Tag: Vertex

Promising Cystic Fibrosis Drug Targets the Cause, Not the Symptoms

By Andrew Moseman | February 25, 2011 4:50 pm

A study of an experimental drug from the company Vertex, called VX-770, successfully reduced lung problems in CF patients, and the company hopes to try for approval of the drug later this year. If all goes well, doctors may soon have their first drug to treat the cause of this devastating disease, instead of just combatting the symptoms.

Cystic fibrosis is a genetic disease that impairs lung and digestive function. In particular, the normally thin layer of mucus in the lungs thickens up and impairs breathing; this happens because patients have a faulty version of a protein that helps clear mucus.

About 1800 different mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene have been implicated in the disease. The gene encodes a molecular channel that shuttles chloride ions across cellular membranes, and people with two mutated copies develop mucus-filled lungs susceptible to infection. Few patients live to see their 30s. In 1989, CF became the first disease pinned to a specific gene mutation, without the benefit of knowing the protein first. [Nature]

This newest test was a Phase III trail of Vertex’s drug, which was funded in part by the Cystic Fibrosis Foundation. The treatment goes after one major genetic mutations that causes the disease, called G551.

Read More

CATEGORIZED UNDER: Health & Medicine
NEW ON DISCOVER
OPEN
CITIZEN SCIENCE
ADVERTISEMENT

Discover's Newsletter

Sign up to get the latest science news delivered weekly right to your inbox!

80beats

80beats is DISCOVER's news aggregator, weaving together the choicest tidbits from the best articles covering the day's most compelling topics.
ADVERTISEMENT

See More

ADVERTISEMENT
Collapse bottom bar
+

Login to your Account

X
E-mail address:
Password:
Remember me
Forgot your password?
No problem. Click here to have it e-mailed to you.

Not Registered Yet?

Register now for FREE. Registration only takes a few minutes to complete. Register now »