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Denufosol improves Cystic Fibrosis in clinical trials

Cystic fibrosis is a genetic disease that causes a thick, sticky mucus to build up in the lungs and digestive tract. A clinical trial of a new drug called denufosol found that those given the medication for six months prevented some of the accumulation and improved performance on lung tests.

The Cystic Fibrosis Foundation estimates that about 30,000 children and adults in the United States have the condition that clogs the lungs and leads to life-threatening lung infections. The mucus also obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.

Cystic fibrosis is caused by a genetic mutation that disrupts the cystic fibrosis transmembrane regulator (CFTR) protein, an ion channel. Denufosol tetrasodium inhalation solution works by correcting ion transport in patients to enhance airway hydration and mucus clearance by increasing chloride secretion, inhibiting sodium absorption and increasing ciliary beat frequency.

Frank J. Accurso MD of the University of Colorado and colleagues included about 350 children with cystic fibrosis aged five and older in the study called TIGER-1, The Transport of Ions to Generate Epithelial Rehydration. All had a forced expiratory volume (FEV1) at least 75% of normal, indicating normal to mildly impaired lung function characteristic of early cystic fibrosis.

The patients were randomized to receive either 60 milligrams of inhaled denufosol three times daily or a matching placebo for 24 weeks. This was followed by another 24-week open-label safety extension phase.

The patients on the new medication had increase in FEV1 of 0.048 L, approximately 2% over baseline. In addition, the researchers found further lung improvement by 0.115L by the end of the open-label phase. The placebo group, when switched over to denofusol in the open-label phase, also improved by a mean 0.078 L in FEV1.

Big improvements weren't expected, says Accurso, because the drug is primarily designed to prevent or delay loss of lung function rather than act as a rescue therapy.

Denufosol appeared to be safe without serious adverse events or impaired growth of the young patients, suggesting it could be suitable as an early intervention. Intervention for cystic fibrosis early in its course has the potential to delay or prevent progressive changes that lead to irreversible airflow obstructions, the researchers say in their study published in the American Journal of Respiratory and Critical Care Medicine.

A second phase III trial, which is called TIGER-2, is ongoing which will incorporate a longer placebo-controlled treatment phase. Inspire Pharmaceuticals is targeting a potential US commercial launch of the drug for 2012, pending FDA approval.

Source reference:
Accurso FJ, et al “Denufosol Tetrasodium in Patients with Cystic Fibrosis and Normal to Mildly Impaired Lung Function” Am J Respir Crit Care Med 2011.

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Health warning for Dukan Diet

FRANCE'S most popular weight-loss regimes, including the number one Dukan diet, are ineffective and potentially dangerous to people's health, doctors have warned. The Agence Nationale de Sécurité Sanitaire has issued a warning over 14 of the most fashionable diet regimes in France. Researchers at the Institut Pasteur in Lille assessed each regime, including Atkins and Montignac, for its nutritional value and potential side-effects. Head of nutritional research Jean-Michel Lecerf, who led the study, said the diets disrupted the body's natural metabolism and led to serious nutritional imbalances.

In nearly all the diets, the protein content was typically much higher than the recommended daily intake, especially the Dukan diet, which is France's top-seller. Some of the diets contained 10 times less fibre than the recommended level and up to twice as much salt. They also lacked vital vitamins and minerals. The study also pointed to an increased risk of fractures and other bone problems, muscle wastage and cardiovascular problems in some of the regimes.

Dr Lecerf said that in 95 per cent of cases, people who follow a dietary regime regain weight as soon as they finish. In some instances, the weight they regain is greater than the amount lost. He said: “Each regime is less effective than the one before, and the weight gain afterwards is greater each time.” According to the study, about 70 per cent of people in France have followed some sort of weight-loss programme, many without consulting a doctor beforehand.

The Régime Dukan is the most popular diet in France at the moment. Like Atkins, it is high in protein in the initial “attack” phase, but low in fat. Next comes the “cruise” phase, with protein-only days and protein-and-veg days. Potatoes are banned, as are high-calorie vegetables such as peas, carrots and sweetcorn.

More than two million copies of the Dukan book Je ne sais pas maigrir have been sold in France.

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