Cystic Fibrosis - Renewed Hopes Through Research

Citations Over TimeTop 10% of 2012 papers

Abstract

To our knowledge no other membrane/peptide system offers such flexibility for design and analysis.Many TM sequences have been and are being studied by others (Marsh, 1996).However, for the most part, incorporation into bilayers leads to a mixed orientation with helical dipoles present in both directions.Such mixing of dipole orientations is prevented in our oligo-lysine adducted M2GlyR peptide system.This review will describe our understanding of how these peptides form channels with a range of anion selectivity and conductance properties, through a combination of electrophysiology, biophysical structural studies and computer simulations. Current CF therapiesWhile a number of therapies for CF are currently in development, many of the treatment options presently available to patients are palliative in nature, addressing one or more symptoms of the condition and acting to reduce the severity of these effects and their associated risks.Diet/nutritional supplements Proper diet and nutrition may reduce the impact and risk of CF-related conditions such as diabetes and osteoporosis.The lungs of CF patients appear to have fewer natural antioxidants than those without the condition, which may contribute to repeated infection and persistent inflammation.Patients may benefit from an increase in their intake of antioxidants through diet or supplements to fight this inflammation.Further promise may lie in drugs that work to build antioxidants in the lungs.AquADEKs ® by Yasoo Health, Inc. is a vitamin supplement specifically formulated to meet the antioxidant needs of those with CF, and is commercially available.It has been shown to improve lung function, normalize vitamin levels in plasma and reduce neutrophilic inflammation (Sagel et al., 2011).Enzyme replacement therapies Pancreatic enzyme products work to increase the digestion and absorption of fats, proteins, and starches while promoting the absorption of certain vitamins in those with CF, who often suffer from malnutrition due to enzyme deficiencies.The U.S. Food and Drug Agency (FDA) has approved a number of these products, including Zennen® (Eurand Pharmaceuticals), Creon ® (Abbott Laboratories) and Pancreaze™ (Ortho-McNeil Pharmaceutical), while others, such as Pancrecarb® (DCI) and Ultrase ® (Axcan Scandipharm) are still awaiting clinical trials.Liprotamase ® (Alnara Pharmaceuticals) is a non-porcine pancreolipase enzyme therapy, which has completed a phase 3 clinical trial.However, the new drug application submitted by Alnara was rejected by the FDA on the grounds of insufficient data demonstrating the efficacy of the drug (Lowry, 2011).Antibiotics/anti-infectives Due to the increased risk of disease caused by the excess and/or viscous mucus that is characteristic of CF, patients are often treated with antibiotics to circumvent chronic infections, such as that of Pseudomonas aeruginosa.Antibiotics can be administered orally, intravenously, via inhalation through devices such as metered dose inhalers (MDIs), or through implanted devices such as a port or Peripherally Inserted Central Catheter (PICC) (Gibson et al. 2003).TOBI® (tobramycin solution for inhalation) and recently developed Cayston® (aztreonam solution for inhalation) are two approved and commonly prescribed antibiotics for patients with CF, and both are effective against P. aeruginosa.TOBI ® is approved to treat P. aeruginosa infections by concentrating the delivery of the antibiotic to the airways.Developed by Novartis Pharmaceuticals and widely

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