New Drug May Treat Cystic Fibrosis

Inherited diseases such as cystic fibrosis can be caused by genetic "nonsense mutations" that disrupt the way human cells make proteins. David Bedwell, Ph.D., a professor in the University of Alabama at Birmingham (UAB) Department of Microbiology, says scientists are now closer to producing drugs that will fix this disruption and drastically improve treatment of genetic disease.

Bedwell is a renowned researcher on the select group of genetic alterations called nonsense mutations - DNA alterations that can lead to nonfunctional or missing proteins. He presented recent findings on an experimental drug that may help to treat some cystic fibrosis patients during the Experimental Biology 2010 conference in Anaheim, Calif., April 26. This drug ataluren (formerly called PTC124) also holds promise in treating more than 2,400 different genetic disorders caused by nonsense mutations.

"When you treat a genetic disease, the bottom line is how much of the missing protein do you need to restore to have a therapeutic benefit," Bedwell says. "It comes down to the threshold of protein rescue. For some diseases, it might be 1 percent of protein you need restored, and for other diseases you may need 50 percent of protein restored."

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New drug may help fibrosis patients

U.S. medical researchers say they have demonstrated the effectiveness and safety of a new drug to treat idiopathic pulmonary fibrosis.

Researchers said the drug, pirfenidone, might become the first FDA-approved medicine to treat the condition.

In a Phase III clinical study, investigators said they discovered the oral anti-fibrotic and anti-inflammatory agent slows the deterioration of lung capacity in idiopathic pulmonary fibrosis patients.

Two multinational, randomized, double-blind, placebo-controlled Phase III trials were designed to evaluate the safety and efficacy of pirfenidone for mild to moderate lung function impairment.

Dr. Roland du Bois, a professor at National Jewish Health in Denver, said the results of the trials are very encouraging for idiopathic pulmonary fibrosis sufferers.

"The safety and tolerability of pirfenidone was reassuring," du Bois said. "The principal side effects experienced by patients in the studies were gastrointestinal discomfort and photo-sensitivity, both of which were manageable in the majority of patients.

"When taken in the context of the urgent unmet medical need for new medicines to treat IPF patients, the collective efficacy and safety data from the … studies, corroborated by a similar study in Japan, make a case for the use of pirfenidone in this disease setting."

The researchers presented their findings Sunday in San Diego during the American Thoracic Society's 105th International Conference.

Source : www.timesoftheinternet.com

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FDA approves new drug for enzyme replacement

The Food and Drug Administration recently announced the approval of Creon (pancrelipase), a pancreatic enzyme replacement product designed to help patients with cystic fibrosis and others with exocrine pancreatic insufficiency (EPI) digest and absorb nutrients from foods.

Creon is the first FDA-approved delayed-release pancreatic enzyme replacement product to be marketed in the United States as a result of the agency’s "unapproved drugs" initiative. Creon, which contains a mixture of digestive enzymes extracted from pancreases of pigs, helps patients who lack the enzymes needed to digest fats, proteins, and sugars from food. Creon is approved for use in pediatric and adult patients.

The FDA had required Solvay Pharmaceuticals, the manufacturer of Creon, to submit a Risk Evaluation and Mitigation Strategy (REMS), which the agency has approved. The REMS includes a medication guide advising patients of risks associated with high doses of Creon and the theroretical risk of transmission of viral disease from pigs to patients. A rare bowel disorder, fibrosing colonopathy, can result from a patient’s use of Creon in high doses.

While this condition is serious and may require surgery, patients’ chances of having this condition may be reduced through adherence to dosing instructions stipulated in the labeling. The risks of a rare bowel disorder and viral transmission described in the medication guide are considered to be risks related to all porcine (pig)-derived pancreatic enzyme products, including Creon.

Instructions for dosing based on weight and age should be followed carefully. Creon may be sprinkled on food. Because Creon is a delayed-release drug, patients should never crush or chew the capsule, as this would cause an early release of the enzymes and a reduction in enzyme activity.

“The approval of Creon will allow patients access to an approved pancreatic enzyme replacement product that meets FDA standards for effectiveness, safety, and manufacturing consistency,” said Donna Griebel, M.D., director of the Division of Gastroenterology Products within FDA’s Center for Drug Evaluation and Research (CDER).

Source : www.modernmedicine.com

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New drug gives longer life to cystic fibrosis sufferers

People with cystic fibrosis could now live longer after scientists announced the discovery of a new drug, the first major breakthrough in 10 years.

Sydney clinical trials of Bronchitol have shown the drug increased lung capacity. Announcing the results of the trial yesterday, The Children's Hospital, Westmead cystic fibrosis unit director Dr Peter Cooper said it was the first drug in a decade to show results.


"The Bronchitol trial has shown a genuine increase in lung function for CF patients using the drug," he said.

"With better clearance of mucus from the lungs, CF patients are likely to live a better and longer life."

One child is born in Australia every four days with cystic fibrosis, a lung condition that can cause respiratory failure. It is the world's most commonly occurring genetically inherited disease, affecting 75,000 people worldwide.

Sufferers experience repeated lung infections and blockages which cause irreversible damage and eventually respiratory failure.

More than 40 countries took part in the trial, sponsored by the Australian pharmaceutical company Pharmaxis.

The drug is a dry powder that works to break the cycle of lung infection, inflammation and damage.

During the trial, lung capacity of those involved increased by 6 per cent.

Dr Cooper said it was hoped the drug could extend a person's life beyond the average of 37.

"If approved Bronchitol will be among the first inhaled dry powder products registered for cystic fibrosis and offers patients a treatment option with less drug administration time and the equipment maintenance issues associated with nebulised liquids," Dr Cooper said.

For Georgia Jones, 11, who was born with cystic fibrosis and took part in the trial, the drug has changed her life. Her family noticed a distinct change when she began taking Bronchitol.

"The results speak for themselves," mum Sue Georgias said.

"When our trial period ended we made a formal request for Georgia to continue due to the benefits achieved."

Source : www.news.com.au

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