Friday and Saturday (Dec. 6 and 7), 28 researchers, parents and representatives of advocacy groups from across the United States and Europe convene at the Medical Center to determine how a Phase III clinical trial might work for children with Juvenile Batten Disease, should a potential treatment emerge from current or future research.
With incredibly rare diseases, the road map for moving research from the lab to patients is often unclear. Such is the case with Juvenile Batten Disease, a rare neurodegenerative syndrome that erupts with little warning. It first steals sight between 5 and 8 years of age, then cripples cognitive and motor capacities, and is ultimately terminal in the late teens or 20s. Only 1-2 of every 100,000 children are born with the genetic disease, making research into treatments difficult.
“We need to determine how to reach out to and enroll patients, since the population is so small and spread around the world,” said Heather Adams, Ph.D., assistant professor of Pediatrics and Neurology and the conference organizer. “And we need to set the standards for measuring whether a treatment is effective.”
The conference attendees will also assess what registries exist that contain information about patients with Juvenile Batten Disease and how information might be applied to help make a Phase III trial possible. In addition, a handful of trainees will attend the conference, encouraging young investigators to pursue research on Batten Disease with the aim of ensuring that research continues into the next generation.
Read more about the conference here.