Cystic Fibrosis (CF) Research

Cystic Fibrosis Center

Cystic Fibrosis (CF) Research

The Cystic Fibrosis Foundation is aggressively funding research to utilize multiple ways to provide better treatments, and potentially a cure, for cystic fibrosis (CF).

Multiple new therapies are being investigated:

  • CFTR modulation: January 31, 2012 was a landmark day in the history of CF drug development. On that day, the Food and Drug Administration approved VX-770 (Kalydeco), a CFTR potentiator. This is an oral drug that results in more normal function of chloride channels in patients with the G551D mutation. The G551D mutation is known as a gating mutation. The CFTR protein is in the membrane of the cell, but it does not function as a normal chloride channel.

    Kalydeco results in causing the CFTR protein to transport chloride. Although this medication does not work in patients with two copies of the F508, the most common mutation, Vertex Pharmaceuticals is researching a corrector drug, VX-809. This is thought to allow the CFTR protein to be inserted into the membrane of the cell in those patients with an F508 mutation, and then Kalydeco can help the protein function as a chloride channel. Clinical trials are ongoing and currently studying this.
  • Anti-infective: There are multiple anti-infective therapies being researched. In April 2010, a second antibiotic for nebulization was approved by the FDA: Cayston. This is aztreonam solution for inhalation. It is delivered using a novel eFlow device - the Altera nebulizer. This results in treatment times that are approximately 90 seconds in length.

    Other antibiotics are currently in development including aerosolized amikacin (Arikace), aerosolized Fosfomycin/tobramycin, aerosolized Levofloxacin, tobramycin inhalation powder, and ciprofloxacin dry powder inhaler.