KGI Student Team Receives Orphan-Drug Designation for Behçet's Treatment
Team member Stacy Crawford, MS’13, has a personal stake in seeking more effective treatment options for rare disorder
How many people diagnosed with a rare disease have the opportunity to help develop a new drug to treat it — especially at the age of 28? It’s probably safe to assume not many, and that Stacy Crawford, MS ’13, might just be the only one. Crawford was part of a student-team that successfully wrote an orphan drug-designation application for the use of a drug called pentoxifylline for the treatment of Behçet's disease, which she was diagnosed with 12 years ago.
Behçet’s is an autoimmune disease that results from damage to blood vessels throughout the body, particularly veins. The exact cause of Behçet’s is unknown but it tends to develop in people in their 20s and 30s. Although relatively rare in the United States, where the incidence is reported to be around 1 in 20,000, it is much more common in the old “Silk Road” trading route countries of the Mediterranean basin, Middle East and Asia. There, the incidence is around 1 in 10,000 people.
The disease is often difficult to diagnose partly because many doctors aren’t familiar with it and partly because its symptoms, which can include painful mouth ulcers, muscle and joint paint, headaches and extreme fatigue, can be mistaken for other autoimmune diseases such as lupus or rheumatoid arthritis. But, even when properly diagnosed, there is currently no cure for Behçet’s, and treatment options are not always that effective. As a result, Behçet’s patients often lead severely disrupted lives that include frequent hospitalizations.
“My aunt also has Behçet’s, and, although I wouldn’t say that it has severely disrupted her life, it has definitely impacted it in a negative way. She has seen many different doctors and has tried many different drugs for her Behçet’s symptoms. They often work for a while but then they always stop,” said Crawford, who is spending the summer interning at Althea Technologies, a San Diego-based biotech company.
Crawford was able to interest fellow classmates Kate Brown, Kathleen Coquia, Nicholas Generales, Robert Krattliin and Sarah Sotelo in writing an application for the drug pentoxifylline for the “treatment of oral and genital ulcers associated with Behçet’s disease.” The drug isn’t standard treatment for Behçet’s, which is usually treated with cortical steroids, but the group’s research found that it often proved effective in clearing up symptoms where other drug regimens had failed. In fact, some patients even stayed in remission after they stopped taking the drug.
“We looked at all the research out there, including the possible interactions. It was very challenging but breaking it down and assigning everyone different sections definitely helped,” said Crawford, adding that the American Behçet’s Disease Association (ABDA) helped sponsor the team’s application.
An orphan drug is one that has been developed specifically to treat a rare medical condition (also referred to as an orphan disease) such as Behçet’s. Getting an FDA designation qualifies the sponsor of the product for the tax credit and other incentives provided in the 1983 Orphan Drug Act (ODA) and is considered the vital first step in allowing companies to raise money to study and clinically test the drug.
“It’s highly unusual for students to be able to write a drug application themselves, let alone one that is perfectly written. It really didn’t need any editing at all,” said Dr. Tim Cote, a former director of FDA’s Office of Orphan Products Development, who teaches KGI’s class on writing an orphan drug application, along with Dr. Ian Phillips, the head of KGI’s Center for Rare Disease Therapies.
The next step, Phillips adds, will be looking into finding different ways to market and distribute pentoxifylline for the treatment of Behçet’s.
“Helping raise awareness of rare diseases and promoting promising treatments like this one is exactly why the center was created. We also act as catalyst for entities such as the FDA and pharmaceutical companies to come together,” Phillips said, adding that center operates on a completely non-profit basis and any funds raised through this or any other orphan-drug designation will go toward scholarships.
As for Crawford, she says she is thrilled to be able to do something that might benefit other people living with Behçet’s. “I feel fine today, but I know my symptoms might get worse one day, and, if they do, it would be great to have more treatment options,” she said.