Bold and Inspired: Penn to Create Center for Orphan Disease Research and Therapy
August 4, 2011
Back in May, President Amy Gutmann declared that Raymond and Ruth Perelman's history-making gift to endow the Perelman School of Medicine (the largest gift in Penn's history) was "transformational" and "inspiring." She was absolutely right.
Now, thanks to a $10 million gift from an anonymous donor, Penn will launch the first-of-its-kind interdisciplinary center focused on developing treatments, and even cures, for orphan (rare, or neglected) diseases: the new Penn Center for Orphan Disease Research and Therapy.
Why is this so inspired?
Right now, treatments and research for orphan diseases -- those affecting fewer than 1 in 1,500 people -- lag behind therapies for major diseases, such as diabetes and cardiovascular disease. In part this disparity is due to a lack of incentive; established research and drug development enterprises do not see a large enough market to warrant the investment. Yet 7,000 orphan diseases -- usually genetic, and hence chronic -- affect 250 million people (mostly children) worldwide today, even as basic research is revealing new insights into their genetic and biochemical underpinnings and clinical research is demonstrating that they can be treatable with novel therapies.
In the face of such limited progress in finding cures despite promising knowledge, the new Center for Orphan Disease is a boldly decisive -- and brilliantly inspired -- move. It will not only help close the critical gap between knowledge and treatment but provide the essential collaborative relationships that may someday lead an international, coordinated effort in the eradication of orphan diseases.
"There is simply nothing else like it," says Glen N. Gaulton, Ph.D., Executive Vice Dean and Chief Scientific Officer at the Perelman School of Medicine.
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By investing in the necessary research that large pharmaceutical companies do not, the center will spur progress in finding cures, beginning by helping to increase awareness of these diseases, their causes and potential treatment. Already scientists are discovering that clusters of disorders may share common causative mechanisms, and the center will connect researchers and clinicians at Penn and other institutions working in isolation on orphan diseases so that they can collaborate productively. The center will also bring together academic institutions and organizations that fund biomedical research for orphan diseases, and connect investigators to pharmaceutical firms for developing research and therapies.
The new center also builds on Penn's exceptional track record for bringing all its forces to bear on rare diseaes. It was Penn's Center for Research in FOP and Related Diseases, the only center in the world dedicated to fibrodysplasia ossificans progressiva (FOP), which made the all-important discovery of the gene that causes FOP. Penn has also had success in identifying how gene therapies and drug treatments for one disease may prove beneficial in treating other diseases, and Penn researchers are using gene therapy to treat a rare form of inherited blindness, allowing children and young adults who were blind at birth to see.
The timing for the new Center for Orphan Disease Research and Therapy couldn't be better. Now in the sixth year of its Making History Campaign, the University has a newly endowed Perelman School of Medicine and a newly built "geography of medicine" -- physically and purposefully connected medical facilities close to other disciplinary schools and researchers on campus -- all of which makes Penn perfectly poised to marshall the very best of its integrated expertise, resources, and experience and to lead in translating innovative research into the clinic.
"This is a wonderful example of philanthropy in action," says J. Larry Jameson, M.D., Ph.D., Executive Vice President for the Health System and Dean of the Perelman School of Medicine. "The Center will be a natural extension of Penn's expertise in orphan diseases, and this strategic investment will galvanize support for orphan disease research around the globe. I am proud that Penn Medicine is taking a clear leadership position in transforming the health of millions."
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