Research into rare premature aging disorder praised as a model of “what’s right” in biomedical research


Recently, a group of scientists, clinicians and patients gathered in a suburb of Washington, DC to discuss scientific progress in the study of a rare premature aging disorder. This disease, Hutchinson-Gilford Progeria Syndrome’€”often referred to as HGPS or simply progeria’€”is one you may not have heard of, yet. But the Progeria Research Foundation and families of progeria patients have been working hard to increase awareness and raise funds for research into this rare disease that results in death at an average age of only 13. Among the list of speakers at the conference was the Director of the National Institutes of Health, Francis Collins, MD, PhD and researchers from a number of Research!America member organizations.

The topic of diminished research funding was on everyone’€™s mind, even at this internationally attended meeting. Collins spoke to the researchers from North America, Europe and China about this ’€œdifficult moment’€ in biomedical research. He encouraged conference attendees to communicate the value of medical research whenever possible, adding that he is ’€œguardedly optimistic’€ that medical research will turn a corner. In his role as Director of the NIH, Collins told the audience that he is frequently called upon to provide testimony regarding the value of basic and translational research. Not surprisingly, Collins uses progeria research as a model of ’€œwhat’€™s right in medical research.’€ In the 10 years since the genetic mutation responsible for this rare disease was discovered, the field of progeria research has made tremendous advances.

According to Collins, the incredibly fast rate of progress can be attributed to a strong foundation of basic scientific knowledge, the scientific connection between premature and normal aging and cooperation between the families and research community to achieve what is best for the children living with this disease. Collins also pointed to a rapid progression to clinical trials for progeria patients as a model of successful medical research; three different drug trials are either underway or have been completed internationally within a decade of identifying the mutant gene. The NIH director was quick to point out that progression to these clinical trials would not have been possible without the decades of basic scientific research completed well before discovery of the gene responsible for progeria.

Scientists from research institutions like the Buck Institute, Cornell University, and Harvard University presented their work alongside clinical specialists from centers like Brigham & Women’€™s Hospital and Dana-Farber Cancer Institute. This meeting was the seventh scientific workshop organized by the Progeria Research Foundation and it focused on the importance of basic and clinical scientists working together to find a cure for this fatal disorder.  This approach can be sustained with robust federal funding for research.

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If concerted, long-term investments in research are not made, America will lose an entire generation of young scientists.
Brenda Canine, PhD; McLaughlin Research Institute, Montana