeria gene (called progerin) builds up in cells of the children’s vessel walls. This shows us, for the first tine, that there is a direct connection between progerin and heart disease.
And last but certainly not least, the laboratory of Dr. Francis Collins, who discovered the gene for Progeria, has forged ahead and produced a Progeria mouse that carries a genetic defect identical to that of the children. This classic Progeria mouse model shows severe vascular disease, and will be pivotal to our understanding not only how children with Progeria develop heart disease, but even more importantly will be excellent models for testing new treatments such as FTI and genetic therapies in treating and curing Progeria, And this model can also be used to explore cardiovascular disease in general. We are proud to say that our Medical Director, Dr. Leslie B. Gordon, is a co-author. The study appears in this week’s PNAS.
Copies of both PNAS articles can be found at the website of the Proceedings of the National Academies of Science at www.pnas.org
Three studies in one month – WOW! We are really cooking towards the cure. Thank you to all of those researchers who are working hard every day to help children with Progeria and the millions of people who suffer from heart disease throughout the world. And thanks to the families and children, and all of our donors for making this all possible.
TOGETHER, WE WILL FIND THE CURE!