Matthew Cox was 6 weeks old when he was sent to Riley Hospital for Children in Indianapolis with pneumonia. It was only then that he was diagnosed with cystic fibrosis. Doctors believed he would only live to be a teenager.
Now 23 years later the life expectancy for CF patients is 37. Bruce Cox, Matthew's father, said his son is thriving and a new drug has dramatically changed his life. Kalydeco opens the door to cells to allow chloride in, which helps the body's digestive juices and keeps excessive mucus from forming.
Cox said his son will always live with the damage his lungs have suffered until now, but said his hope is that with the new drug there will be no new damage. Children born now within the 4 percent who can take the drug, explained Cox, may never suffer from the life-threatening complications that the disease causes.
CF is caused by a defective gene that causes the body to produce a thick mucus, according to the Cystic Fibrosis Foundation's website. The gene causes a malfunction or total lack of CFTR protein. Mucus clogs the lungs, causing infections, and obstructs the pancreas, which keeps the body from releasing the enzymes that help break down and absorb food. Around 30,000 children and adults in the United States are affected by this disease, 800 here in Indiana.
People who have CF must go through daily treatments to loosen the mucus from their lungs, as well as do numerous breathing treatments, and take synthetic enzymes to help with their digestive track. Putting on weight and keeping it on is always a challenge.
Annie Lickliter, who has two teenagers with CF – Carly, 15, and Park, 18, – said because it only affects a small percentage of the population the government doesn't fund the research as much as they do for diseases that affect a larger segment of the population. It wasn't until 1989 that the gene that causes CF was discovered. After that it was a race to develop drugs that could counter the gene's effect on the body.
Since the recent success of Kalydeco, which allows 4 percent of the CF population to lead a more normal life, there is optimism that more drugs will soon be approved. According to the Cystic Fibrosis Research Inc News, currently the makers of the drug, Vertex, have plans to begin testing several other drugs that could help a broader segment of the CF population. Since 2011 the CF foundation has been working in collaboration with Vertex, investing up to $75 million over five years to speed up the development of drugs that attack the underlying cause of CF.
This Saturday night the Indiana Chapter of the Cystic Fibrosis Foundation will be hosting its 9th annual fundraiser in the Tower Bank lobby. Lickliter, chair of this year's event committee, said 90 cents of every dollar raised there would go toward more research. Last year's event raised $110, 000.
The event, Lutheran Health Network Wine Opener, is presented by the Mary Cross Tippmann Foundation; there will be a VIP reception 5:30-7 p.m. with entertainment provided by Kyrou's Music Studio. The main part of the evening will take place 7-10 p.m. There will also be a silent auction.