Turmeric corrects cystic fibrosis defect, in mice

Treatment for the underlying problem that causes the lung disease cystic fibrosis might be as close as the kitchen.

Curcumin, a major component of turmeric, allows a defective cell protein that results from the genetic mutation responsible for cystic fibrosis to do its job, at least in animal experiments.

As described in the journal Science, Dr. Michael J. Caplan, from Yale University in New Haven, Connecticut, and colleagues gave curcumin to mice bred with the genetic mutation that occurs in about 90 percent of cystic fibrosis patients.

This mutation, known as delta-F508, involves the cystic fibrosis transmembrane conductance regulator (CFTR) and leads to production of an incorrectly folded CFTR protein. Because it is misshapen, the protein is destroyed by the cell's housekeeping machinery and never gets to its intended location in the cell membrane.

The defect causes problems with mucus buildup in the lungs and respiratory difficulties. Cystic fibrosis is the most common life-threatening disease resulting from a genetic mutation among Caucasians.

In mice carrying the mutation, feeding them curcumin at doses well tolerated by humans corrected an electrical abnormality characteristic of the disease. This effect was only seen in animals with a mutated CFTR gene, not in mice completely lacking the gene.

Caplan's team also found that treating hamster kidney cells with curcumin allowed mutated CFTR proteins to reach the cellular membrane.

The results suggest that "curcumin and curcumin derivatives represent promising new candidate compounds that may prove useful in the search for small-molecule pharmacotherapies for cystic fibrosis and for other protein-folding diseases," the researchers state.

SOURCE: Science, April 23, 2004602.