Author Archive

Jan

CFFC Facebook Posts – January 2021 – Part 1

01/01/2021 - FDA Expands Labels for 3 Vertex CFTR Modulators – Let's start the new year with good news on the scientific front. The expanded approvals will increase the number of people who can benefit from the advances in CFTR modulators. 01/02/2021 - I Am a Nurse with Cystic Fibrosis, and I Got The COVID-19 Vaccine – For anyone torn about the COVID vaccine – or their place in line, this blog may reassure you about accepting the chance at the earliest possible point in time. 01/04/2021 - What I Learned Going Off of Trikafta – This blog is a reminder that there is no perfect solution and you have to pay attention to what your body is telling you. 01/05/2021 - Small Study Shows Tobramycin Safely Treats Lung Infection in Infants With CF – There's been a lot of news on the improvements in adult treatments. One way to improve adult health is to get an earlier start on fighting lung infections. 01/06/2021 - New class of antibiotics active against a wide range of bacteria – This study is not CF-specific; however, the approach is likely to work with resistant bacteria that impact people living with CF. 01/07/2021 - Dancing the Frenetic Routine of a Mom to a Child with CF – There is some level of comfort in routine, but that doesn't make life any less hectic. 01/08/2021 - Arcturus to Develop ARCT-032 as RNA Therapy for CF – RNA therapies are not just for COVID. The exciting thing about this research is that it would fix the CFTR protein regardless of the mutation. 01/09/2021 - Managing Other Health Conditions With CF – As the title of this blog suggest, it will remind you that CF kids are not exempt from other health issues. However, the main story is self-reliance and resilience. 01/10/2021 - Feedback Opportunity: CF Genetic Counseling Recommendations – Here's an opportunity to contribute your thoughts relating to genetic counseling. The researchers are particularly interested in hearing from parents of children 10 years old ...

Jan

The identification of the CF gene has had several impacts, including neonatal screening and advising on the ramifications of a CF diagnosis. Cystic Fibrosis Foundation circulated a request for input on recommendations relating to genetic counseling. Here is the text of their announcement. Topic: CF Genetic Counseling Opportunity: Feedback Survey From: Researchers at Johns Hopkins University, Brigham and Women's Hospital, and Mount Sinai Beth Israel A research team and expert workgroup of CF providers and parents are seeking feedback on a list of recommendations that aim to improve access to CF genetic counseling, with a focus on parents of infants who have had a positive CF newborn screening result. While they are specifically looking for parents of children with CF under age 10, all community feedback is appreciated. These recommendations are a key deliverable of a CF Foundation funded screening improvement grant entitled, “Overcoming barriers to genetic counseling in cystic fibrosis care centers.” This project was reviewed by the Institutional Review Board at the Icahn School of Medicine at Mount Sinai. The anonymous survey will take approximately 15 - 20 minutes to complete; longer if you would like to provide in-depth feedback on the larger white paper. The research team and working group will review and incorporate your critical input. If you are interested in participating in this opportunity, please complete the survey by clicking on the button below by Thursday, January 21st at 11:59 p.m., ET. The survey includes links to a comprehensive draft white paper and an executive summary of the recommendations. COMPLETE SURVEY If you have any questions about this opportunity, please email Elinor Langfelder-Schwind at Elinor.schwind@mountsinai.org.   Thank you in advance for your feedback and we look forward to sharing what we learn with you.   Best Regards,Elinor Langfelder-Schwind, M.S., C.G.C.Mount Sinai Beth Israel Karen S. Raraigh, M.G.C., C.G.C.Johns Hopkins UniversityRichard B. Parad, M.D., M.P.H.Brigham and Women’s Hospital

