Leveraging Partnerships and Personalized Medicine to Find a Cure

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The sixth scientific workshop of the Progeria Research Foundation (PRF) earlier this year highlighted many ways that an increased understanding of the genetic underpinnings of Progeria is informing promising new approaches for treating the disease.  A record 140 attendees congregated from 10 different countries to hear expert oral presentations and view poster presentations. Doctors and scientists – who often work in separate worlds, either in the clinic or in the lab – inspired one another as they came together to share new discoveries and directions for future research.  Speakers included leading scientists in the fields of heart disease, aging, genetics and lamins.

Researchers at the workshop had a unique opportunity to meet some of the children who are benefiting from their work.  Children and their parents talked about what it’s like to live with Progeria, and answered questions from the attendees who wanted to better understand how they can continue to conduct beneficial research.  Leslie Gordon, PRF’s medical director, shared Progeria’s journey from obscurity, through gene discovery, on to treatment trials, and presented a vision of where the field is headed in the push towards new treatments and a cure. 

We heard from clinical trial investigators, who shared updates on the progress of their current studies.  We heard from world renowned scientists such as George Martin, Elizabeth Nabel, and Judy Campisi, who addressed the links between normal aging, cardiovascular disease, inflammation and Progeria.  And we got a glimpse into the future from several scientists who are working on cutting edge strategies for future drug development and gene therapy for Progeria.  

Dr. Tom Misteli (NCI) presented a drug development assay which uses genetic strategies to measure chemicals that decrease or eliminate the production of the disease-causing protein, progerin.  His promising studies point us in the direction of new treatments or even a cure for Progeria.

 Dr. Fyodor Urnov looked to the possibility of a genetic cure for Progeria using “genome editing” with zinc finger nucleases.  This method of repairing genetic mutations within cells of the human body is now in the first stages of clinical trials for other diseases, and may be applied to Progeria in the near future.

PRF is proud that Progeria research has made such tremendous progress, moving from bench to bedside in only 10 years.  This would not be possible without the collaborations and innovative partnerships that have been established over the last decade.  From our collaboration and partnerships with NIH (NHGRI, NCI and NHLBI), to pharmaceutical companies like Merck Schering-Plough to first-rate hospitals like Children’s Hospital Boston,  we are moving at lightning speed in the direction of finding a cure.

Audrey Gordon, Esq. is the President and Executive Director of the Progeria Research Foundation

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