Beur schreef op 1 november 2019 15:34:
By Carolyn Y. Johnson
Oct. 31, 2019 at 5:15 p.m. GMT+1
Long-awaited cystic fibrosis drug could turn deadly disease into a manageable condition
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.”
The drug is the product of decades of steady, incremental scientific work that began with research in academic laboratories and was pushed forward and funded by patient advocates through an unusual “venture philanthropy” model now being emulated by other patient groups. The leap forward was preceded by many steps — Trikafta is the fourth therapy developed by Vertex Pharmaceuticals, a Boston-based company that has built a lucrative franchise around the disease.
www.washingtonpost.com/health/2019/10...