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 May 7, 2007 - October 2009: First-Ever Progeria Clinical Drug Trial Marks Historic Moment in Progeria Research History!

                 
After only 7 years in existence, we became scientifically ready to begin this trial with a drug that shows great promise to effectively treat children with Progeria, and in the process we may help millions of older people who suffer from heart disease and other, aging-related conditions.
Researchers have now identified a potential drug treatment for children with Progeria, called FTIs.  For the first time, we have in front of us a possible treatment for children with Progeria. Exciting times!
 
The Progeria clinical drug trial began on May 7th, 2007 with two children arriving in Boston, MA for their first of seven visits over a 2-year period. At this first visit, they were given extensive tests and their first doses of the drug. An average of two families have been flying to Boston each week since then, and in October 2007, the trial became fully enrolled. The trial is expected to end in October 2009, with results published in 2010. 
 

Megan proudly wears her 1-year Trial Medal, which she received at the end of her May 2008 trip to Boston
 
 
 
 
“I know of no other rare genetic disease that has gone from gene discovery to clinical trial in under four years - a phenomenal testament to the hard work of The Progeria Research Foundation.” 
Francis Collins, MD, PhD, Director of the National Human Genome Research Institute that mapped the human genome, workshop speaker and co-discoverer of the Progeria gene.
 
 
 
 
 
 
 
 
 
 

Julieta, from Argentina.


Twenty-eight (28) children from sixteen countries are participating, ages 3 to 15 years. Children return to Children’s Hospital Boston every four months, for testing and to receive new drug supply, and stay in Boston for 4-8 days each visit.  While at home, their doctors keep a close watch over the children and submit periodic health reports to the Boston research team.  For the duration of the trial, an average of 2 children per week are traveling to Boston to participate. 
 

“The two Megans”, both 6 years old, in Boston for the clinical trial in May 2007.
Children Originate from the Following Countries:
 Argentina
Belgium
Canada
Denmark
England
India
Israel
Italy
Japan
Mexico
Pakistan
Poland
Portugal
Romania
USA
Venezuela

Michiel, 8 ½ , from Belgium with Hayley, 9 ½ , from England in June 2007 at Children’s Hospital Boston during their first visit.
 
 The Progeria Clinical Research Drug Trial:
Who, Where, When, How and How Much…
 
The clinical trial is led by Mark Kieran MD, PhD, Director, Pediatric Medical Neuro-Oncology, Dana-Farber Cancer Institute and Children’s Hospital Boston; Assistant Professor, Departments of Pediatrics and Hematology/Oncology, Harvard Medical School. Dr. Kieran is a pediatric oncologist with extensive experience with the drug under study (farnesyltransferase, or FTI) in children.
 
The clinical trial is a collaborative effort. The children are being seen by physicians at Children’s Hospital Boston, Dana-Farber Cancer Institute, and Brigham and Women’s Hospital, all Harvard University institutions. In addition, physicians and scientists from The Warren Alpert Medical School at Brown University, UCLA, and NIH are helping to make this trial a success. Many individuals are working together to perform this research.  
 
How did we get to this point? In 2003, The Progeria Research Foundation’s collaborative research team discovered the Progeria gene.  This discovery not only led to further understanding of Progeria, but scientists now know that studying Progeria can help us learn more about heart disease and the normal aging process that affects us all.
 
Since the gene discovery, the support of researchers, clinicians, families of children with Progeria and people like YOU have brought us to another crossroads in the search for a treatment. Researchers have identified a potential drug treatment for children with Progeria, called farnesyltransferase inhibitors (FTIs), and have conducted studies in the lab that support a human trial with the drug. Click here for more details on the research.
 
How will this drug work in Progeria?
The protein that we believe is responsible for Progeria is called progerin. In order to block normal cell function and cause Progeria, a molecule called a “farnesyl group” must be attached to the progerin protein. FTIs act by blocking (inhibiting) the attachment of the farnesyl group onto progerin. So if the FTI drug can block this farnesyl group attachment in children with Progeria, then progerin may be “paralyzed” and Progeria improved. Click here for more information on FTIs.
 
Progeria cells become normalized when FTIs are applied. Capell et al., PNAS, 2005
Normal cell                           Progeria cell            Progeria cell after being treated with FTI         
 
 
What will the trial cost PRF?  We estimate the trial will cost PRF $2 million dollars. This will pay for clinical testing, translators, travel, food, lodging, and certain medical costs during that 2 ½ year time period. 
 
 Support the Progeria Clinical Drug Trial
 
Your donations are helping to make this trial possible.
 
PRF needs to raise approximately $2 million dollars to fund this trial, and thanks to the generous people, foundations and businesses that have supported the trial thus far, we have now raised approximately $1.85 million – WOW!
 
As a small non-profit … WE NEED YOUR HELP! Please help us reach our $2 million goal by designating your donation to the Clinical Trial in your check memo or in the “in honor of” section of the on-line form.
 
This is the time to find a treatment for Progeria.             
 
Please Donate to the FTI Clinical Trial.
Together, we WILL find the cure!