Big Pharma is EVIL.
A new cystic fibrosis therapy dramatically improved patients’ lung function and showed clear signs of targeting the genetic root of the disease, instead of just alleviating symptoms — a breakthrough so long-sought that many doctors and patients are moved to tears when talking about it.
The data, being unveiled Thursday at a national conference in Tennessee and simultaneously published in two leading medical journals, was so persuasive that the Food and Drug Administration last week approved the three-drug combination, called Trikafta — five months ahead of the agency’s deadline. The drug could benefit 90 percent of patients with the disease, a major advance over previous drugs that worked in a tiny fraction of the people with the disease or had more modest effects.
“I’m overjoyed,” said Francis Collins, the director of the National Institutes of Health, who was part of one of the teams that in 1989 discovered the gene defect that causes cystic fibrosis. “Thirty years along, with many bumps along the road and so many people waiting and hoping that something like this would happen — and here we are.”
A new cystic fibrosis therapy dramatically improved patients’ lung function and showed clear signs of targeting the genetic root of the disease, instead of just alleviating symptoms — a breakthrough so long-sought that many doctors and patients are moved to tears when talking about it.
The data, being unveiled Thursday at a national conference in Tennessee and simultaneously published in two leading medical journals, was so persuasive that the Food and Drug Administration last week approved the three-drug combination, called Trikafta — five months ahead of the agency’s deadline. The drug could benefit 90 percent of patients with the disease, a major advance over previous drugs that worked in a tiny fraction of the people with the disease or had more modest effects.
“I’m overjoyed,” said Francis Collins, the director of the National Institutes of Health, who was part of one of the teams that in 1989 discovered the gene defect that causes cystic fibrosis. “Thirty years along, with many bumps along the road and so many people waiting and hoping that something like this would happen — and here we are.”
5 comments:
Wow! And just yesterday I read of a study medication at Northwestern that is able to trick the bodies of celiac disease sufferers into NOT attacking gluten. This would be life-changing, and it has possible future applications for MS and other auto-immune disorders.
Oh man. Hubby died, basically, it having weakened him to the point of being vulnerable to the immediate cause, from Autoimmune Hemolytic Anemia. (They had his count coming back up, but something just blew. No way to know what, after everything they did to try to get him back.)I will channel my urge to be upset that this wasn't in the pipe earlier to praying for it to come online ASAP to help as many pts as possible! Go, Plainfield Susans everywhere! (PS = very close lifelong friend, a retired Eli Lily pharmacologist.)
Sybil, here you go: https://www.upi.com/Health_News/2019/10/22/Northwestern-unveils-potential-celiac-disease-treatment/4091571762889/?mpse=1
They are moderately far along with the celiac angle, but developing this for other autoimmune conditions is a way off. One of my daughters is so sensitive to gluten that she cannot tolerate any cross-contamination, and cannot receive Holy Communion. But she can live if she's careful with what she eats. A niece has MS, and like your husband's condition, this is life-threatening. So let's both pray for this research to prove valid and to blossom to save other people.
And, Sybil, I understand the upset about treatments that were too late to save a loved one. My mother died of cirrhosis of the liver, for no apparent reason (she hardly drank at all), in 1982. She'd had a couple of transfusions in her life, back before they knew about Hepatits C. We figure that's what killed her, but we had no idea at the time. Now, I see ads for the drug that cures it.
That would hurt indeed, K. Hang in.
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