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PRF Continues Aggressive Research Agenda

Results from the Progeria triple drug trial were published online by the journal Circulation on July 11, 2016**. This clinical trial added two additional drugs, pravastatin and zoledronic acid, to the already successful drug lonafarnib. The trial was co-funded by PRF and The National Heart, Lung and Blood Institute, and conducted by an expert team at Boston Children’s Hospital, the #1 children’s hospital in the US.  While no significant improvements were found over and above the lonafarnib single therapy,  PRF continues to identify promising drug candidates that may offer children with Progeria longer, healthier lives – like our new two-drug trial.  This brand new clinical trial adds the drug everolimus to lonafarnib, with the goal of improving the children’s health more than lonafarnib alone.  Click here for more details in our press release.

Meghan, 15 years old, Brennen, 7 years old, and Lindsay, 12 years old, participated in the triple trial.  They had their first visits for the 2-drug trial in June 2016, thanks to your support.

All three children have been taking lonafarnib for several years, and are now adding everolimus in the hopes that the 2 drugs will be more effective together.

In an effort to identify life-saving treatments and the cure for Progeria,  PRF has funded 4 clinical trials at Boston Children’s Hospital, and 62 scientific studies. We are thrilled these trials revealed that lonafarnib improves the children’s cardiovascular system and modestly extends their lifespan.  The triple trial results demonstrate the need for us to continue our aggressive strategy, as the search for more effective treatments, and ultimately the cure, continues.

In an accompanying editorial about the triple trial results, National Institutes of Health Director and study author Francis Collins, MD PhD, wrote, “… additional therapeutic options are emerging, and there is more momentum than ever in the basic and clinical research communities.”

** Gordon, et. al., Clinical Trial of Protein Farnesylation Inhibitors Lonafarnib, Pravastatin and Zoledronic Acid in Children with Hutchinson-Gilford Progeria Syndrome, Circulation, 10.1161/CIRCULATIONAHA.116.022188