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Therapy of the protein
- Question
- Hello,
We have learned in September by the "INSERM" (french national institute for health and medical research), about the discovery of 2 molecules acting on deltaF508 CFTR protein. What is the difference between theses 2 molecules and Vertex’s molecules (Ivacaftor® and Lumacaftor®) that are tested in combination for efficacy in a phase 3 trial for patients carrying deltaF508 mutation. Thanks
- Answer
- Hello
These new molecules are correctors acting on class 2 mutations (like ∆F508) and can be compared only to Lumacaftor® (Ivacaftor® is a potentiator acting on mutations other than class 2, like G551D). The mode of action of molecules discovered by the INSERM is to interrupt the interaction between deltaF508 CFTR (mutated protein) and a protein named the keratine 8, which is present in the cell. The disappearance of this link facilitates the deltaF508 CFTR's migration to the membrane, where it can exercise a certain level of activity.
Lumacaftor® acts directly on the mutated CFTR protein. This action allows the protein to reach the surface of the membrane.
Best regards,
Anna Ronayette
- 08.01.2014
