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Hereditary diseases

Naturally-occurring peptide shows promise for CF treatment

Thymosin α1 is found to have strong potential to prevent the progression of cystic fibrosis.

Multiple treatments are available for cystic fibrosis (CF) but, while combinations of these improve life expectancy, no single drug has the ability to both control inflammation and rectify functional defects in CF. However, researchers may now have discovered a drug that can.

Thymosin α1 (Tα1) is a synthetic version of a naturally occurring peptide found in the thymus gland. The commercial version of Tα1 (Zadaxin) has been approved as an immunomodulator for 15 years in 37 countries and has an excellent safety record.

The study found that Tα1 corrected genetic and tissue defects in the lung and gastrointestinal tracts of mice with CF, and also in cells from individuals with the p.Phe508del CF mutation. Tα1 works by regulating inflammation and increasing the stability of mutant CF transmembrane conductance regulators.

Publishing their results in Nature Medicine[1], the researchers conclude that Tα1 has strong potential as a single-molecule-based therapeutic agent capable of preventing CF progression.

Citation: Clinical Pharmacist DOI: 10.1211/CP.2017.20202607

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Supplementary images

  • X-ray of lungs with cystic fibrosis

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