Otwarcie drzwi do badań klinicznych nad mukowiscydozą

Otwarcie drzwi do badań klinicznych nad mukowiscydozą
Październik 15 06:41 2015 Print This Article

There have never been as many new CF drugs in development as there are today. In the second plenary at NACFC, Dr. George Retsch-Bogart outlined the progress we’ve made, the road ahead and the changes needed to make it all possible. Read on for my key takeaways.

October 9, 2015

I came away from the second NACFC plenary, “Opening Doors to CF Clinical Research: Change Is Coming,” in awe of how far we have come in clinical research and excited about what is coming in the next year.

George Retsch-Bogart, M.D., a pulmonologist at the University of North Carolina and faculty director of network development at the CF Foundation’s Therapeutics Development Network, started his talk with a moving clip of Linda Bowman, a 54-year-old with CF who began participating in clinical trials before the first CF drug, Pulmozyme, was approved. Linda reminds us all why clinical research is so important: It has added years to the lives of thousands of people living with CF.



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Developments in clinical research mean that someday soon people with CF will simply expect to grow old; our hope for a cure will become their reality. Dr. Retsch-Bogart described how doors are opening in CF research, and progress is being made not only indisease-modifying drugs like Kalydeco™ and Orkambi™ but also in treatments to help people with CF manage the symptoms of their disease, like treatments for MRSAinfections and best practices for pulmonary exacerbations.

This has been a year of unprecedented growth in CF research. Almost three times the number of new studies have started up this year than in previous years, with 38 major studies underway and a pipeline full of innovative approaches to treat the symptoms and underlying cause of CF.

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What will it take to perform these clinical trials? The latter half of the plenary shifted focus from the trials themselves to what could be the most challenging aspect of carrying them out: increasing participation in CF clinical research. Even with an outstanding network of research centers, participation of people living with CF in clinical research is at the heart of moving research forward.snap_001

Dr. Retsch-Bogart shared results from a survey of people with CF and their families about their needs and preferences around clinical trials, such as wanting to hear directly from their doctor about whether a clinical trial is right for them.

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He also discussed the importance of making it possible for all people to participate in research studies even if they are not taking place at their care center. In another video, Dr. Susanna McColley and Ryan Maguire, the parent of a 6-year-old with CF, talked about the experience of being referred to another center for a clinical trial, illustrating the importance of collaboration between care centers.

Now more than ever it will take the entire CF community to help move clinical research forward. Change is coming in clinical research, and with that change comes the promise of new therapies for all people with CF, turning the hope of a vibrant future into a reality.

If you did not have the chance to watch this plenary live,   watch a recording of the second plenary here.

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