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  • Home > Blog > Research News > Mesothelioma Research Could Benefit from the Penn Center for Orphan Disease Research and Therapy
Posted on Thursday, Jul 28, 2011

Mesothelioma Research Could Benefit from the Penn Center for Orphan Disease Research and Therapy

Patients affected by rare and orphan diseases, and their advocates fighting for more research and increased funding to bring effective treatments to the marketplace, just got a big boost from a $10 million anonymous gift to the University of Pennsylvania.   Penn has put the funds to good use by establishing the Penn Center for Orphan Disease Research and Therapy focused on discovering novel treatments for rare diseases.   There are close to 7,000 rare diseases, including mesothelioma, AIDS and meningitis, of which less than 200 of them currently have any effective treatments.

J. Larry Jameson, M.D., Ph.D., Executive Vice President for the Health System and Dean of the Perelman School of Medicine said, “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.”

Several of Penn’s staff have been leading the way in mesothelioma research, and they have been rewarded for their groundbreaking efforts.  Steven M. Albelda, M.D., vice chief, Pulmonary, Allergy, and Critical Care Division, director of Lung Research and director, Thoracic Oncology Laboratories, received the Wagner Medal for Excellence in Mesothelioma Research award in 2010.  In addition, Sunil Singhal, M.D., Thoracic Surgeon, Assistant Professor, who treats pleural diseases, including mesothelioma, was one of the recipients of the National Lung Cancer Partnership’s Young Investigator Research Grants.

Orphan diseases in the United States are those that affect fewer than 200,000 people.  Currently, an estimated 25 million people in the United States are living with an orphan disease.  Patients with rare diseases often have to travel across the country for treatment at a facility with a team of doctors and surgeons that specialize in their disease.  Specialists are aware of the latest research and they are able to develop a more informed treatment plan than would a physician who does not specialize in the disease.

In a press release announcing the Center, Penn Medicine explains the Center’s function as:

“Working together with other academic institutions, the National Institutes of Health, Pharma, and private philanthropy, the Center aims to reduce dramatically the technological and financial burden of investigators working in isolation by supporting a range of collaborative approaches:

  • Robotics for large-scale drug screening,
  • Biospecimen repositories to store samples,
  • Genotyping and bioinformatics services to identify causative genes,
  • Cell-based systems for developing new tests,
  • Nano-scale systems for developing new ways to deliver drugs, and
  • Small and large animal models for testing potential treatments.”

Although Penn did not specifically mention the International Rare Disease Research Consortium, focused research on rare diseases through this Center, could aid the consortium in meeting their goals of providing diagnostic tools for each of the rare diseases, and to develop 200 new treatments by 2020.