Spotlight on Progeria

Children’s Health Update — Basic Research Leads to New Treatments

(NAPS)—Funding for basic medical research is paying dividends when it comes to battling many diseases. In one instance, it has helped to develop a treatment for a condition that ages children before their time…and may hold clues to aging-related heart disease.

Hutchinson-Gilford Progeria Syndrome (HGPS)—progeria, as it’s more commonly called—has been described as out of control, rapid aging in children. In the past, progeria was simply too rare to attract much scientific attention.

Approaches were limited to physical and occupational therapy, special nutrition and adult-based strategies to control related heart and circulatory disease.

Recently, however, a possible treatment for this “untreatable” disorder has emerged from basic cell biology, the Human Genome Project, and a new use for a “failed” cancer drug. The Human Genome Project was funded by the National Institutes of Health (NIH) and taxpayers’ dollars.      More >>

Translating Progeria: A Bench-to-Bedside Story

Sunday, December 14
12:15 pm–1:45 pm
Moscone Center, Room 304
Supported by The Ellison Medical Foundation

Years of basic curiosity-driven research into the structure and function of the nuclear lamins by a handful of cell biologists has illuminated our understanding of the mechanism(s) responsible for the premature aging disease, Hutchinson-Gilford Progeria Syndrome (progeria).

Unfolding with incredible speed, this remarkable story and its exciting denouement: the recent implementation of drug trials on progeria patients will be showcased on Sunday, December 14, 2008, at the ASCB Annual Meeting in San Francisco. The special translational session detailing the bench-to-bedside saga will feature an all-star lineup of expert panelists. Chaired by ASCB President, and pioneering nuclear lamin researcher, Bob Goldman, the panel will include:

  • Leslie Gordon, Founder and Medical Director, Progeria Research Foundation, and parent of a child with progeria
  • Francis Collins, former Director of the Human Genome Research Institute, NIH
  • Mark Kieran, Director, Pediatric Medical Neuro-Oncology and head of progeria trial, Children’s Hospital, Boston

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