Breathing easier
Keeping Faith Team
Imagine a world in which people die before they turn 37-years-old. As they age, they suffer from a buildup of thick mucus secretions that block their airways and lead to lung infections. These lung infections can result in numerous surgeries, the development of osteoporosis, or a combination of Type 1 and Type 2 diabetes. This is the world of a Cystic Fibrosis patient.
Cystic Fibrosis is currently considered a terminal disease. However, this may be coming to an end in the near future. The Food and Drug Administration approved Kalydeco in January of 2012. Kalydeco is the first drug to treat the source cause of one of the genetic mutations of the disease. Kalydeco targets the G551D mutation; a mutation that only four percent of the Cystic Fibrosis population have. The implementation of Kalydeco as a treatment option offers hope to those suffering with Cystic Fibrosis and to their families. Kalydeco (pronounced like kaleidoscope) will be available to cystic fibrosis patients in a pill form that will be taken as part of their daily regimen.
Mary Calvert, the Development Director of the Tampa Bay chapter of the Cystic Fibrosis Foundation states the foundation is all about the research, “This is the next generation of drugs. We put a $75 million investment into this drug, and it happened; it’s here. It’s the tip of the sword.”
“We have the science; we have the pharmaceutical partners; we have everything we need except the money,” said Calvert. There have been 30,000 people in the United States diagnosed with Cystic Fibrosis and there are over 1,500 different gene mutations. The Cystic Fibrosis Foundation shows in their Drug Development pipeline that the next form of Kalydeco/VX-809 is currently in Phase 2 of 3 phases, and it targets the Delta F508 mutation, covering 90 percent of the Cystic Fibrosis population. “This proves that our methods are working, it proves that our version of philanthropy is working, and it is making a difference. There isn’t a drug that a patient takes that the foundation did not have a hand in and the more money that we raise, the faster this (Kalydeco/VX-809) will come to market,” said Calvert.
The foundation is working with pharmaceutical companies to screen over 5 million compounds to perfect the Kalydeco/VX-809 treatments, and they hope to see it released within the next two years. “The science is all there. We are learning as we go, and no one else is doing this. We are the first people to venture in to brand new frontiers, whether it be for Cystic Fibrosis or any other genetic disease,” Calvert said.
Cystic Fibrosis is a genetic disease caused by anomalies in human genes. Healthy genes create proteins called Cystic Fibrosis transmembrane regulator (CFTR), a Cystic Fibrosis patient has genes that lack these proteins or their genes aren’t making the protein correctly. This causes their bodies to develop thick, sticky mucus fluids. The addition of Kalydeco into the daily routine that Cystic Fibrosis patients undergo will help to correct their genetic mutation.
“We have identified four possible kids (in the Tampa Bay area) that will be on Kalydeco and we are hoping that they get started on it in the next month. This is a disease that we can make go away. We can take it from deadly to chronic and make it go away. We can make a difference,” Calvert said. “When you come together as a group, you can do it.”
