Cell Death and Differentiation: A Novel Treatment of Cystic Fibrosis Acting On-Target

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Cystic Fibrosis in Focus

ReachMD

4 episodes

5 months ago

Cystic fibrosis (CF) is one of the most prevalent global congenital disorders, affecting some 70,000 individuals worldwide and 30,000 children and adults in the United States. While clinical efforts are expanding worldwide to improve diagnostic testing and treatment approaches for this progressive lung disease, targeted therapies for CF against any specific mutation remain elusive. The Cystic Fibrosis in Focus series gathers the latest information from leading medical experts devoted to advancing knowledge in pursuit of treating patients with CF. With education spanning topics such as pinpointing CF's underlying genetic basis, identifying specific mutations, improving clinical detection methods, and providing more targeted therapies, The Cystic Fibrosis in Focus series delivers the critical tools needed to improve care for CF patients. In Partnership with

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Cell Death and Differentiation: A Novel Treatment of Cystic Fibrosis Acting On-Target

Cystic Fibrosis in Focus

9 years ago

Cell Death and Differentiation: A Novel Treatment of Cystic Fibrosis Acting On-Target

A novel treatment of cystic fibrosis acting on-target: cysteamine plus epigallocatechin gallate for the autophagy-dependent rescue of class II-mutated CFTR.

Tosco A, De Gregorio F, Esposito S, De Stefano D, Sana I, Ferrari E, Sepe A, Salvadori L, Buonpensiero P, Di Pasqua A, Grassia R, Leone CA,Guido S, De Rosa G, Lusa S, Bona G, Stoll G, Maiuri MC, Mehta A, Kroemer G, Maiuri L, Raia V.

Cell Death Differ. 2016 Aug;23(8):1380-93. doi: 10.1038/cdd.2016.22. Epub 2016 Apr 1.

Abstract
We previously reported that the combination of two safe proteostasis regulators, cysteamine andepigallocatechin gallate (EGCG), can be used to improve deficient expression of the cystic fibrosistransmembrane conductance regulator (CFTR) in patients homozygous for the CFTR Phe508del mutation. Here we provide the proof-of-concept that this combination treatment restored CFTR function and reduced lung inflammation (P<0.001) in Phe508del/Phe508del or Phe508del/null-Cftr (but not in Cftr-null mice), provided that such mice were autophagy-competent. Primary nasal cells from patients bearing different class II CFTR mutations, either in homozygous or compound heterozygous form, responded to the treatment in vitro. We assessed individual responses to cysteamine plus EGCG in a single-centre, open-label phase-2 trial. The combination treatment decreased sweat chloride from baseline, increased both CFTR …