The Progeria Research Foundation. For the Children. For the Cure.

Peabody, MA: The Progeria Research Foundation – a local charity that is transforming an ultra-rare disease from total obscurity to global awareness and an FDA-approved treatment, and onward toward the cure!



Progeria is an ultra-rare, fatal, genetic, “rapid- aging” disease. Without treatment, children with Progeria die of heart disease at an average age of 14 ½ years.

Children and young adults with Progeria share a unique and similar appearance. Initial signs include skin tightening and growth failure. Other symptoms in the years that follow include loss of body fat and hair, joint stiffness, premature atherosclerosis, as well as heart failure and strokes. The Progeria Research Foundation’s (PRF) mission is to discover treatments and the cure for Progeria and its aging-related disorders, including heart disease, and is the only organization in the world solely dedicated to this undertaking.

PRF was founded in 1999 by a Peabody family, in response to the diagnosis of a family member – Sam Berns - and the complete lack of resources and research on Progeria. Within four years of its founding, the PRF Genetics Consortium discovered the Progeria gene, a collaboration led by Dr. Francis Collins, White House Science Advisor and former Director of the National Institutes of Health (NIH). PRF continues to make extraordinary progress, including the discovery of a now-FDA-approved treatment (lonafarnib), which is giving these children and young adults stronger hearts and longer lives; a lifespan increase of more than a 30% with long-term use of lonafarnib! SO amazing!!

Sam, the nephew of Chamber member and PRF President Audrey Gordon, passed away in 2014 at the age of 17, leaving a legacy that continues to inspire not only those at PRF, but also millions around the world with the Emmy Award-wining HBO documentary, Life According to Sam, and his wildly popular TEDx talk, ‘My Philosophy for a Happy Life,’ which recently surpassed 100 million cross-platform views, available here

Much of PRF’s progress is due to its comprehensive and mission-focused programs and services that transform the care of the children and the field of Progeria research. These programs include the International Progeria Patient Registry that currently contains registrations from 30% of the world’s living Progeria population spanning 50 countries, the Progeria Cell and Tissue Bank that has distributed essential research materials to 218 research labs in 26 different countries, a Medical and Research Database that has culminated in a 130-page patient care handbook, a vibrant research grant funding program that has awarded 85 grants to labs in 15 countries, a private, online community platform to connect families impacted by Progeria, Clinical Drug Trials conducted at Boston Children’s Hospital (BCH), and a thriving series of international scientific workshops that are crucial to defining research and treatment strategies to support growth, collaboration, and success. By centralizing all resources in one entity, PRF has optimized the pace of Progeria research toward the cure at an unparalleled rate.

What was once a completely neglected disease is now at the forefront of scientific interest and efforts. Moreover, scientists have made a definitive connection between Progeria, heart disease and normal aging. Thus, finding the cure for one of the rarest diseases on earth may also help millions of adults with heart disease, and the entire aging population.
 
What’s next for treatments and cure?
In the coming year, in collaboration with Korean-based study sponsor PRG S&T, PRF is hoping to kick off a new clinical trial with a drug called Progerinin which, taken in combination with lonafarnib, may be more effective than lonafarnib alone. In addition, PRF is investing substantial funds into ground-breaking, novel genetic and other drug therapies that will lead to the cure. PRF has recently taken the first patient-involved steps toward a clinical trial in the cutting-edge field of RNA therapy – SO exciting!!

To learn what you can do to help PRF get to the cure faster, please visit www.ProgeriaResearch.org. Thank you!!!
 
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