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http://www.scienceda...40422224005.htm

Scientists Correct Cystic Fibrosis Defect In Mice With Turmeric Extract

TORONTO -- Scientists at The Hospital for Sick Children (Sick Kids) and Yale University School of Medicine have found that a compound in the spice turmeric corrects the cystic fibrosis defect in mice. This research is reported in the April 23, 2004 issue of the journal Science.

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."

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http://www.scienceda...40525061756.htm

Study Finds Lung Transplants Increase Survival Of Cystic Fibrosis Patients By More Than Four Years

ORLANDO, Fla. -- Lung transplantation increases the survival of patients with cystic fibrosis by almost 4.5 years on average, according to a study presented at the American Thoracic Society International Conference in Orlando on May 24.

"Initially the risks from lung transplantation for cystic fibrosis patients are quite high, but the risk drops over time, and it becomes worth the risk in the long run," said study co-author Roger D. Yusen, MD, MPH, Assistant Professor of Medicine in the Divisions of Pulmonary and Critical Care Medicine and General Medical Sciences at Washington University School of Medicine in St. Louis.

Cystic fibrosis (CF) is the most common lethal genetic disease in Caucasians, but it affects all races and ethnic groups. The disease affects about 30,000 people in the United States. Approximately 1,000 new cases are diagnosed every year. People with CF live an average of 33 years. They develop severe lung disease, with a combination of airway obstruction, infection, and inflammation that accounts for the majority of deaths from the dis¬ease.

CF patients who receive lung transplants to treat their disease must have a double lung transplant in order for the surgery to be effective, said Dr. Yusen. Lung transplants are not common--approximately 1,000 are performed each year in the United States, and only 150 of them, or 15%, are performed on CF patients. "In the United States, lungs are distributed to patients on the waiting list based primarily on their waiting time, whereas the system for patients waiting for heart or liver transplant depends highly on medical urgency," explained Dr. Yusen.

However, a proposal currently being considered prioritizes patients on the waiting list for lung transplantation based on the medical urgency and the probability of survival after transplantation.

Dr. Yusen and colleagues studied all patients with CF who were on the waiting list for lung transplantation at Washington University School of Medicine and Barnes-Jewish Hospital in St. Louis since the program began in 1988. The study included 247 patients, who were followed through 2002. Most were Caucasian, reflecting the CF population as a whole, and the average age at the time of placement on the waiting list was 28. All the patients had severely impaired lung function and severe symptoms when they were placed on the waiting list.

Of the 247 patients in the study, after one year, 84% of patients were alive while waiting for a transplant, and an equal percentage were alive after having a transplant. After two years, 67% of those waiting for a transplant were alive, compared with 76% who had a transplant. After five years, 55% of transplant patients were alive.

Though transplantation had a significant early death risk compared with continued waiting, the risks significantly decreased over time. Within 15 months, the survival benefit from transplantation became apparent. Based on an average survival of 5.7 years after transplant, the researchers determined that lung transplantation improved the life expectancy of patients with CF by almost 4.5 years.

"Next, we hope to see if transplanted patients are not only living longer, but enjoying a better quality of life than those who remain on the waiting list," Dr. Yusen said.

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http://www.scienceda...40701083818.htm

Surprising Finding Could Lead To New Treatment For Cystic Fibrosis

WINSTON-SALEM, N.C. – The surprising finding that people with cystic fibrosis (CF) produce too little airway mucus – rather than too much, as it commonly believed – could lead to more effective treatments for the genetic disorder, say researchers at Wake Forest Baptist Medical Center.

"It has always been thought, but never proven, that CF causes the body to produce too much abnormally thick mucus that accumulates in the lungs and intestines," said Bruce Rubin, M.D., professor of pediatrics. "However, we have now shown that these patients actually have very little mucus in their airways. This finding could change the way we think about CF treatment."

The research is reported online today in the American Journal of Respiratory Cell and Molecular Biology.

CF is a genetic disease that affects about 40,000 children and adults in the United States. The disease is characterized by frequent respiratory infections, breathing difficulties, and eventually, permanent lung damage. Physicians have always believed that the airways fill with mucus, which normally lubricates and protects the respiratory system. Because people with CF have chronic cough and infection it has long been assumed that the airways were full of mucus.

Rubin and colleagues, however, have shown otherwise. They collected sputum from 12 patients with CF and 11 participants without lung disease and analyzed the contents. Participants with CF had significantly less (70 percent and 93 percent) of two proteins that form mucus than participants with healthy lungs.

"This showed unequivocally there is much less mucus in the CF airway," said Rubin, a pediatric pulmonologist at Wake Forest Baptist's Brenner Children's Hospital.

The research was conducted by Markus Henke, M.D., while he was completing a fellowship at Wake Forest Baptist in Rubin's laboratory. He is now at Philipps-University in Marburg, Germany. Henke has since analyzed the sputum from 35 CF patients and said the results are consistent with the earlier findings.

The researchers have shown that the substance clogging the lungs of CF patients is actually pus. They suspect that the airway in CF patients is chronically infected and that it fills with pus. They also suspect that mucus may actually protect the airway from infection. To test their theory, they will conduct a study in animals to determine if mucus can effectively "soak up" the bacteria that they believe is reproducing in the airway of CF patients.