Statistically, there are another 150-200 children unknown and untreated, 2/3 of whom we believe are in India and China
The Progeria Research Foundation was founded in 1999 and is currently aware of 166 children and young adults worldwide that are living with Progeria
An interview with Audrey Gordon, President and Executive Director of PRF.
The mission of finding 60 in India, your comments?
We’re currently aware of 166 children and young adults worldwide with Progeria. Statistically, there are another 150-200 children unknown and untreated, 2/3 of whom we believe are in India and China. For this reason, we launched our campaign, Find the Children – 60 in India with Progeria in 2019. In affiliation with MediaMedic Communications based in Mumbai, our campaign leverages partnerships with local associations, physicians, and government institutions, as well as digital and traditional media outlets to assure the greatest possible reach in such a vast, diverse and multi-lingual country as India.
What is the objective of The Progeria Research Foundation(PRF) ?
PRF is the only organization in the world whose mission is to discover treatments and the cure for Progeria and its aging-related disorders, including heart disease. Over the course of PRF’s 20-year history, this rare condition has gone from obscurity to gene finding, to the first Progeria clinical trials, to a first-ever treatment, to worldwide awareness – all at a pace virtually unheard of in the scientific community. And while helping this small population of children, Progeria’s connection to common heart disease and aging has tremendous implications for us all.
The Progeria Research Foundation has many services to help these children, including genetic testing to confirm diagnosis, a cell and tissue bank, treatment guidelines for daily care, clinical drug trials, and programs that enable those with Progeria to access the treatment lonafarnib. But in order to help these children, we first have to find these children. Please help us on this quest to find children with Progeria! To learn more, please visit www.progeriaresearch.org.
What are the medical developments so far?
Through Progeria clinical drug trials funded and coordinated by The Progeria Research Foundation (PRF), we’vediscovered a treatment, lonafarnib, which has been shown to give children with Progeria stronger hearts and longer lives. Without treatment, children with Progeria die of heart disease at an average age of 14.5 years.
Since 2007, PRF has funded and led four clinical trials, all of which have taken place at Boston Children’s Hospital,that have included the study of lonafarnib’s effect, and the effect of other drugs in combination with lonafarnib, on Progeria. These trials have involved children from over 30 countries, including India. Last year, EigerBioPharmaceuticals,with whom PRF is working closely, submitted to the U.S. Food and Drug Administration (FDA) the first part of an application seeking approval of lonafarnib as a treatment for Progeria. FDA approval would enable access to lonafarnib by prescription in many countries, including India.While we are thrilled that lonafarnibimproves some aspects of Progeria, we continue to search for even more effective treatments, andultimately the cure.
To this end, we also invest in the exploration of drugs targeting other biological pathways,as well as the cutting-edge gene editing field, which is exploding at a rapid pace.
How does PRF help children get treatment?
PRF and our partner, EigerBioPharmaceuticals, have found a way to help get the drug lonafarnib to children with Progeria who live outside the U.S., by way of a Managed Access Program (MAP). MAP allows every qualified child and young adult with Progeria to get lonafarnib in their home countries where MAP is available, including India. This program helps to ensure that anyone with Progeria can have a chance at longer, healthier lives, while the search for better treatments and cure continues.