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.

The Strive study tested the drug in 161 patients with the mutation and showed that a measure of air exhaled, called forced expiratory volume, increased after 24 weeks and 48 weeks in comparison to placebo…. In the primary goal of the trial, the drug showed an improvement in forced expiratory volume of 10.6% in those on the drug through 24 weeks and 10.5% through 48 weeks. It also showed improvement in other measures, including weight gain, fewer respiratory symptoms and reduction of salt in sweat, a symptom of the disease. Patients on the drug were 55% less likely to experience a pulmonary exacerbation, or worsening of the disease that requires antibiotics, the company said. [Wall Street Journal]

The mutation that accounts for the majority of cases worldwide is called the delta F508. Later this year, Vertex says it plans to release data on the success of treating that mutation with a combination of VX-770 and another drug, VX-809. For now, researchers are celebrating this first success at treating the disease and not merely the symptoms.

“These are potentially game-changing data in cystic fibrosis,” Mark Schoenebaum, an analyst at ISI Group, said in a research note. “VX-770 is the first drug to show good data in a Phase III that actually modifies the disease by binding to a defective protein and fixing it.” [Reuters]

Related Content:
DISCOVER: Cystic Fibrosis: The Promise of a Cure (when we covered the efforts way back in 1994)
DISCOVER: The Second Coming of Gene Therapy
DISCOVER: What’s Wrong With American Medicine
The Loom: Cystic Fibrosis? Blame Eve

Image: iStockphoto

CATEGORIZED UNDER: Health & Medicine
  • S. Allen

    This is so very exciting! Breakthrough medications don’t happen all that often, many people have waited a long time for this one!

  • Jim Stevenson

    Wish this would work on COPD. It would save many more lives.

  • Jim Stevenson

    Wonderful news for so many people.


    What wonderful hope this gives so many people suffering from Cystic Fibrosis, such a debilitating disease!
    Thank you VERTEX.

  • Joe

    Try curry mixed with black pepper. Cheap and unpatentable. Cure the people. End the lies.

  • Sue and Steve Glee

    Having 2 children, 12 and 9 with CF and lung and liver complications, we have been praying for this drug to get past phase 3 of the research studies towards helping to subside their symptoms. Thank you, thank you , thank you Vertex Pharmaceuticals, and we will pray for it to be on the market ASAP.

  • Anonymous

    What a great find. Now if we could develop the lemongrass cancer cure to this point… Also I’m fairly sure curry and black pepper don’t alter genetic structure.

  • Cathy

    A friend of a friend in Israel died of CF last week. She was shortlisted for a lung transplant, but no organs were available before it was too late. She died at the age of 23.

    Any progress is big news for this terrible disease. I wish your children the best of luck, Sue and Steve.

  • Matt B.

    Okay, Joe, are you supposed to eat it, inhale it, inject it, smoke it, shove it up your butt, or what? Not to mention curry isn’t a single thing, so it’s somewhat variable. If you really wanted to help people, you’d give real instructions.

  • Matt S.

    Think he meant turmeric, which is a component of some curries. To the best of my knowledge that was only shown to be effective in CF mice. My wife gives our kids turmeric anyway in the hope that it will help them. This drug (drugs if you include 809) didn’t come about because some greedy drug company was looking to make a ton of money. There isn’t much to be had, and the R and D on a new drug isn’t worth the risk for an orphan (small number of people have it) disease like CF. No, it came about because the CF foundation, which is funded to a great degree by the CF community (those afflicted and those that love them) gave vertex the money to research and develop the drug. I’m not sure that the pharmaceutical companies are always the good guys. They definitely aren’t the bad guys in this case. My kids don’t have G551, they have D508. Hoping that the combo 770 and 809 trial turns out well. I’ll have one hell of a party!


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