Dec

CFFC Facebook Posts – December 2020 – Part 2

12/16/2020 - Moderna’s COVID-19 Vaccine FDA Briefing Mentions Cystic Fibrosis – In a sequel to his proclamation on getting the COVID vaccine, Gunnar Esiason shares some observations about the Moderna vaccine clinical trials that related specifically to CF. 12/17/2020 - Birthdays Are Bittersweet – This blog offers insight into a routine occurrence that may challenge the mental health of those living with CF. 12/18/2020 - Wintertime and cystic fibrosis – Our friends in the UK have some suggestions for getting through the winter that are just a useful on this side of the Atlantic Ocean. 12/19/2020 - Despite COVID-19, I Participated in a CF Clinical Trial – While it may seem that the entire world came to a stop with the COVID pandemic, critical functions have continued with safety adaptations. 12/20/2020 - Antifungal drug improves key cystic fibrosis biomarkers in clinical study – This study identified an alternative method for creating chloride channels for those with mutations not helped by CFTR modulators. 12/21/2020 - The Existential Crisis of Going From Dying to Living – CFTR modulators not only affect physical health; they also impact mental health. 12/22/2020 - Holidays in the Hospital – Hospital stays could be less frequent with new treatments and COVID protocols, but it could still happen on a holiday (or birthday). This story is about making the most out of the situation we find ourselves in. 12/23/2020 - Potential Antibiotic Appears to Effectively Treat M. abscessus Infections – Researchers continue to seek – and find – treatments for resistant bacteria that attack CF lungs. 12/24/2020 - An avocado a day keeps your gut microbes happy, study shows – Avocados don't necessarily replace “an apple a day,” but it may be a good addition to your diet. 12/25/2020 - CF Trust Partners With CF Foundation to Develop Gene-editing Therapies – International cooperation comes to CF genetic therapy research. A nice holiday present. 12/27/2020 - Experts at NACFC Gave Me Facts About ...

Dec

CFFC 2020 – The Year in Review

To say that 2020 was "unusual" would be an understatement. It was a year aptly described by Charles Dickens in his opening in The Tale of Two Cities: "It was the best of times; "It was the best of times, it was the worst of times, ... it was the age of wisdom, it was the age of foolishness ..." The "worst of times" culprit was the COVID-19 pandemic; more specifically, the health and safety protocols implemented to mitigate the spread of the virus. The primary components of the protocols (masking, personal distancing, sanitizing and avoiding large groups) were familiar to the CF community. That said, the increased isolation of the population at large aggravated the separation regularly experienced by people living with CF. The Cystic Fibrosis Foundation set an early example by cancelling group activities and converting its outreach to virtual platforms. Most significantly, their flagship program, the North American Cystic Fibrosis Conference, was live-streamed and the archived videos were made available to the public. On a smaller scale, the Western New York CFF chapter conducted their social gatherings on-line. CFFC was also affected. We cancelled our annual Education Day event as a safety precaution for our scheduled speakers as well as the potential attendees. We are in the process of re-imagining the program for a return in 2021. We also hope to put our Living with CF Golf Tournament back on the calendar for 2021. The lead "best of times" story was the expanded access to Trikafta, the game changing CFTR modulator developed by Vertex. We shared stories relating the new life Trikafta users are experiencing. While Trikafta does not work for everyone, we also shared reports of research into additional CFTR modulators and other approaches intended to bring similar relief to the rest of the CF population. CFFC continued our Hospital Help Packet and Clinic Baggie programs but at slower pace as a result of the COVID-related changes at URMC (aided by the benefits of Trikafta). The "special" environment of 2020 inspired two new efforts. With the help of a couple of sewing volunteers, we distributed 46 face masks to 11 families in April. We also brightened up the holidays for 15 families with the delivery of wreaths (14) and one poinsettia. These stories, and more, ...

