• First-Ever Progeria Clinical Drug Trials for Progeria: PRF is funding and co-coordinating a $2 million, first-ever clinical drug trial for Progeria, taking place in Boston with partners Children’s Hospital Boston (CHB), Dana Farber Cancer Institute and Brigham and Women’s Hospital. . The trial drug is called a farnesyltransferase inhibitor, or FTI, a drug that has shown great promise in the laboratory and in animal models.  28 children from 16 countries are enrolled, ages 3 to 15 years, and it will end in December 2009. Trial results are expected to be published in a scientific journal in 2010. If the drug proves effective, it will be a remarkable step forward in the pursuit of a cure.
  
  Since the start of the first trial, researchers identified two additional drugs that, when used in combination with the current FTI drug being tested, may provide an even more effective treatment for children with Progeria than the single drug. Not wanting to wait until the first trial was complete to save precious time in the quest to find effective treatments, PRF moved quickly to explore these additional treatment options. PRF and CHB began a second clinical trial for Progeria in August 2009. The "Triple Drug Trial" is much larger than the first, open to up to 45 children: those in the current trial and another 13-18 children that were either too young for the first trial, or not known to PRF during enrollment for that trial.

• Cell & Tissue Bank: provides medical researchers with genetic and biological material from Progeria patients and their families, so that research on Progeria and other aging-related diseases can be performed to bring us closer to finding the cure.  PRF has collected an impressive 71 cell lines from affected children worldwide (with ages ranging from 8 months to 17 years), and 40 lines from their immediate relatives.
  

• Medical & Research Database: The database is a collection of medical records of Progeria patients from all over the world. The data is rigorously analyzed to determine the best course of treatments to improve the patient’s quality of life. This information is invaluable for the attending physician to guide the family through the best course of action.  To date, PRF has enrolled 77 children in the program, and published three formal healthcare recommendations: Cardiac Care, Nutrition, and Occupational Therapy/Physical Therapy. The database was critical in determining the primary clinical outcome parameter for the first-ever clinical drug trial for Progeria.
  

• Diagnostic Testing: PRF developed a diagnostic test for Progeria in the wake of the 2003 gene discovery so that children, their families and medical caretakers can for the first time be given a definitive, scientific diagnosis. This can translate into earlier diagnosis, fewer misdiagnoses and early medical intervention to ensure a better quality of life for the children.
  

• Scientific Workshops on Progeria: PRF has organized five scientific conferences that have brought together scientists and clinicians from all over the world to share their expertise and cutting edge scientific data, and foster collaboration in the fight against this devastating, lethal disease.
  

• Publications and Research: A major goal of PRF is to promote awareness about Progeria and the progress being made in the field of Progeria research.  Dozens of publications on Progeria, many of which acknowledge PRF grant support or PRF’s support of providing cells, have been published in well-known, respected scientific journals read by thousands of researchers worldwide. The average number of scientific publications on Progeria since 2002 is more than 10 times that of the previous 50 years.
  

• Research Grants: PRF has awarded 24 research grants totaling $1.7 million through peer review by our volunteer Medical Research Committee. Awards of up to $100,000, for up to two years, have allowed innovative new research in Progeria to thrive.
  

• Web Site/Public Awareness: ProgeriaResearch.org provides visitors with access to the latest information on Progeria research, support, and education for families and caregivers. PRF’s Web site enjoys an average of approximately 15,000 visitors per month, and PRF’s newsletters reach nearly 10,000 people in 49 countries. Since the gene discovery, PRF’s story has appeared on CNN, Primetime Live, Dateline, The Today Show, in Time magazine, People magazine and The New York Times, and dozens of other media outlets that have a keen interest in Progeria and PRF’s work. In the summer of 2006, PRF kicked off a national public awareness campaign featuring the voices of Ted Danson and Mary Steenburgen. The PSA was endorsed by the Advertising Council, and aired on the Astrovision in Times Square. In January 2007, PRF and the Progeria Clinical Drug Trial were featured on the front page of The Wall Street Journal, reaching millions of readers worldwide.
  
• Progeria Family Outreach - The PRF Progeria Family Message Board and Chat Room: In keeping with our mission of continuing to provide valuable resources for families of children with Progeria, PRF has created a private message board Web site. This on-line tool helps the families get to know each other, and develop a support network of people with whom they can share concerns and ideas on how best to care for their children. 

WHO’S WHO AT PRF?