New Tool in the Fight Against Cystic Fibrosis

Human tissue with cystic fibrosis developed in laboratory

(RxWiki News) Tissue is the issue when it comes to developing new drugs. Scientists have begun to develop tissue in the lab for testing new cystic fibrosis treatments.

Researchers have figure out how to develop human tissue that has the mutation that causes cystic fibrosis.

New drugs could be developed and tested using the human tissue leading to more accurate results in the near future.

"Ask your doctor about current cystic fibrosis treatments."

The study was led by Jayaraj Rajagopal, M.D., from the Harvard Stem Cell Institute. Researchers first developed induced pluripotent stem (iPS) cells before they could create human tissue. The tissue that was created, lung epithelium, line the surface of the lungs.

Cystic fibrosis causes scarring and damage to the lungs that cannot be reversed. The stem cells came from skin of cystic fibrosis patients.

Cystic fibrosis is caused by a genetic mutation of the protein, cystic fibrosis transmembrane conductance regulator (CFTR). The most common mutation is called delta-508 which removes an amino acid in the 508th position of the CFTR protein. The delta-508 mutation is responsible for 70 percent of all cystic fibrosis cases and for 90 percent of cystic fibrosis cases in the United States, notes researchers.

Having a tool like laboratory grown human tissue can be vital to the development of new drugs. The tissue is disease-specific and contains two common cystic fibrosis mutations. The tissue that was developed by the Harvard researchers contains the delta-508 mutation as well as the G551D mutation.

Cystic fibrosis currently has no cure but recent treatments have led to drastic improvements in mortality rates. Previously, many individuals with cystic fibrosis died as children or teenagers. Due to new treatments, the average life expectancy for cystic fibrosis is around 30 years of age.

Recently, the Food and Drug Administration (FDA) approved the drug Ivacaftor which corrects the G551D mutation. Unfortunately, the G551D mutation only affects two percent of individuals with cystic fibrosis. Despite this limited use, Ivacaftor shows that drugs can be developed to battle against cystic fibrosis. Ivacaftor worked successfully in the tissue created by the Harvard research team.

Previously, getting human lung tissue was difficult because it did not last long in a laboratory, delaying any screening process for drug development according to researchers.

While the development of human tissue that has cystic fibrosis is encouraging, researchers have plenty of work ahead. The tissue to be used to screen drugs that could be used against the delta-508 mutation or cystic fibrosis in general. 

No funding information was provided.

This study was published in the April edition of Cell Stem Cell.

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Review Date: 
April 10, 2012