Emerging drug treatments for cystic fibrosis

Abstract

Although the gene for cystic fibrosis was discovered in 1989, a definitive cure remains elusive for this deadly orphan disease. Advances in nutritional rehabilitation, antibiotics, mucolytics and delivery of care have improved survival to a median age of 37.5 years; however, the psychosocial, personal and financial burdens of this lifelong chronic illness remain considerable. The current portfolio of investigational therapeutics is explored here and placed in a context of therapeutic target and predicted clinical benefit. Partnership between large and small pharma, the Cystic Fibrosis Foundation and academia should be fostered to accelerate therapeutic development.

Keywords::

• absorption
• cAMP
• chloride
• ClC-2
• calcium-activated chloride channel
• cystic fibrosis transmembrane conductance regulator
• endoplasmic reticulum
• epithelia
• gene therapy
• inositol polyphosphate
• ion channel
• mannitol
• mucolytic
• plasma membrane
• premature termination codon
• secretion
• sodium