| It's always a good day when the director of the National Institutes of Health says your newly approved medicine is turnings dreams into reality. Studies in the New England Journal of Medicine and the Lancet this week laid out the striking benefits Vertex Pharmaceuticals' Trikafta can offer to 90% of patients with cystic fibrosis, an inherited disease that causes mucus build-up and chronic infections in the lungs. They also help explain why U.S. regulators approved the medicine last week, five months ahead of schedule. Researchers first discovered the cystic fibrosis gene in August 1989, triggering hope for a gene therapy that could reverse damage to a critical protein that helps balance the amount of salt and water in cells. It didn't work. The patients' immune systems quickly rejected the repaired cells. A decade later, Vertex started working with the Cystic Fibrosis Foundation to find treatments that could help damaged proteins work more effectively. Twenty years later, victory. Patients taking Trikafta had demonstrably healthier lungs just a month after treatment. Critically, it appears to be correcting the underlying cause of the disease, said Raksha Jain, the senior author of one paper and an associate professor of medicine at the University of Texas Southwestern. For many patients and families, it's the answer to a prayer. For NIH Director Francis Collins, it's the fulfillment of a song titled "Dare to Dream" that he wrote shortly after the identification of the gene. "The lyrics expressed hope that the gene discovery would lead to effective treatments for cystic fibrosis—that someday we would see 'all our brothers and sisters breathing free,' Collins wrote in an editorial in the New England Journal of Medicine. "It is profoundly gratifying to see that this dream is coming true."—Michelle Fay Cortez Here's what else we're watching: Vapor trails. Some researchers are doing whatever they can to discredit studies suggesting e-cigarettes are dangerous. Who are they? Who pays them? Where did they come from? And why are they so ubiquitous online? Powder row. Johnson & Johnson said that multiple lab tests found no sign of asbestos in a recalled lot of baby powder, putting the health products giant at odds with U.S. regulators who got different results. Safety dance. The FDA wants to help drugmakers find ways to produce more medicines in the U.S., as quality concerns with foreign-made drugs keep surfacing. Congress is asking if the agency has enough inspectors. Prices are too low. Or so says a top FDA official, who says driving drug prices toward the cost of production has led to unintended pitfalls like shortages. But one imperiled cancer drug has made it back to market. Listen up. Want to hear more from Prognosis? Then listen to our podcast, which has explored superbugs, what we share when we share our medical data online, and more. Download it here on Apple devices, and here on Android.  Got this newsletter forwarded to you? Sign up to get it every Thursday by clicking here.
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