Top leaders from industry, government, science and patient advocacy groups shared the challenges and opportunities of developing drug therapies to treat rare disease at a recent mini-symposium hosted by KGI's Center for Rare Disease Therapies.

The symposium ("New Strategies for Rare Disease Therapies") was paired with the second annual meeting of the center's advisory board.

"This year's meeting was a great success because we had the right mix of high level participants - the director of the Office of Orphan Product Development at the Food and Drug Administration, the VP of the National Organization of Rare Disorders, the VP of Corporate Strategy at Celgene, and leaders of Amylin, Sigma Tau and Amgen combined with academic clinician scientists from UC San Diego. For KGI this was a national meeting, open to our students to learn from experts," said Ian Phillips, the Center's director and Norris Professor of Applied Life Sciences.

The Center, which was launched last year as one of the initiatives led by President Sheldon Shuster to create research centers at KGI, is devoted to finding drug therapies for rare diseases that affect nearly 25 million Americans. No treatment is available for most of the 7,000 identified rare diseases, and for patients who can be treated the cost is often prohibitive.

Rare diseases range from the more familiar like cystic fibrosis and muscular dystrophy to the largely unknown like inherited metabolic inclusion disease, a condition that can destroy muscle cells.

Phillips mentioned several symposium highlights including:

Chris Garabedian, vice president of corporate strategies for Celgene, talked about the limitations companies face in developing new drugs to treat rare diseases. Those limitations include funds, regulations and intellectual property issues;
Dr. Richard Haas, director of the Mitochondrial Disease Laboratory at UC San Diego, gave a detailed presentation on various types of mitochondrial diseases, obstacles to treatment and possible new therapies;
Three KGI students - Brent Thompson, Aditya Kelkar and Sonali Talele - spoke about their summer internships with the Pharmaceutical Research and Manufacturers of America, and the FDA's Office of Orphan Products Development;
The Center will host a conference at KGI early next year for biotech and pharmaceutical company employees to learn how to write applications under the Orphan Drug Act. The FDA will send a team to run the workshop, and be assisted by trained KGI students.

"The application process is really fairly simple, but the FDA has a reputation for making things complicated so most people are afraid of it," Phillips said. "The conference will help demystify the application."

The non-profit Center's mission is to bring national attention to the impact and severity of rare diseases through advocacy, educating students and working with scientists whose research may be instrumental in finding new treatments for rare diseases.

Following the symposium, the Center's advisory board discussed the possibility of establishing an online journal with KGI students to review articles and provide commentaries on rare diseases and the creation of an electronic, updated, searchable database on rare diseases.

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