Dec

CFFC Facebook Posts – December 2020 – Part 1

12/01/2020 - An explainer on genetic therapies and cystic fibrosis – UK's Cystic Fibrosis Trust published a brief primer on DNA and genetic therapies. 12/02/2020 - Not Everyone Has Access to Miracles – This blog is a good look at the two sides of a miracle. 12/03/2020 - Choosing to Not Pursue Lung Transplant – This story has two themes: (1) The decision to pursue lung transplantation is not an easy one, and (2) the introduction of Trikafta may provide more time to wrestle with the decision. 12/04/2020 - The COVID 19 vaccine and CF – The UK is ahead of the US on the authorization of a COVID-19 vaccine. The Cystic Fibrosis Trust take on the situation may give us some insight into our future (although the details are bound to be different). 12/05/2020 - Music May Make Airway Clearance Sessions More Enjoyable for Children – Here's an addition to your inventory of techniques that improve treatment compliance. 12/08/2020 - 12 tips to help you manage your finances over the festive season – The economic ramifications of the pandemic have produced money worries for many families. UK's Cystic Fibrosis Trust provided some advice for managing personal finances during the holiday season. The article lists resources specific to Great Britain; however, there are equivalents in the the US. For example, NY's HEAP program for heating assistance (https://otda.ny.gov/programs/heap/) and CFF's Compass program (https://www.cff.org/Assistance-Services/About-Compass/) 12/09/2020 - Mental Health Post-Transplant – Those who have successful transplantation will tell you that there are trade-offs. This blog reminds us that mental health is a significant component of CF post-transplant as well as before. 12/10/2020 - Children with CF experience mild COVID illness – Researchers funded by UK's Cystic Fibrosis Trust developed data that should ease the concerns about children living with CF. 12/11/2020- Yes, I Will Get The COVID-19 Vaccine When It’s My Turn – With the recommendation for Emergency Use Authorization of the Pfizer COVID-19 vaccine, your decision regarding vaccination is about to get real. Here is Gunnar Esiason's take on the ...

Dec

Survey Opportunity: Genetic-based Therapies

Cystic Fibrosis Foundation is seeking input regarding genetic-based therapies. Here is the announcement from Community Voice: The CF Foundation is conducting a survey to better understand the community’s perspective on genetic-based therapies consisting of mRNA therapy, gene-editing, and gene therapy. As research in this area increases, it is important that we hear from the community to better understand your perspectives and current knowledge of genetic based therapies as well as willingness to participate in genetic-based therapy clinical trials.   The CF Foundation is interested in hearing from all adults with CF and their families; particularly those with nonsense and rare mutations. Results from this survey will be used to better understand the community’s perspective and inform future genetic-based therapy research, clinical trials, and communications.   If you would like to participate in this anonymous, 6 - 8 minute survey, please click on the button below by Monday, December 28th at 11:59 p.m., ET.   Complete the Survey   If you have any questions about this opportunity, please email communityvoice@cff.org.    Thank you for your time and consideration.   Best Regards, JP Clancy, MD Vice President of Clinical Research Cystic Fibrosis Foundation

Dec

CFFC Facebook Posts – November 2020 – Part 2

11/16/2020 - The unique support a sibling can bring – We often talk ab out the important role parents play in CF care, but siblings are also an key part of the support network. 11/17/2020 - Why It Didn’t Matter That My Future Wife Had CF or a Lung Transplant – CF doesn't define the life of spouses and more than it defines the life of the person with CF. 11/18/2020 - Key advance for printing circuitry on wearable fabrics – Isn't science wonderful. Can you imagine sending key data to your doctors, or receiving doses of medicine, simply by wearing a shirt with embedded technology. 11/19/2020 - Praising the Caregivers Who Helped Me Endure – This blog is a salute to National Family Caregivers Month. 11/20/2020 - CF Foundation Seeks Input from Communities of Color – CFF is seeking to make their approach more inclusive. 11/21/2020 - A. fumigatus Lung Infections Linked to Age, Long-term Antibiotic Use – We know long-term antibiotic use has consequences. This study points to a correlation with fungal infections. 11/22/2020 - New effective and safe antifungal isolated from sea squirt microbiome – Yesterday we posted a link to a study on fungal infection risk. The study in this link points to an answer to deal with drug resistant fungus strains (including Aspergillus fumigatus). 11/24/2020 - How I Found Acceptance for My Trikafta Weight Gain – Just in time for Thanksgiving, this blogger talks about coming to grips with one of the “benefits” of Trikafta. 11/25/2020 - NACFC 2020 Sessions Available on YouTube – Here's your opportunity to take a deep dive into current information on CF. Cystic Fibrosis Foundation has created a YouTube page that contains the livestream archive from the 2020 NACFC. 11/26/2020 - Thanksgiving isn’t Lost This Year – A timely message from Gunnar Esiason. While we may not be able to enjoy some traditions, there is still many reasons to give thanks. Happy Thanksgiving everyone. 11/27/2020 -

