Allan Goldstein co-authored a paper on a potential new drug for the genetic disease affecting 70,000 people worldwide.
A George Washington University researcher helped discover a potential new drug to treat and stop the progression of cystic fibrosis, a genetic disease that leads to persistent lung infections and limits the ability to breathe.
The drug, Thymosin α1 (Tα1), is a targeted therapy that corrects genetic and tissue defect and significantly reduces inflammation seen in cystic fibrosis patients.
Allan Goldstein, School of Medicine and Health Sciences professor emeritus in residence of biochemistry and molecular medicine, co-authored a paper about the drug along with research partners at the University of Perugia and the University of Rome.
“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,” Dr. Goldstein said. “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.”
Cystic Fibrosis affects about 70,000 people worldwide and 30,000 people in the United States. Cystic Fibrosis stems from gene encoding mutations in the protein called the cystic fibrosis transmembrane conductance regulator (CFTR), which is important to maintaining salt and water balance in the lungs. This mutation results in a misfolded CFTR protein, and its premature degradation leads to persistent lung inflammation.
Tα1 is a synthetic version of naturally occurring peptide that corrects the multiple tissue defects in the CFTR seen in isolated cells from cystic fibrosis patients. Tα1 significantly reduces the inflammation seen in cystic fibrosis and increases CFTR maturation, stability and activity. Tα1 offers a strong potential to be a single-molecule therapeutic agent to treat and stop the progression of cystic fibrosis.
The paper was published Monday in Nature Medicine. It was co-authored by Dr. Goldstein and Luigina Romani at the University of Perugia and Enrico Garaci at the University of Rome in Italy.