UNMC participated in research that led to the development of a new cystic fibrosis drug that will dramatically improve the lives of some with the condition and could lead to improved therapies for many others with CF.
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| John Colombo, M.D. |
The drug — Kalydeco — was approved recently by the Food and Drug Administration.
“Kalydeco addresses the underlying cause of CF,” said Robert Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation, will address the new drug and other developments involving the disease at CF conference at UNMC this weekend. “The science behind the drug has opened exciting new doors to research and development.”
A strong leader
Dr. Beall has been a driving force in the progress being made in CF treatments, said John Colombo, M.D., professor of pediatrics pulmonology and director of the cystic fibrosis program at UNMC and Children’s Hospital & Medical Center.
“Dr. Beall pulls the strings for everything in CF,” Dr. Colombo said. “He has an amazing mind. He’s the guy behind the basic science who has been able to get the scientists together.”
About the disease
Cystic fibrosis is an inherited chronic disease that affects the lungs and digestive system of about 30,000 people in the United States (70,000 worldwide). A defective gene and its protein product cause the body to produce unusually thick, sticky mucus that:
- Clogs the lungs and leads to life-threatening lung infections; and
- Obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.
In the 1950s, few children with cystic fibrosis lived to attend elementary school. Today, advances in research and medical treatments have further enhanced and extended life for children and adults with CF. Many people with the disease can now expect to live into their 40s and beyond.
A changing tide
Kalydeco and other advances stand to improve the CF picture even more, Dr. Colombo said.
“CF treatment is evolving,” he said. “It’s an exciting time in this field.”
This is great news! My daughter, Charlene Marsh, was born with Cystic Fibrosis and during the last six years of her life was treated by Dr. Colombo and his staff. She received the best care at the UNMC and the nurses loved and cared for her as if she was one of their own. I look forward to hearing more about the advancement in CF. My thanks to all who have a heart for research in Cystic Fibrosis.
As a person with CF in their family, I am excited to hear of the great new developments in CF research. Thank you Dr. Beall, et al.