A New Treatment For Cystic Fibrosis?

Credit: yourgenome/Flickr

Credit: yourgenome/Flickr

Treatment with the molecule Thymosin α1 may decrease inflammation and correct genetic defects causing cystic fibrosis

Cystic fibrosis (CF) is a chronic condition which afflicts around 70,000 people around the world. Current treatments can manage the disease to a large degree but lifespan remains curtailed at roughly 40 years of age - commonly due to complications such as infection and antibiotic resistance. 

"Right now there are multiple treatments for cystic fibrosis, and while these have improved life expectancy dramatically, there is still only a lifespan of about 40 years for patients. No one treatment can stand alone. We developed a single treatment that can potentially correct the genetic defect that causes cystic fibrosis and decrease the inflammation that happens as a result"

Rescuing CFTR by Thymosin α1(T α1) acting as a corrector of the misfolded protein (p.Phe508del-CFTR) and potentiator of normal chloride channel activity. EC, epithelial cells. CFTR, cystic fibrosis transmembrane regulator protein. WT, wild-type (normal epithelial cells). Credit: University of Perugia

Rescuing CFTR by Thymosin α1(T α1) acting as a corrector of the misfolded protein (p.Phe508del-CFTR) and potentiator of normal chloride channel activity. EC, epithelial cells. CFTR, cystic fibrosis transmembrane regulator protein. WT, wild-type (normal epithelial cells). Credit: University of Perugia

A mixed team of researchers have now reported that a synthetic version of a peptide isolated from the thymus, Thymosin α1 (Tα1),  can in fact correct many defects in both mice and human individuals with CF. CF is caused by various types of mutations affecting an ion channel in a number of regions of the body; leading to impaired water influx or efflux, which in turn disrupts the body's crucial barriers to its external environment and prevents proper mucus clearance. Tα1 appears able to stabilise the faulty CFTR channel protein which is the target of CF mutations - improving protein maturation and function. In their experiments it was also able to reduce inflammation, which is a critical step in CF pathology. 

Thymosin α1 has in fact already been approved for clinical use for 15 years, for its immune therapeutic activity in viral infections and immunodeficiency diseases. It has consistently been shown to have a favourable safety profile, and while it's not available in the US market it could potentially be approved for use relatively quickly if it successfully passes further testing. 

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