Miracle Drug for Cystic Fibrosis

Cystic fibrosis is a hereditary disease that causes thick, sticky mucus to build up in the lungs. Decades ago children born with CF would live only into their teens.

Today, the life expectancy has increased to 37 and a new miracle drug is expected to have even better results.

Out of the 25 pills eight-year-old Caleb takes every day for CF, Kalydeco is his favorite.

"So it is very, very, very important and very, very, very helpful," says Caleb Nolan.

Kalydeco is the first medication to target the underlying cause of CF, a single protein called CFTR that doesn't work right.

"In the past we've only been able to treat the complications of cystic fibrosis," explains Dr. Frank J. Accurso, a Professor of Pediatrics University of Colorado Denver, School of Medicine and Children's Hospital Colorado.

CFTR doesn't allow fluids to clean the surface of the lungs that can cause deadly infections.

"The new treatment, Kalydeco, actually makes the protein work better," says Dr. Accurso.

It clears out the lungs, reducing symptoms, and possibly also preventing further damage to the lungs.

Caleb still has to use a machine to shake loose mucus in his lungs, but he's feeling healthier than ever before.

Melissa Nolan
Caleb's Mother
"To know that we have something that we give him now every day that's changing the way his body feels, correcting the underlying defect, there aren't any words for that," says Caleb’s mom Melissa Nolan.

Because seeing her son feeling better, says it all.

Right now, Kalydeco is only approved for use by four-percent of CF patients with a specific gene mutation.

However, the FDA has now fast-tracked Kalydeco-combination-treatments that may help up to 65-percent of cystic fibrosis patients in the near future.

The drug costs $300,000 dollars a year, but almost all insurance companies help with the cost.

MEDICAL BREAKTHROUGHS
RESEARCH SUMMARY

TOPIC: Miracle Drug for Cystic Fibrosis
REPORT: MB #3698

BACKGROUND: Cystic Fibrosis a hereditary disease that affects about 300,000 children and adults in the U.S. This disease is found in the lungs and digestive system and it causes the affected organ to produce unusual sticky, thick mucus. Eventually the mucus causes the lungs to clog and affects the pancreas' ability to absorb and break down food. In earlier years, cystic fibrosis patients could expect to live until they are around 12, but now, medicine has extended lives of these patients all the way until their 40s. (Source: http://www.cff.org/aboutcf/)

CAUSES: Cystic Fibrosis is a hereditary disease that includes a defect in a gene that changes a protein. This protein performs the movement of regulating salt in and out of cells. The disease is passed down by the child's parent, but the gene has to be in both parents for the child to inherit the disease. If only one copy of the gene is present, the disease will not be passed along to the child, but if their child decides to reproduce, they may have a baby that will have cystic fibrosis. (Source: http://www.mayoclinic.com/health/cystic-fibrosis/DS00287/DSECTION=causes)

SYMPTOMS: The signs of having cystic fibrosis vary depending on how severe the disease is. Most symptoms begin during infancy, but others may notice symptoms around adolescence or adulthood. Symptoms may improve or worsen in time, but normal signs of cystic fibrosis include:
* Feeling out of breath
* Wheezing
* Lung infections (Source: http://www.mayoclinic.com/health/cystic fibrosis/DS00287/DSECTION=symptoms)

NEW TECHNOLOGY: A new drug has been fast tracked by the FDA to help cystic fibrosis patients. Kalydeco was recently approved to treat 4 percent of cystic fibrosis patients who have a specific gene mutation. This drug is the key to allowing patients live longer and healthier lives. The fast track program decreases the time of FDA approval and allows patients to receive their medications faster. Kalydeco has helped patients with symptoms such as breathing, wheezing, and the ability to exercise. (Source: http://www.highbeam.com/doc/1G1-329581975.html)

FOR MORE INFORMATION, PLEASE CONTACT:

Elizabeth Whitehead
Media Relations Specialist
Children's Hospital Colorado CF Center
(720) 777-2522
cysticfibrosis@childrenscolorado.org

If this story or any other Ivanhoe story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Marjorie Bekaert Thomas at mthomas@ivanhoe.com.


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