In the News

"A combination of cysteamine and epigallocatechin gallate seems to restore the CFTR function in a mouse model of Cystic Fibrosis"

Cystic fibrosis transmembrane conductance regulator (CFTR) repairing strategies are an emerging option in Cystic Fibrosis (CF) therapy as it addresses the primary cause of CF, namely the loss-of-function of CFTR. The authors of a study published in Autophagy investigated whether the oral administration of cysteamine and epigallocatechin gallate (EGCG), a green tea flavonoid – readily available compounds that the authors had anticipated would rescue CFTR function - in a mouse model of CF which has an F508del mutation in CFTR, would improve CF symptoms in these mice.

The authors demonstrated highly promising results in the mouse model and are testing the clinical efficacy of these compounds in CF patients homozygous for the F508del mutation. This endeavor has been an entirely academia driven exercise and may summon a new era of orphan drug discovery. OrphaNews spoke to the researchers who are involved in crafting this treatment approach 

Member of the research team - Dr. Valeria Raia

"Eureka moment offers hope of cystic fibrosis cure" -

Video in article (below):

"New Cystic Fibrosis drug treatment may be 'potential game-changer'"

"Scots researchers in cystic fibrosis 'game-changer' study" 

"Novel Cystic Fibrosis Treatment Shown To Be Effective as Mucoactive Antimicrobial, Antibiofilm Agent"

Cystic Fibrosis game-changer

New strategy to treat inherited disease 

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