Cystic fibrosis (CF) is fatal genetic disease in which thick mucous clogs the lungs and the pancreas due to problems with the secretion of ions and fluid by cells of the airways and gastrointestinal tract. Normal secretion depends upon the function of a protein called CFTR (cystic fibrosis transmembrane conductance regulator), which was discovered at The Hospital for Sick Children in 1989. Mutations in the gene encoding CFTR are responsible for cystic fibrosis. In the most common form of cystic fibrosis, the CFTR protein is trapped inside the cell, and is therefore unable to carry out its proper function at the cell surface.

The laboratories of Drs. Marie Egan, Michael Caplan (both at Yale University School of Medicine), and Gergely Lukacs (Sick Kids) demonstrated in a mouse model that curcumin treatment can release the mutant CFTR protein from this inappropriate compartment inside the cell and allow it to reach its proper destination, where it is able to function. Furthermore, oral curcumin treatment was able to correct characteristic cystic fibrosis defects in a mouse model of the disease. Curcumin is a compound found in turmeric, and is what gives the spice its bright yellow colour and strong taste.

"We were able to prove at the cellular level what the Yale group observed in the mouse model of the disease," said Dr. Gergely Lukacs, a senior scientist in the Cell and Lung Biology Research Programs at Sick Kids and associate professor in the Department of Laboratory Medicine and Pathobiology at the University of Toronto. "After having received curcumin treatment, mice with the genetic defect that causes CF survived at a rate almost equal to normal mice. The CFTR protein also functioned normally in the cells lining the nose and rectum, which are areas of the body affected by cystic fibrosis."

Dr. Michael Caplan, the study's senior author and professor in the Department of Cellular and Molecular Physiology at Yale University School of Medicine, said: "In the next phase of this research, we will work to determine precisely how curcumin is achieving these effects and to optimize its potential as a possible drug. Plans are underway for a human clinical trial of curcumin, which will be carried out under the auspices of Cystic Fibrosis Foundation Therapeutics, Inc."

### Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation (US), is working with Seer Pharmaceuticals on a Phase I clinical trial of curcumin to assess safety and dosage parameters in humans. The trial will be conducted through CFFT's Therapeutics Network in four to six sites and will include approximately 25 patients.

This research was supported by the Canadian Cystic Fibrosis Foundation, the Canadian Institutes of Health Research, The Hospital for Sick Children Foundation, Alyward Family/Pitney Bowes Gift Fund, the US Cystic Fibrosis Foundation, the US National Institutes of Health, and a sponsored research grant from Seer Pharmaceuticals to Yale University.

The Hospital for Sick Children, affiliated with the University of Toronto, is Canada's most research-intensive hospital and the largest centre dedicated to improving children's health in the country. Its mission is to provide the best in family-centred, compassionate care, to lead in scientific and clinical advancement, and to prepare the next generation of leaders in child health. For more information, please visit http://www.sickkids.ca.

Note: If no author is given, the source is cited instead.

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