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In just 5 years, gene discovery to clinical trial of potential treatment
Date:12/14/2008

are no treatments for the disease.

Speaking at the symposium will be:

o Robert D. Goldman, Ph.D., a pioneer in basic research relevant to understanding how the HGPS gene mutation disrupts human body cells. The head of cell biology at Northwestern University Medical School in Chicago (and ASCB's current president), Dr. Goldman has long studied the normal structure and function of a major component of the scaffold-like network of proteins just inside the membrane that surrounds and protects the cell's nucleus. His research team identified the component, called nuclear lamins, as the culprit in HGPS;

o Gene-hunter Francis Collins, M.D., Ph.D., the former National Human Genome Research Institute (NHGRI) director who headed the research team that pinpointed HGPS' genetic mutation in 2003. Just two years later, NHGRI scientists identified the class of experimental cancer drugs, called farnesyl transferase inhibitors (FTIs), that can prevent the cell damage caused by the gene mutation and thus might provide an effective therapy against the disease;

o Harvard Assistant Professor Mark Kieran, M.D., Ph.D., the principal investigator of the Boston Children's Hospital phase 2 clinical trial that is evaluating a FTI drug in children with HGPS. He is director of Dana-Farber Cancer Institute's Pediatric Medical Neuro-Oncology;

o Leslie Gordon, M.D., Ph.D., parent of a child with the disease and medical director of the Progeria Research Foundation, which is funding the clinical trial and which has been the "mover and shaker" in accelerating research on the disease.

The symposium will be held at 12:15 𔂿:45 pm, Sunday, Dec. 14, at San Francisco's Moscone Center.

The 28 children enrolled in the clinical trial range in age from 3 to 15 years old and come from 16 countries. The two and one-half year phase 2 clinical trial, which began in 2007, is evaluating the FTI drug lonafarnib. In clinical studies in people
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Contact: Cathy Yarbrough
sciencematter@yahoo.com
858-243-1814
American Society for Cell Biology
Source:Eurekalert

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