Northeastern’s Michael Pol­lastri, an asso­ciate pro­fessor of chem­istry and chem­ical biology, and the Flatley Dis­covery Lab’s Bridget Cole are on the front lines of the search for a cystic fibrosis cure. Photo by Christo­pher Huang.

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.