What’s In The Future for Cystic Fibrosis Patients?

The medical and scientific world makes significant advances in new treatments for cystic fibrosis every year. Cystic fibrosis patients previously had a lifespan of only 16 years when the disease was first recognized in the 1940’s. They now are living well into their adult years with the average age of death 39 years old. With a longer lifespan come other complications for cystic fibrosis patients. Researchers and professionals continue to search for more treatment choices for this noncurable disease.

In 1975 a new blood test was developed and approved by the Federal Drug Administration that helps detect carriers of the mutant gene that causes cystic fibrosis. The cystic fibrosis diagnosis analyzes DNA to find out if the mutant gene is present in the body. This is a controlled test performed in specific labs and is available only with a referral from a doctor.

Researchers are now focusing on a protein called CFTR or cystic fibrosis transmembrane conductance regulator. This new research is changing the focus of treatment for patients with cystic fibrosis. Work is now being done to make the CFTR protein work, as it should in the body of a cystic fibrosis patient. Clinical trials are being done to find out if a new drug can help restore the function of the cystic fibrosis transmembrane conductance regulator. The clinical trial will include healthy patients as well as patients suffering from the disease. The trial will help decide if this new drug will help thin the mucus in cystic fibrosis patients.

Enzymes that normally appear in the lungs can cause complications for cystic fibrosis patients. They may damage lungs by allowing inflammation and decrease the lung’s function. Inhibitors found in healthy lungs normally protect the lungs from these enzymes called proteases. Cystic fibrosis patients do not make enough inhibitors to protect the lungs from the effects of protease enzymes. Researchers are working on antiprotease inhalers that could help to reduce infections and lung infections.

A new class of drug is also being considered for use in thinning the mucus that builds up in cystic fibrosis patients. This new class of drug is drawn from an unlikely source. The tiny microorganisms that cause “red” tides on the coasts of Florida are now being tested for use in treating the effects of cystic fibrosis. These microorganisms that kill fish, contaminate shellfish, and cause severe respiratory problems in humans is now being tested to help prolong the life of a person with CF. Scientists looking for ways to combat the effects of “red” tide have found it is also helpful in relieving symptoms of cystic fibrosis. They believe it may increase the movement of the cilia. Cilia are the thin hairlike substances in the lungs that move fluid across the surface of the lungs. This increased movement helps break up thick mucus and helps keep it from sticking to the lungs and air passages. Tests are continuing to decide if this treatment will be helpful in the future treatment of cystic fibrosis.