February 25, 2011 by staff
Cystic Fibrosis, The Cystic Fibrosis Foundation and Vertex Pharmaceuticals in Cambridge, Massachusetts have worked closely to develop and test a new drug that treats the underlying causes of cystic fibrosis (CF), and announced that the first attempts were very fruitful.
It is estimated that nearly 30,000 citizens of the United States and, 500 in the UK suffer from cystic fibrosis, an inherited disease that is caused by a mutated gene. The disease attacks the organs of the body of thick mucus like substance, particularly the lungs and digestive system making it extremely difficult for people to breathe properly or digest food.
More than years after the gene behind cystic fibrosis was identified, a pill that would solve the problem for some people is closer to reality.
Experimental medicine twice a day, goes by the name of VX-770 code. Now there are promising results from a study of people with a specific genetic mutation that affects about four percent of patients with cystic fibrosis.
Lung function was obtained by 10 percentage points on average among those who received the drug compared to those receiving placebo. The improvement was still present six months almost a year after the start of the study.
This improvement is much more “a big problem for CF patients,” Robert Beall, president of the Cystic Fibrosis Foundation says Shots. “He is taller than any other clinical intervention we have ever had in cystic fibrosis.” Beall said there were no significant side effects.
The foundation has supported the development of drugs, including VX-770, and provided 75 million and Vertex for the drug so far. If the medication he is on the market, the foundation will receive royalties on sales.
This pill for people carrying the gene mutation G551D cystic fibrosis, allows a defective protein in cell membranes do a better job moving. Lisa Jarvis has more details on this blog in the haystack. But a key takeaway is that the drug reaches to one of the causes of cystic fibrosis in some individuals rather than just relieve their symptoms.
Now, to be perfectly clear, all we know about the latest press releases. And the Food and Drug Administration have not submitted the drug for review if Vertex plans to do later this year.
But these results, even with many reservations, suggest that the drug is on track. Vertex share jumped 15 percent Wednesday, investors including VX-770 looks even better than they had expected.
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