Thursday, June 13, 2013
A team at University Medical Center Utrecht has developed a rapid and quantitative in vitro
intestinal cell-based assay for CFTR
function that could help prospectively identify responders to targeted cystic
fibrosis therapies.1 The researchers are now planning to test how
well the approach can predict drug responses in the clinic.
CF researchers and industry scientists
agreed the assay could be a powerful tool for compound validation and said it
has the potential to guide clinical-trial design if the results are found to
correlate with patient response.
Cain, C. SciBX 6(23); doi:10.1038/scibx.2013.564 Published online June
1. Dekkers, J.F. et al.
Nat. Med.; published online June 2, 2013; doi:10.1038/nm.3201 Contact: Jeffrey
M. Beekman, University Medical Center Utrecht, the Netherlands e-mail: email@example.com
2. Lukacs, G.L. &
Verkman A.S. Trends Mol. Med. 18, 81-91 (2012)
3. Neuberger, T. et al.
Methods Mol. Biol. 741, 39-54 (2011)
4. Sato, T. et al.
Gastroenterology 141, 1762-1772 (2011)
5. Sato, T. & Clevers,
H. Science 340, 1190-1194 (2013)
6. Yu, H. et al. J.
Cyst. Fibros. 11, 237-245 (2012)
7. 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 e-mail:
Hubrecht Institute, Utrecht, the Netherlands
McGill University, Montreal, Quebec, Canada
Proteostasis Therapeutics Inc., Cambridge, Mass.
Reata Pharmaceuticals Inc., Irving, Texas
Royal Netherlands Academy of Arts and Sciences, Amsterdam, the Netherlands
University Medical Center Utrecht, Utrecht, the Netherlands
The University of Texas Southwestern Medical Center, Dallas, Texas
Vertex Pharmaceuticals Inc. (NASDAQ:VRTX),