A McGill University-led team has shown
that Vertex Pharmaceuticals Inc.'s
lumacaftor and other cystic fibrosis transmembrane conductance regulator
corrector compounds target only one of two sequential steps required for proper
folding of the mutated protein.1 The findings suggest there is a
clear rationale for developing combinations of correctors to treat patients
with cystic fibrosis, three of which have been detailed at recent scientific
Philip Thomas, professor of physiology at UT Southwestern
Medical Center and a cofounder of Reata Pharmaceuticals Inc., told SciBX
that the study was the natural follow-on to the two-step folding hypothesis
described by his lab and the Lukacs lab last year.
Cain, C. SciBX 6(24); doi:10.1038/scibx.2013.589
Published online June 20, 2013
1. Okiyoneda, T. et al.
Nat. Chem. Biol.; published online May 12, 2013; doi:10.1038/nchembio.1253
Contact: Gergely L. Lukacs, McGill University, Montreal, Quebec, Canada
Goor, F. et al. Proc. Natl. Acad. Sci. USA 108, 18843-18848
3. Rabeh, W.M. et
al. Cell 148, 150-163 (2012)
4. Mendoza, J.L. et
al. Cell 148, 164-174 (2012)
5. Cain, C. SciBX 5(8);
6. He, L. et al.
FASEB J. 27, 536-545 (2013)
7. Dekkers, J.F. et al.
Nat. Med.; published online June 2, 2013; doi:10.1038/nm.3201
8. Osherovich, L. SciBX
Cystic Fibrosis Foundation, Bethesda, Md.
European Cystic Fibrosis Society, Karup, Denmark
Genzyme Corp., Cambridge, Mass
GlaxoSmithKline plc (LSE:GSK; NYSE:GSK), London, U.K.
The Hospital for Sick Children, Toronto, Ontario, Canada
McGill University, Montreal, Quebec, Canada
Pfizer Inc. (NYSE:PFE), New York, N.Y.
Proteostasis Therapeutics Inc., Cambridge, Mass.
Reata Pharmaceuticals Inc., Irving, Texas
(Euronext:SAN; NYSE:SNY), Paris, France
Traffic Therapeutics Inc., Montreal, Quebec, Canada
The University of North Carolina at Chapel Hill School of
Medicine, Chapel Hill,
The University of Texas Southwestern Medical Center, Dallas, Texas
University of Toronto, Toronto, Ontario, Canada
Vertex Pharmaceuticals Inc. (NASDAQ:VRTX), Cambridge, Mass.