Where are we in finding a cure for CF?

The CF Foundation was formed in 1955. This organization supports ongoing research to find new therapies to treat CF, and one day, the cure. CF research has had an huge impact on the length and quality of life for patients with CF. In 1989, scientist identified the gene that causes CF. Within a year, normal copies of the gene had been made and added to CF cells in test tubes correcting the defect. Then scientist developed a way to get the normal genes into laboratory animals using a modified inhaled cold virus. This also worked. In 1993, gene therapy was given to the first person with CF to temporarily correct the defect. However, it is hard to by-pass our immune system and there were problems with the virus used to transport the gene. Questions still remain on how the gene should be packaged, what is the best way to get the packaged gene into the lungs, what will the long term results be and how long will they last. Scientists are working everyday to move forward as gene therapy is felt to be the best way to correct the basic defect. It is important to remember if gene therapy was instituted today, it will not repair any current lung damage, only stop progression of disease. So, it is very important to keep up with your treatments and stay healthy.

Another alternative therapy explored focuses on the gene’s protein product, CFTR (cystic fibrosis transmembrane conductance regulator). This protein’s job is to make a tiny channel for salt and water to move so organs are covered in a thin layer of mucus. When this channel is defective, the balance of salt and water is lost and the body makes thick, sticky mucus. Thick mucus traps the bacteria and clogs airways. Scientists are looking at ways to restore the healthy CFTR protein.

Many CF medicines are being researched through clinical trials. Researchers are always looking for new antibiotics, mucus thinning agents, and anti-
inflammatory medications. New airway clearance therapies are being explored. Lung transplants are serious and difficult procedures, but growing success rates has made them a treatment option for some patients with severe lung disease.

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Can generic enzymes be substituted for name brand?

No. Most insurance requires pharmacies to provide generic drugs when possible and for most of your child’s medicine, this is fine. However, for enzymes this can be a dangerous mistake as the FDA doesn’t regulate pancreatic enzymes. This means the generic product can contain a different amount of enzymes than what is stated on the label. These generic enzymes also differ in ability to resist inactivation by the stomach acid, and the pH required for release of enzymes. Because of treatment failures in patients with CF after taking generic enzymes and the association of fibrosing colonapathy with high daily doses, the FDA initiated a process to require firms to obtain an approved application to market these generic products. But it will take some time before they are removed from the market place or until firms have completed required testing. So, until The FDA approved bioequivalent product becomes available, pharmacist shouldn’t substitute for brand name enzymes.

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Why is TOBI® taken in cycles?

An intermittent 28 day on drug/ 28 day off drug dosing regimen was selected for TOBI® for 3 reasons:

1.A previous clinical study showed treatment effect was maintained for 28 days after the end of 28 day treatment period.
2.Animal studies showed that histological changes in the respiratory tract resolved during a 28 day off drug period.
3.Intermittent therapy may reduce the risk for bacteria to become resistant to this antibiotic.

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Why can’t TOBI® be administered during The Vest therapy?

There are currently no studies using TOBI® during The Vest therapy.  It is the manufacturer’s recommendation to use TOBI® after The Vest therapy as the lungs are opened up and cleaned out. This will allow further drug deposition and the medicine to “sit” in the lungs and fight infection without being worked out during therapy.

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Can exercise be used a substitute for airway clearance treatments?

Exercise is important for everyone to stay healthy and can stimulate a cough bringing up mucus. There are currently no studies showing exercise is equal to airway clearance. So, the current recommendation is to use exercise in conjunction with airway clearance. Intensity and duration of exercise is difficult to measure in young children who are active. It is important to encourage activities such as bike riding, swimming, and skating to develop a healthy lifestyle. Exercise in a formal athletics program is much more regimented and may be substituted for airway clearance with physician approval and as long as pulmonary function is maintained. For example, a high school student in track or football that runs 3 miles during practice and lifts weights for strength training 3 days a week may only do the morning airway clearance treatment on these days when they have practice or a game, health allowing. In exercise physiology, there are many variables to monitor including target heart rate, oxygen consumption, cardiac output, pulmonary function, and ventilatory response. Depending on disease severity, physical activity may be limited or oxygen required. Exercise also burns calories so be sure to supplement your diet to prevent weight loss. Dehydration is also a risk during exercise, especially in the heat. So drink plenty of liquids, avoid colas and carbonated beverages, and add extra salt into you diet. Discuss your exercise plan with your physician.

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Can I take over the counter vitamins?

Vitamins A, D, E, and K are “fat-soluble” vitamins meaning they require fat for absorption in the body. Patients with CF have trouble absorbing fats, so they are usually deficient in these vitamins. There are water-soluble vitamins made especially for CF patient to maintain adequate vitamin levels. These include ADEK and Vitamax. Over the counter vitamins may not be absorbed as they are fat soluble. Talk with your nutritionist or physician about which vitamin is right for you.

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What are the hospital’s visiting hours?

For more information on Arkansas Children’s Hospital visiting policies see
http://www.archildrens.org/your_visit/visiting_policies.asp.

May I stay overnight with my child during hospitalization?

For more information on Arkansas Children’s Hospital visiting policies see
http://www.archildrens.org/your_visit/visiting_policies.asp.

May brothers or sisters visit during hospitalization?

For more information on Arkansas Children’s Hospital visiting policies see
http://www.archildrens.org/your_visit/visiting_policies.asp.