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Every child showed improvement in the first-ever Progeria clinical drug trial, which was funded and coordinated by The Progeria Research Foundation
For The Progeria Research Foundation:
Megan Lustig (PRF)
Office: (202) 955-6222
Mobile: (301) 467-7869
For Boston Children’s Hospital:
RELEASE DATE: September 24, 2012: Results of the first-ever clinical drug trial for children with Progeria, a rare, fatal aging disease, demonstrate the efficacy of farnesyltransferase inhibitors (FTIs). The clinical trial, completed at Boston only six years after scientists identified the cause of Progeria, showed that the FTI lonafarnib had a positive effect on weight gain, hearing, bone health and, most importantly, cardiovascular health. The study results were published today in Proceedings of the National Academy of Sciences (Epub ahead of print).
“To discover that some aspects of damage to the blood vessels in Progeria can not only be slowed by the FTI lonafarnib, but even partially reversed within just 2.5 years of treatment is a tremendous breakthrough, because cardiovascular disease is the ultimate cause of death in children with Progeria,” said Leslie Gordon, M.D., Ph.D., lead author of the study, and Medical Director of PRF, and mother of a child with Progeria.
Progeria Linked to Normal Aging Process
Previous research shows that the Progeria-causing protein progerin is also produced in the general population and increases with age. Researchers plan to continue researching the effect of FTIs, which may help scientists learn more about the cardiovascular disease that affects millions, as well as the normal aging process.
PRF’s Impressive Record
“PRF provides a model for disease research organizations, and is a good example of a successful translational research organization, moving from gene discovery to clinical treatment at an unprecedented pace,” said Dr. Kieran. “From 1999, when the organization was founded, to today, PRF has identified the genetic mutation that causes the disease, funded preclinical research, completed this trial, initiated a second trial, and is currently working with our team here at Boston Children’s Hospital to plan yet another trial with drugs that, like FTIs, have shown exciting results in Progeria cells and animal models. That’s a remarkable track record of accomplishment.”
CLICK HERE for the press release (PDF)
* This footage is offered free and unrestricted for broadcast use on behalf of
The Progeria Research Foundation.
Leslie B. Gordon, MD, PhD
Clinical Trial Co-Chair
Medical Director, The Progeria Research Foundation
Download Broadcast Quality File (2:22 / 720x480 / MP4 / 161 MB)
Mark Kieran MD, PhD
Clinical Trial Principal Investigator
Director, Pediatric Medical Neuro-Oncology, Children’s Hospital Boston/Dana-Farber Cancer Institute
Download Broadcast Quality File (1:49 / 720x480 / MP4 / 133 MB)
Audrey S. Gordon, Esq.
President and Executive Director, The Progeria Research Foundation
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Sandy and Steve Nighbor
Parents of Clinical Trial Participant Megan
Download Broadcast Quality File (1:49 / 720x480 / MP4 / 132 MB)
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