Rare Disease Report

PRF Awards Four More Research Grants

OCTOBER 12, 2016
Progeria Research Foundation

Since 1999, PRF has provided over $7 million to fund 64 grants for Progeria-related research projects performed in 18 states and 13 countries. The projects have led to important discoveries about Progeria, heart disease, and aging, as interest in Progeria research continues to thrive.


Jed William Fahey, ScD
Assistant Professor and Director, Cullman Chemoprotection Center, Johns Hopkins School of Medicine, Department of Pharmacology & Molecular Sciences, and Bloomberg School of Public Health, Department of International Health, Center for Human Nutrition, Baltimore, MD.
 “The capacity of plant-derived isothiocyanates to surpass the efficacy of sulforaphane, with reduced toxicity to Progeria cell lines.”
Recent studies have shown that the chemical sulforaphane, which is found in broccoli, improves the health of cultured cells derived from children with Progeria.  However, the doses likely needed to treat people are much higher, and might cause unacceptable toxicities.  Dr. Fahey will test over 100 compounds closely related to sulforaphane, to examine whether any are better at improving signs of Progeria but not cause cellular toxicity, at doses that might be reasonable for humans.


Silvia Ortega-Gutiérrez, PhD
Associate Professor, Universidad Complutense de Madrid, Spain; Ramón y Cajal Scholar, Fulbright Scholar, The Scripps Research Institute, La Jolla, CA.  
“New isoprenylcysteine carboxylmethyltransferase (ICMT) inhibitors for the treatment of progeria"
A chemical called an ICMT inhibitor has been shown to improve Progeria in the laboratory, by blocking the pathway used by cells to produce the toxic protein progerin.   Dr. Ortega and her team aim to develop new ICMT inhibitors for the treatment of the Progeria by performing what drug companies call “medicinal chemistry”, where a chemical is developed for use as a medicine in humans. 
Roland Foisner, PhD
Professor of Biochemistry, Medical University Vienna and Deputy Director, Max F. Perutz Laboratories, Vienna, Austria. Editor-in-Chief, Journal Nucleus.
“Contribution of endothelial cell dysfunction to cardiovascular disease in progeria and implications for diagnostic and therapeutic targets”
Artery walls contain several layers containing different types of cells that can play different roles in cardiovascular disease development, including a middle layer of smooth muscle cells, and the inner layer of endothelium. Dr. Foisner has created a mouse model expressing progerin in the vascular endothelium. He will investigate how progerin impairs endothelial cell function and how this affects heart function.
Juan Carlos Izpisua Belmonte, PhD
Professor, Gene Expression Laboratories at The Salk Institute for Biological Studies, La Jolla, CA; 
“The use of novel technologies to identify and validate potential therapeutic compounds for the treatment of Hutchinson-Gilford Progeria Syndrome”
Dr. Belmonte and his team will use a well-established technique pioneered in their lab to model the vascular smooth muscle characteristics associated with the most acute symptoms of heart disease in children with Progeria.  Using these cells, they will perform a high-throughput screening to discover compounds that prevent progerin production and improve cell shape.

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