Progeria Quick Facts
MISSION: To Discover Treatments and the Cure for Hutchinson-Gilford Progeria Syndrome and its Aging-related Disorders
PRF BY THE NUMBERS:
- Identified children living with Progeria: 100* in 36 countries
- PRF-sponsored Progeria Clinical Drug Trials: 3
- Grants funded: 45, totaling nearly $5 million
- Cell lines in the PRF Cell & Tissue Bank: 189
- Children in PRF’s Medical & Research Database: 120
- International Scientific Workshops on Progeria: 7
- Number of languages into which PRF’s program and medical care materials are translated: 20
- Increase in scientific publications since the Progeria gene discovery: 1,500%
*Includes 10 children in the Progeroid Laminopathy category that have a mutation in the Lamin pathway, but do not produce progerin
What is Progeria?
Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), is a rare, fatal genetic condition of accelerated aging in children. All children with Progeria die of the same heart disease that affects millions of normal aging adults (arteriosclerosis), but instead of occurring at 60 or 70 years of age, they may suffer strokes and heart attacks even before age 10. Remarkably, the intellect of children with Progeria is unaffected, and despite startling physical changes in their young bodies, these extraordinary children are intelligent, courageous, and full of life.
What is PRF?
The Progeria Research Foundation (PRF) was established in 1999 by the parents of a child with Progeria, Drs. Leslie Gordon and Scott Berns, and many dedicated friends and family who saw the need for a medical research resource for the doctors, patients, and families of those with Progeria. Since that time, PRF has been the driving force behind the Progeria gene discovery and the first-ever Progeria drug treatment. PRF has developed programs and services to aid those affected by Progeria and the scientists that conduct Progeria research. Today, PRF is the only non-profit organization solely dedicated to finding treatments and the cure for Progeria. PRF is hailed as a prime example of a successful translational research organization, moving from creation, to gene discovery, to first-ever drug treatment in just 13 years.
Total Dollars Raised
1999 through 2012: $13,756,467
85% of PRF’s annual expenses are consistently dedicated to its programs and services.
The support we have received made the Progeria gene discovery, the Progeria clinical trials and all of our other extraordinary progress possible. With the help of current and new supporters, we will win this race against time and find treatments and the cure for these special children. Moreover, Progeria discoveries are teaching us about the heart disease and the aging that affects us all.
PRF’S PROGRAMS & SERVICES
- First-Ever
Progeria Clinical Drug Trials and
Treatment: PRF-Sponsored Clinical Drug Trials bring children
from around the world for promising treatments that may help to improve disease, and may even extend the lives of children
with Progeria. In 2012, history was made with
the discovery that a farnesyltransferase
inhibitor, or FTI, is the first-ever treatment for Progeria - a remarkable step forward in the pursuit of a
cure. PRF continues to
explore other promising drugs, and is currently co-funding and coordinating the
“Triple Drug Trial”, testing FTIs with 2 other drugs. In total, PRF has brought 47 children from 24 countries to Boston for treatment trials.
- PRF’s International Progeria Registry maintains centralized information on children and families living with Progeria. This assures rapid distribution of any new information that may benefit the children.
- Cell & Tissue Bank: PRF’s Bank provides researchers with genetic and biological material from Progeria patients and their families, so research on Progeria and other aging-related diseases can be performed to bring us closer to the cure. PRF has collected an impressive 113 cell lines from affected children worldwide (ages 2 months to 17 years) and 71 lines from their immediate relatives, including 9 Induced Pluripotent Stem Cell (iPSC) lines.
- Medical & Research Database: The Database is a centralized collection of medical information from Progeria patients worldwide. The data is rigorously analyzed to help us understand more about Progeria and devise treatment recommendations. In 2010, this analysis contributed to PRF’s comprehensive healthcare Handbook on Progeria aimed at optimizing quality of life. The Handbook is available in English, Spanish and Portuguese.
- Diagnostic Testing: This program was developed in the wake of the 2003 gene discovery so that children, their families and medical caretakers can get 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 7 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 disease.
- Research Grants: PRF has awarded 43 research grants totaling over $3.5 million through peer review by our volunteer Medical Research Committee. Awards of up to $100,000 per year, for up to three years, have allowed innovative new research in Progeria to thrive.
- Publications and Research: Both clinical and basic scientists have utilized the PRF grants, cells and tissues, and database; their discoveries are published in top-notch scientific journals. The average annual number of scientific publications on Progeria since 2002 is more than 15 times that of the previous 50 years.
- PRF Translation Program: In touch with the world. With a prominent global presence, PRF eliminates barriers to communication for patients and their families around the world. This initiative has succeeded in translating PRF program and medical care materials into 20 different languages.
- Public
Awareness: ProgeriaResearch.org provides access to the
latest information on Progeria research and support for families. PRF’s
newsletters reach over 30,000 people in 61 countries. PRF’s story has appeared on CNN, The Dr. Oz
Show, Primetime, Dateline and The Today Show, in Time and People
magazines, The New York Times, The Wall Street Journal and many other
widely-read media outlets. PRF and its partner GlobalHealthPR also manage a
global awareness campaign called Find the
Other 150, to further PRF’s efforts to find children
with Progeria worldwide so they can get the unique help they need.
WHO’S WHO AT PRF?
Audrey Gordon, Esq., President and Executive Director, Co-Founder
Ms. Gordon oversees all administrative aspects of The Progeria Research Foundation, including the Board of Directors’ activities, fundraising, grant submissions, medical research projects administration, public awareness initiatives, and volunteer programs.Leslie B. Gordon, MD, PhD, Medical Director, Co-Founder
Dr. Gordon co-founded PRF with friends and family after her son, Sam, was diagnosed with Progeria. Dr. Gordon oversees PRF’s research-related programs, and is a co-chair for the Progeria clinical drug trials. She is an Associate Professor of Pediatrics Research at the Warren Alpert Medical School of Brown University and Hasbro Children’s Hospital in Providence, RI, and a Staff Scientist at Boston Children’s Hospital and Harvard Medical School.Scott D. Berns, MD, MPH, FAAP, PRF Chairman of the Board, Co-Founder
Dr. Berns, Sam’s father, is a co-founder of The Progeria Research Foundation and serves as Chairman of the Board. He is a Board Certified Pediatrician and Clinical Professor of Pediatrics at the Alpert Medical School of Brown University. He is also Deputy Medical Director and Senior Vice President of Chapter Programs at the March of Dimes.Tina, Brandon, Brittany and Zach Pickard, PRF’s Ambassador Family
The family of 5-year-old Zach lives in Lexington, Kentucky, and is an integral part of PRF’s efforts to raise public awareness, involve other families in our programs, and raise funds for research. We appreciate the time and effort they devote to these important activities.
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