Cystic fibrosis (CF) used to only be a pediatric disease, but with disease-modifying medications, newer antibiotics, and better nutrition, survival rates have increased, making CF now a disease of adults.
Cystic Fibrosis Emerging Managementon Wednesday will review the different medications available for treating CF and will discuss the important differences that exist for long-term survival, which may allow physicians and researchers to further refine their approach to the management of CF. The session starts at 8:45 am in room 214C of the convention center.
“Primarily, our session will focus on presenting data of different medications—what their cost is, how clinically effective they are, and then explain what we are hoping to achieve with these medications over time, and how the survival can be improved by these medications,” said Shahid Sheikh, MD, associate professor of clinical pediatrics at Ohio State University College of Medicine, noting the mean survival for CF was less than five years in the 1940s and 1950s, and now, it’s 42 to 43 years.
“We’ll also review what other therapies might be in the pipeline, which adult pulmonologists need to be aware of.”
Many of the medications Dr. Sheikh and his co-presenters will review involve modifying the CFTR gene. CFTR is a gene that provides instructions for making the CFTR protein, which functions as a channel across the membrane of cells that produce saliva, sweat, and mucus to help maintain the balance of salt and water on many surfaces of the body. When mutations in the CFTR gene occur, it leads to a buildup of thick mucus.
“Over the last few years, there have been a number of studies to reverse the effect of the damaged CFTR gene so these chloride channels and blockages can be reworked,” he said. “As the therapy depends on the type of defect, not every therapy is for everyone—it all depends on what the underlying effect is.”
CFTR modifying medications will be discussed during the session, Dr. Sheikh added.
“The whole philosophy is that we should start these medication before any lung damage is present, so that we not only reverse the disease process, but block the initiation of the disease process, so these kids can have as close to a normal life as possible.”
A study published earlier this year shows the mean survival in Canada was about eight-10 years more than mean survival in the U.S., Dr. Sheikh said. He points to Canada’s universal health care system as one reason for its higher survival rates.
“Everyone gets the same care and therapy, and they don’t have to worry about the cost of the medications or getting connected to the health-care system,” Dr. Sheikh said. “In the U.S., we are divided into two or three different health-care systems. One is a private insurance company, second is the government-sponsored Medicare and Medicaid options, and third, is the group of patients that have limited access or no insurance. It is well known that patients and families that have no insurance or have limited insurance have a harder time getting the medication they need and getting connected to the health-care system.”