Northeastern University and the Flatley Discovery Lab, a Boston-based independent, nonprofit organization, are collaborating to develop a cure for cystic fibrosis, a devastating genetic disease often overlooked by major pharmaceutical companies.
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis conductance regulator (CFTR) gene that cause thick, sticky mucus to build up in the lungs, digestive tract and other areas of the body. Until recently, most CF drugs focused on treating the symptoms of the disease and not the underlying cause of nonfunctioning CFTR protein.
Northeastern and Flatley researchers believe small-molecule chemicals that bind directly to the CFTR to restore proper function will be significant and critical drug discovery tools to better understand the underlying cause of CF and develop new medicines. Few for-profit companies are willing to invest in developing a drug for cystic fibrosis, as CF’s relatively small patient base would likely not generate enough profit to cover development costs.
“The nonprofit and academic environment is well-positioned to contribute to finding cures for these diseases, particularly when powerful partnerships are made with the industrial sector,” said Michael Pollastri, an associate professor of chemistry and chemical biology who specializes in drug discovery for rare and understudied diseases.
The Flatley Discovery Lab, founded in 2009 by Boston entrepreneur John Flatley, is a small but highly-skilled research organization with extensive experience in drug discovery and development.
During the last year, the Flatley Discovery Group has screened more than 300,000 drug compounds, looking for existing formulas that may have some effect on the mutation that causes cystic fibrosis. This year, the group expects to screen some 500,000 more compounds, running each drug through a high-throughput screening platform.
Northeastern researchers will then bring their expertise to the collaboration by modifying the small number of promising compounds that appear to have some impact on correcting the CFTR mutation, and advancing the most viable options through the drug development process. With those compounds as a base, researchers hope to then develop a new compound that could begin to move through the Food and Drug Administration’s trial process.
“We are excited to have Northeastern work with our team to develop chemistry capabilities that could lead us further toward a drug candidate,” Flatley said. “Having a personal stake in the cure for CF and available funds from the Flatley Foundation has allowed us to take risks counter to the current capital market’s risk aversion.”
Research in a new dedicated lab in Northeastern’s Hurtig Hall began late last year. Two recently graduated Ph.D. chemists will work with Pollastri and the Flatley team. The partnership could lead to exciting experiential learning opportunities, particularly through co-op for undergraduates in the College of Science.
The team hopes to develop a promising compound during the next year. Ultimately, a successful treatment must cure cystic fibrosis in a safe and effective manner — a major challenge since many CF patients who would require the drug currently use many different medications to treat their condition.
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