Is There a Cure for Cystic Fibrosis?

As with many conditions, CF research is funded by dedicated organizations that raise funds, secure donations, and fight for grants to keep researchers working toward a cure. Here are some of the main areas of research right now.

Gene replacement therapy

A few decades ago, researchers identified the gene responsible for CF. That gave rise to the hope that genetic replacement therapy might be able to replace the defective gene in vitro. However, this therapy hasn’t worked yet.

CFTR modulators

In recent years, researchers have developed a medicine that targets CF’s cause, rather than its symptoms. These drugs, ivacaftor (Kalydeco) and lumacaftor/ivacaftor (Orkambi), are part of a class of drugs known as cystic fibrosis transmembrane conductance regulator (CFTR) modulators. This class of drugs is designed to affect the mutated gene that’s responsible for CF and cause it to properly create bodily fluids.

Inhaled DNA

A new type of gene therapy may pick up where earlier gene therapy replacement treatments failed. This newest technique uses inhaled molecules of DNA to deliver “clean” copies of the gene to the cells in the lungs. In initial tests, patients who used this treatment showed modest symptom improvement. This breakthrough shows great promise for people with CF.

None of these treatments are a true cure, but they’re the greatest steps toward a disease-free life many people with CF have never experienced.

Today, more than 30,000 people are living with CF in the United States. It’s a rare disorder — only about 1,000 people are diagnosed each year.

Two key risk factors increase a person’s chances of being diagnosed with CF.

• Family history: CF is an inherited genetic condition. In other words, it runs in families. People can carry the gene for CF without having the disorder. If two carriers have a child, that child has a 1 in 4 chance of having CF. It’s also possible their child will carry the gene for CF but not have the disorder, or not have the gene at all.
• Race: CF can occur in people of all races. However, it’s most common in Caucasian individuals with ancestry from Northern Europe.

Complications of CF generally fall into three categories. These categories and complications include:

Respiratory complications

These aren’t the only complications of CF, but they are some of the most common:

• Airway damage: CF damages your airways. This condition, called bronchiectasis, makes breathing in and out difficult. It also makes clearing the lungs of thick, sticky mucus difficult.
• Nasal polyps: CF often causes inflammation and swelling in the lining of your nasal passages. Because of the inflammation, fleshy growths (polyps) can develop. Polyps make breathing more difficult.
• Frequent infections: Thick, sticky mucus is prime breeding ground for bacteria. This increases your risks for developing pneumonia and bronchitis.

Digestive complications

CF interferes with the normal functioning of your digestive system. These are a few of the most common digestive symptoms:

• Intestinal obstruction: Individuals with CF have an increased risk of intestinal obstruction because of the inflammation caused by the disorder.
• Nutritional deficiencies: The thick, sticky mucus caused by CF can block your digestive system and prevent the fluids you need for absorbing nutrients from getting to your intestines. Without these fluids, food will pass through your digestive system without being absorbed. This keeps you from getting any nutritional benefit.
• Diabetes: The thick, sticky mucus created by CF scars the pancreas and prevents it from functioning properly. This may prevent the body from producing enough insulin. Additionally, CF may prevent your body from responding to insulin properly. Both complications may cause diabetes.

Other complications

In addition to respiratory and digestive issues, CF can cause other complications in the body, including:

• Fertility issues: Men with CF are almost always infertile. This is because the thick mucus often blocks the tube that carries fluid from the prostate gland to the testes. Women with CF may be less fertile than women without the disorder, but many are able to have children.
• Osteoporosis: This condition, which causes thin bones, is common in people with CF.
• Dehydration: CF makes maintaining a normal balance of minerals in your body more difficult. This can cause dehydration, as well as an electrolyte imbalance.

In recent decades, the outlook for individuals diagnosed with CF has improved dramatically. Now it’s not uncommon for people with CF to live into their 20s and 30s. Some can live even longer.

Currently, treatment therapies for CF are focused on mitigating the signs and symptoms of the condition and the side effects of treatments. Treatments also aim to prevent complications from the disease, such as bacterial infections.

Even with the promising research currently underway, new treatments or cures for CF are still likely years away. New treatments require years of research and trials before governing agencies will allow hospitals and doctors to offer them to patients.

If you have CF, know someone who has CF, or are just passionate about finding a cure for this disorder, getting involved in supporting research is quite easy.

Research organizations

Much of the research into potential CF cures is funded by organizations that work on behalf of people with CF and their families. Donating to them helps ensure the continued research for a cure. These organizations include:

• Cystic Fibrosis Foundation: CFF is a Better Business Bureau-accredited organization that works to fund research for a cure and advanced treatments.
• Cystic Fibrosis Research, Inc.: CFRI is an accredited charitable organization. Its primary goal is to fund research, provide support and education to patients and families, and raise awareness for CF.

Clinical trials

If you have CF, you may be eligible to participate in a clinical trial. Most of these clinical trials are conducted through research hospitals. Your doctor’s office may have a connection with one of these groups. If they don’t, you may be able to reach out to one of the above organizations and be connected to an advocate who can help you find a trial that is open and accepting participants.