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The Future of CF

By Victor L. Roggli, MD

No one knows what the future may hold. However, based on the progress that has been

made in the diagnosis and management of CF in the past several decades, one can make an

educated guess about what to expect. I have seen many changes in the treatment of CF over my lifetime, and it is clear that these changes have resulted in a longer life span and a better quality of life for patients with CF. The following is a discussion of my opinions about the future for CF emphasizing medical management. As you will see, I believe that the future for patients with CF is a bright one.

I anticipate four major areas of improvement in CF care. The first regards portability.

When I was first diagnosed with CF, it was difficult for me to travel far because of bulky

equipment necessary for delivery of nebulized medications. Now nebulizers have become

increasingly compact (and hence portable) including hand-held battery-operated devices.

Similarly, treatment for acute exacerbations required hospitalization with delivery of intravenous antibiotics in bags with complicated monitoring equipment. Now, much of this treatment occurs as an outpatient with the advent of PICC lines and plastic balls of self-administered antibiotics. Similarly, oxygen delivery devices have shrunken in size with increased portability. I anticipate that this trend towards portability and mobilization will continue with advances in the design of oxygen delivery devices.

The second area involves a more efficient delivery of treatment. All the various inhalation

treatments required for CF patients take considerable amounts of time. One way I have dealt with this is to multi-task, although that is somewhat difficult to manage with the 20 minutes of vibration from the vest twice per day. In the future, more efficient methods for delivery of these medications will be available to CF patients and their caregivers. One example is the Trelegy device, which delivers a short-acting bronchodilator, a long-acting bronchodilator, and a steroid by means of a single breath inhalation, once per day.

The third area of improvement will involve more effective treatments. Pseudomonas

bacteria are very hardy organisms that can be difficult or impossible to eradicate from the CF

bronchial tree. More effective antibiotics will be available in the future based on potential

molecular vulnerabilities of the tiny beasts. Much of the damage to the lungs of CF patients

derives from their own inflammatory cells causing collateral damage as they attack the bacteria. More efficient anti-inflammatory agents to control this response with minimization of side effects can be expected. Finally, for those individuals for whom transplantation is the most viable option for the severely damaged lungs, we can anticipate further improvements in the transplantation process, including procurement procedures, the surgery itself, and the post-operative treatment. I suspect that improved approaches to preventing rejection of the lung allograft will also be developed, with more effective anti- rejection medications and less likely development of opportunistic infections (as well as more effective treatment of these infections when they do manage to occur).

The fourth area of improvement will involve approaches that will directly improve the

function of the CFTR protein. One approach to the problem of a defective protein is to replace the defective genes that direct the synthesis of the protein. There have been a number of advances in the delivery of a normal CFTR gene to the respiratory system, and I anticipate that there will be further improvements in the efficiency of the delivery of the protein, further increases in time required for additional treatment (due to turnover of the respiratory cells that normally occurs), and fewer chances of significant side effects. Another approach is to chaperone the defective protein to the surface of the cell where it can do its job, and to improve its ability to do that job. The use of modulators like Symdeco and Trikafta are examples of treatments that improve the function of CFTR and in turn improve respiratory function and decrease the occurrence of exacerbations. A side benefit is improved nutrition due to more effective production of bicarbonate by the pancreas.

It is likely that there will come a time when CF is no longer a disease that will destroy lives, cause unbearable hardships on families, and leave parents without a child, and children without a brother or sister. The advances described above will take considerable research effort, large amounts of money, and most of all, time. No one can say exactly when such treatments will be available to patients with CF and their families. Of course, once these problems are solved, new diseases will be discovered, and new medical problems will arise. But CF will eventually become, like the black plague or the great flu epidemic of 1918, just a memory of the past. For those patients who suffer from CF now, the future is brighter than ever before. And there is hope.


About the Author: Victor Roggli is a professor of pathology at Duke University Medical Center in Durham, NC. He was diagnosed with CF in 1963 at Vanderbilt in Nashville, TN. He has had genetic testing twice and is a deltaF508 homozygote. He grew up on a farm in Tennessee and is the third of four siblings. He enjoys Duke basketball and is a karaoke enthusiast, having recorded more than 300 songs. He is married to Linda, the love of his life, and has a daughter, two step-sons and two grandchildren. He is 68 years old, still working full time with no plans to retire.

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