A study of an experimental drug from the company Vertex, called VX-770, successfully reduced lung problems in CF patients, and the company hopes to try for approval of the drug later this year. If all goes well, doctors may soon have their first drug to treat the cause of this devastating disease, instead of just combatting the symptoms.
Cystic fibrosis is a genetic disease that impairs lung and digestive function. In particular, the normally thin layer of mucus in the lungs thickens up and impairs breathing; this happens because patients have a faulty version of a protein that helps clear mucus.
About 1800 different mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene have been implicated in the disease. The gene encodes a molecular channel that shuttles chloride ions across cellular membranes, and people with two mutated copies develop mucus-filled lungs susceptible to infection. Few patients live to see their 30s. In 1989, CF became the first disease pinned to a specific gene mutation, without the benefit of knowing the protein first. [Nature]
This newest test was a Phase III trail of Vertex’s drug, which was funded in part by the Cystic Fibrosis Foundation. The treatment goes after one major genetic mutations that causes the disease, called G551.
The results were so staggering that they stopped the study ahead of schedule to get the word out: A giant study by the National Cancer Institute of more than 50,000 heavy smokers has found a 20 percent reduction in deaths among patients who received a CT scan to catch potential cancer as opposed to a simple X-ray.
“This is huge,” said Dr. Reggie Munden, a University of Texas M.D. Anderson diagnostic radiologist who led the research conducted at the Houston cancer center, one of 33 sites nationally. “It’s a massive ray of hope that we can now offer a scientifically proven test to people at risk of lung cancer and pick up tumors before they’re considered lethal.” [Houston Chronicle]
Participants in the study, which began in 2002, had smoked about a pack a day for at least 30 years (or the equivalent—two packs for 15 years). They received a screening via either CT scan or X-ray three times a year. While the X-ray group lost 442 people to lung cancer, the CT group saw only 354 lung cancer deaths.
Your lungs know a bitter sensation when they taste one.
Yes, taste. In a Nature Medicine study, Stephen B. Liggett and company found receptors on the smooth muscle in the lungs that respond to bitterness, similar to the bitter taste buds on the tongue. And, Liggett found, the receptors’ reaction to bitterness is to relax the muscles, and therefore to expand airways. That was totally unexpected, he says, and opens intriguing possibilities for pulmonary treatment—for example, asthmatic symptoms could be treated by exposing these receptors to bitter compounds.
Like tastebuds on the tongue, the receptors react to bitterness, but unlike tastebuds they do not send any signals to the brain. The researchers thought the taste receptors might have evolved as a protection against toxic plants [Boston Globe]
Step 1: Take a rat lung. Step 2: Strip away all of its living cells, leaving only a fibrous “scaffold” of connective tissue. Step 3: Bathe the scaffold in lung cells taken from newborn rats, and put the whole thing in a bioreactor to let the cells multiply and spread. Step 4: A few days later, when the reconstructed lung is again filled with blood vessels and alveoli, transplant the organ into a living rat. Step 5: Watch in awe as the lung begins to function.
That’s the short version of the experiment Yale University researchers just published in Science. The study was a result of a change in direction for lead researcher Laura Niklason:
Niklason spent several years trying to create a synthetic lung scaffold, but in the end concluded it was too difficult. “I decided I couldn’t do it, and probably nobody else could either,” she said. [National Geographic]