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My NACFC 2015 "Nuggets"

Sunday, October 18, 2015

This post is my second of three blog posts about my experience at the 2015 North American Cystic Fibrosis Conference, the largest international gathering of CF clinicians, researchers, patients* and parents.  To read my first post, click here.

There is way more information than I could easily share on my blog so I highly recommend watching the NACFC plenary sessions that were recorded live: Plenary 1 (future of personalized medicine)Plenary 2 (about clinical trials)  and Plenary 3 (about mental health).  I also recommend reading fellow CF mom Rebecca's blog for other NACFC medical/science updates.

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To easily share what I learned at NACFC, I am sharing them in "nugget" form:

Nugget #1: We have seen an increase of MRSA in CF lung cultures in the last 20 years.  There are currently limited treatment options for MRSA. A trial funded by the CFF called STAR-TOO shows there is benefit to early intervention for newly acquired MRSA.




Nugget #2: The Inhaled Vancomycin Therapy (AeroVanc) study for those chronically infected with MRSA is preparing to start Phase III trials in 2016.  If it succeeds in Phase III clinical trials and approved by the FDA, it will be the first inhaled antibiotic to treat MRSA.




Nugget #3: There may soon be a new way of doing pediatric PFTs.  It's called the Multiple Breath Washout (MBW) and Lung Clearance Index (LCI).  It may eventually come to a pediatric CF Care Center near you.  MBW/LCI was selected as the primary outcome for efficacy in the European Ivacaftor/Lumacaftor study in younger children with CF ages 6-11.  It was also chosen to be the primary outcome in the Saline Hypertonic In Preschoolers (SHIP) study.




Nugget #4: There looks to be a new standardized way of evaluating "silent" CF disease progression in very young children.  It's called Pragma-CF.  This is the work of ArestCF, the Australian Respiratory Early Surveillance Program.




Nugget #5: We are in the age of personalized medicine, particularly with regard to Cystic Fibrosis.  Some people benefit from drugs while others do not. The CFF has recognized this and are actively looking for more tools to figure out the right therapies for each person.




Nugget #6:  Scientists believe personalized model systems (such as intestinal organoids, nasal cells and stem cells) may eventually help predict clinical benefit of new therapies.




Nugget #7: Novel biomarkers may have the ability to give us more information on patients' disease progression.  Doing so may eventually accelerate clinical trials and streamline therapies.  An example of this is the biomarker of mucus.





Nugget #8: The life expectancy of those with Cystic Fibrosis has increased over the last 30 years.  Evidence-based medicine is part of that success.  This slide shows when medications have been introduced to regular CF care and how they correlate:



Nugget #9: Thankfully, this year there are more medications/treatment protocols being evaluated to treat CF than ever before:




Nugget #10: This year is also the year that patient participation in clinical trials for CF is higher than ever before (even higher than was projected).





Nugget #11: The way the CF community does clinical trials needs to be adjusted.  In a recent CFF Caregiver study regarding clinical trials, it was found that 77% of the patient/caregivers report symptom relief as the primary goal of pulmonary exacerbation treatment.  However, respondents thought doctor's primary goal of antibiotic treatment was equally recovery of lost lung function and relief of symptoms.  The study found that overall, patients/caregivers are willing to participate in clinical trial studies, trust their physicians more than study researchers, expressed legitimate concerns regarding treatment duration and consider symptom resolution a high priority.  According to the CF Foundation, understanding the needs of the CF community will hopefully help CF researchers make clinical trials more conducive to successful research.





Nugget #12:  The CF Foundation just turned 60 years old.  It used to be called the "National Cystic Fibrosis Research Foundation." (Just a random fact I thought was interesting.)



Nugget #13: CEO Bob Beall has officially stepped down from leading the CF Foundation.  Dr. Preston Campbell has taken over as CEO.  But, thankfully, Bob Beall will continue to work at the CF Foundation, he will just no longer be leading as CEO.  Bob explained, "this is a train that you can't get off of."  


If you've heard about the biggest "nugget" from NACFC regarding Cystic Fibrosis and mental health, I haven't forgotten about it.  I'll post my thoughts in a few days on the topic in my third and final blog post about NACFC 2015.

One Response to “My NACFC 2015 "Nuggets"”

  1. Very interesting overview of this presentation. Hope that those who attended learned a lot from it! Thanks for sharing.

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