A research team led by Marty Burke, associate dean of research at Carle Illinois College of Medicine, in collaboration with Mike Welsh and his team at the University of Iowa, has developed a new treatment that potentially could help all people with cystic fibrosis (CF), regardless of genetic mutation.
cystetic Medicines, a new company founded by Burke and Welsh recently secured $25 million in financing to develop the treatment into a transformative new therapy for people with CF.
CF is a progressive disease that causes persistent lung infections and, over time, limits the ability to breathe. People with CF have a missing or defective ion channel protein in their lungs needed to fight infections. There is no cure, but many treatments have been developed to reduce symptoms and health complications. The problem is about 10 percent of people with CF have genetic mutations that do respond to those treatments.
For 15 years, Burke’s research team has been working to find small molecule ion channels that can replace the missing protein, acting like a prosthesis on the molecular scale. Burke and Welsh and teams’ previously published research in the journal Nature found that the ion channel-forming drug amphotericin, approved to treat fungal infections, restored infection-fighting properties in cells collected from people with CF as well as in pigs with the disease.
New research, published this month in the Journal of Cystic Fibrosis, showed positive results in the first human trials looking at an established CF biomarker. “This research is exciting because it suggests that amphotericin may have potential to become the first gain-of-function treatment that could help everybody with CF, including those that cannot benefit from current treatments,” Burke says.
The investment of capital from Deerfield Management, a healthcare investment firm, and Illinois Ventures will allow cystetic Medicines to advance efforts to translate the molecular prosthetic into clinic settings. Chief Technology Officer Jeff Weers is leading research and development of a portable dry powder inhaler.
“Our goals in the next two years are to develop a dry powder formulation, manufacture it at large scale to support non-clinical safety and biodistribution studies, and then move to Phase 1 clinical studies, where we further examine the treatment,” Weers said.
Emily’s Entourage, a nonprofit focused on speeding life-saving research and drug development for people with CF mutations that can’t respond to current therapies, provided a grant to support part of the research. The group’s co-founder, Emily Kramer-Golinkoff, says she is eager to invest in a diversity of therapeutic approaches that show potential.
“We catalyze life-saving drug discovery and development by supporting the expeditious generation of early data, which can then get on the radar screen of larger funding organizations,” she said. “We have been thrilled to support Dr. Burke’s novel approach, and excited that his research proves our model.”
Burke, also a faculty member at the nascent Carle Illinois College of Medicine, challenges students at the engineering-based medical school to drive health innovation through research.
“At Carle Illinois we are committed to empowering our emerging physician-innovators to take their best ideas to improve health, develop those ideas in the lab, and then, hopefully, translate them into the clinic where they can improve patient care and outcomes,” he said. “We are excited to have this story developing at Carle Illinois, and we are sure there will be many more stories to come.”