Class Project Results in FDA Orphan Drug Designation

Rasmussen’s encephalitis is a devastating disease, causing young children to suffer as many as 100 seizures per day, loss of brain function, and early death. As with many rare diseases, there is no cure or treatment.

Thanks to work undertaken by a Keck Graduate Institute (KGI) student team, however, there may be an eventual therapy to help children with Rasmussen’s encephalitis. Through a project in their Fall 2015 “Writing an Orphan Drug Application” class, Nina Butingan, Andrew Browne, and Daniel Park identified Rituxumab as a potential treatment for this rare disease. The U.S. Food and Drug Administration (FDA) also saw promise in their idea and recently issued an orphan drug designation, the first step in the process of approving a new therapy for a rare disease.

Norris Professor of Applied Life Sciences Ian Phillips teaches the course that exposes students to FDA regulations, rare diseases and orphan drugs, and encourages their active participation in learning. Phillips is also the director of KGI’s Center for Rare Disease Therapies. 

“Because it’s an anti-inflammatory, Rituxumab was being used for other treatments, such as for rheumatoid arthritis, but not for brain issues,” Phillips says. “I thought they had a unique use of a drug that’s widely used in humans.”

The students researched and compiled the necessary data and clinical reports to make a compelling case for the drug’s use in treating Rasmussen’s encephalitis. They also spoke with a physician who had led an encouraging clinical trial cited in their work.

But Phillips notes that the success of their application was far from assured. Not all are approved. Submissions typically come from companies, not students. The FDA guidelines are rigorous, with applicants required to present the science behind their idea, show that the proposed drug is safe for use with humans and provide evidence that the condition qualifies as a rare disease.

“We realized we had a good paper, but we didn’t realize how good,” says Butingan, who led the student team and had first suggested that they focus on Rasmussen’s encephalitis. “This was a combination of hard work and luck. We were lucky to find such a promising agent. We knew that a cure would be difficult, so we were looking for a drug that would improve the quality of life for patients by lessening symptoms.” 

With an orphan drug designation in place, it’s now possible for clinical trials to follow. Though Butingan, Browne and Park have graduated—each completed KGI’s Master of Bioscience program in 2016—they remain eager to see additional progress in bringing a treatment for Rasmussen’s encephalitis to market.

“We want to continue with this drug and are brainstorming about what we can do to further this,” says Butingan, who is currently employed as a science writer for the Children’s Oncology Group, a nonprofit clinical research organization. “I’m optimistic. We saw good results and have to see how far we can take this. Even if we hit a roadblock, we can still raise awareness and advocate for people suffering from this disease.”