Nov

Making Holidays Happier

It is CFFC's mission to support CF patients and families in their daily struggle with cystic fibrosis. In this period of increased isolation, "support" can be as simple as letting people know someone cares. It was the vision of CFFC's president, Karly Schuhart, to brighten up the CF community with a little seasonal decor. So we offered to deliver a holiday wreath or poinsettia to any interested household. Fourteen families - ranging from Batavia to Hornell to Auburn - took us up on the offer. The generosity of our supporters added to the bright holiday spirit. Contributions from five friends of CFFC covered the cost of this year's program. Given the positive reactions of the recipients, we plan to repeat the program in 2021.

Nov

NACFC 2020 Sessions Available on YouTube

Cystic Fibrosis Foundation has announced the availability of videos from the 2020 North American Cystic Fibrosis Conference (NACFC). CFF has created a YouTube page that links to recordings from 56 sessions. You can access this archive here: https://www.youtube.com/playlist?list=PLhoQ6vyZhgqqFW-h_VcTBb1WMuFs3UIWW

Nov

Clinical Trial Alerts – November 2020

Cystic Fibrosis Foundation issued the following clinical trial alerts in November. November 4, 2020 Study to evaluate inhaled AP-PA02 in adults with cystic fibrosis and chronic Pseudomonas aeruginosa (Armata Phase 1b/2 MAD) Status: Enrolling Description: This study will look at the safety and tolerability of inhaled AP-PA02, a bacteriophage drug intended to treat infections in the lung. Bacteriophages are specialized viruses that kill very specific bacterial strains. Multiple doses of the drug will be tested in adults with cystic fibrosis and chronic Pseudomonas aeruginosa to find the best dose. Age: 18 Years and Older Mutation: No Mutation Requirement Fev1% Predicted: 60% or greater Number of Visits: 10 Length of Participation: 31 days ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT04596319 Study to evaluate inhaled AP-PA02 in adults with cystic fibrosis and chronic Pseudomonas aeruginosa (Armata Phase 1b/2 SAD) Status: Enrolling Description: This study will look at the safety and tolerability of inhaled AP-PA02, a bacteriophage drug intended to treat infections in the lung. Bacteriophages are specialized viruses that kill very specific bacterial strains. Multiple doses of the drug will be tested in adults with cystic fibrosis and chronic Pseudomonas aeruginosa to find the best dose.  Age: 18 Years and Older Mutation: No Mutation Requirement Fev1% Predicted: 60% or greater Number of Visits: 8 Length of Participation: 29 days ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT04596319 November 5, 2020 Study to evaluate the effects of the triple-combination modulator, elexacaftor/tezacaftor/ivacaftor in children (PROMISE Pediatric Cohort) Status: Enrolling Description: This observational study will measure the effects of the triple-combination therapy, elexacaftor/tezacaftor/ivacaftor, in children with CF. These drugs are intended to help CFTR protein function closer to normal. This study will look at how treatment with the triple-combination therapy affects children with CF across many different aspects of the disease.  Age: 6 Years to 11 Years Mutation: Two ...