Students Aid Search for Lymphomatic Malformation Cure
Three KGI premed students turned a classroom assignment into a successful effort that could eventually help save the lives of hundreds of small children suffering from lymphomatic malformation.
Daniel Burke, Laura Wilson and Rudy Tsay wrote an Orphan Drug Designation Application on behalf of researchers at the University of Iowa hospitals and clinics for OK-432, a substance that has proven to be effective in treating lymphomatic malformation, a rare and fatal disease that causes large growths on the heads and necks of young children who eventually suffocate from constricted airways.
The students wrote the application for the course "Writing an Orphan Drug Designation," taught by Ian Phillips, PhD, KGI's Norris Professor of Applied Life Sciences.
Phillips is director of KGI's Center for Rare Disease Therapies, which is dedicated to educating and advocating for therapies for rare diseases, which afflict nearly 30 million Americans. Of the 7,000 identified rare diseases, many have no treatments. For those that can be treated, the cost to the patient is often prohibitive.
"This fits exactly with the mission of the Center for Rare Disease Therapies," said Phillips, adding that he was proud of the students' accomplishment.
To receive orphan designation, the proposed drug therapy must target a rare disease (one with a patient population of 200,000 or less) and it must show promise for treating the disorder. OK-432 is manufactured in Japan and was originally developed to treat certain forms of cancer, with moderate success.
While U.S. Food and Drug Administration (FDA) approval of the application does not guarantee the drug itself will be approved, it allows researchers to seek grant funding to conduct further clinical trials and provides incentives to the existing manufacturer to increase production of the drug or for other companies to develop the treatment.
Burke, whose mother is a research nurse at the University of Iowa, took the lead on the project. She coordinates clinical trials and studies on OK-432 for Dr. Richard Smith, who has been testing the treatment under an FDA Investigational New Drug application for more than a decade.
OK-432 consists of a series of four injections with a 96% success rate in treating lymphomatic malformations, which is very rare with only about 100 new cases a year.
"This opens up a lot of opportunities for that drug that has been stuck in clinical trials for the past 10 years," Burke said.
Tsay said his group benefited from an orphan drug designation application workshop conducted by the FDA at KGI last November. They used a checklist provided by the FDA as a template for their application. Some of the rest of it was luck.
"The situation just fit perfectly for us," Tsay said. "We had a good sponsor drug for a very small subset of the population."
Wilson was surprised at the number of drugs that might benefit from orphan drug designation if more people were aware of and understood the process, how easy it is to write an application and how satisfying success feels.
"It's a very disfiguring disease that this drug helps to treat," Wilson said. "What more can you ask for in terms of having a real world impact."
Wilson and Tsay have landed internships with the FDA this summer and will be working with the FDA's Office of Orphan Products Development.
All three students came to KGI for the Postbaccalaureate Premedical Certificate (PPC), a one-year program designed to give premed students the competitive edge in getting into the medical school of their choice. And all three have decided to take advantage of the PPC program's option of staying an additional year to earn a Master of Bioscience (MBS) degree while applying to medical school.
Burke, Tsay and Wilson are not the first KGI students to be involved with successful orphan drug designation applications. Silviya Meletath (MBS '11) helped win FDA approval last year for BPT Pharmaceuticals in Irvine for a drug called ProEnzy that shows promise in treating pediatric Multiple Sclerosis.
By Elaine Regus