A Breath of Fresh Air
Can repurposed drugs improve treatment options for patients with cystic fibrosis?
Maryam Mahdi |
This article is part of our special focus on "traditional" pharma: The Small Molecule Manufacturer (read more here). You can find more articles from The Small Manufacturer here.
The Cystic Fibrosis Foundation has awarded a $692,000, two-year contract to Calibr, the research and development arm of Scripps Research. Calibr’s ReFRAME – a library of over 13,000 small-molecule drugs with proven track records of safety in humans (more) – will be used to identify existing medicines suitable for treating infections caused by Burkholderia cepacia complex, which comprises 22 bacterial species.
According to Arnab Chatterjee, Vice President of Medicinal Chemistry at Calibr, CF remains one of the hardest and most complex diseases to treat – partly due to the number of organs and tissues affected by the lack of a functional cystic fibrosis transmembrane conductance regulator protein.
The Burkholderia cepacia complex poses a serious health risk to patients with cystic fibrosis because the bacteria can cause cepacia syndrome, a life-threatening systemic infection. It also often causes patients to be rejected for lung transplantation
The team will cultivate bacteria to imitate the infection environment triggered by B. Cepacia and use the ReFRAME library to select molecules with antimicrobial activity.
“Many of the bacterial species that are problematic in CF are resistant to currently available antibiotics. Our approach could also be applied to other bacteria relevant to cystic fibrosis, such as Pseudomonas aeruginosa, bacteria which cause chronic inflammation and lung infection in cystic fibrosis patients,” Chatterjee explained.
The Calibr researchers are also working with John LiPuma, a Professor at the University of Michigan, who works closely with CF patients. LiPuma, whose laboratory maintains an extensive culture collection that includes approximately 35,000 strains of respiratory tract bacterial pathogens, will provide patient samples and help the Calibr team accelerate the translation of anti-infective candidates.
The Cystic Fibrosis Foundation has committed to invest $100,000 million to CF research through it’s new “Infection Research Initiative” between 2019 and 2033.