SpecialNeeds.com

Cystic Fibrosis affects 30,000 people in the U.S. It causes an imbalance of salt in the body, which affects the pancreas shortly after birth, impedes proper digestion, and causes airways to fill with mucus, leaving patients vulnerable to lung infections and other problems. Patients are often small and malnourished and tend to have overly salty skin due to sweat glands not working properly. Currently, those diagnosed with cystic fibrosis have an average life expectancy of 37 years, but that could change for some patients with a new drug called Kalydeco (generic name ivacaftor), which has just been approved by the Food and Drug Administration (FDA).

Kalydeco targets a genetic mutation in four to five percent of people with cystic fibrosis. It helps to keep the chloride gateway open in cells, resulting in a restored salt balance in the body and a prevention of future lung disease. This is the first time a drug is aiming at the underlying problem that causes cystic fibrosis, not simply treating the symptoms. The FDA fast-tracked Kalydeco and approved it in only three months, given its early signs of promise in clinical testing.

Lindsay Shipp is a 27-year-old in San Diego, CA, who participated in a closely watched clinical trial for this experimental drug. She loves singing and dancing and dreamed of auditioning on Broadway until her lungs became so bad that she had to give it up. When Shipp took ivacaftor, her lungs cleared to the point that her physician told her during a regular check up that they were functioning at 96 percent. This is essentially the same level as a healthy person. Shipp tells the Los Angeles Times that she collapsed on a bench outside the hospital and sobbed with relief after realizing that she would be able to have a full life.

Kalydeco is not a complete cure for cystic fibrosis because it cannot rid the body of bacterial infections that remain even after mucus has cleared the lungs. However, if given to young enough patients--even newborns--it could allow them to lead essentially normal lives.

Although Kalydeco can only impact a small percentage of cystic fibrosis sufferers at the moment, drug maker Vertex Pharmaceuticals plans to develop other drugs that may help a larger number of patients if used in combination with Kalydeco.

As for Lindsay Shipp, she tells the Times, "I feel like there’s someone else’s lungs inside of me. It’s awesome. I feel like I can do anything."

Read more here.