News

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 ...

Nov

CFFC Facebook Posts – November 2020 – Part 1

11/01/2020 - Topical discussions at the North American Cystic Fibrosis Conference – This news item from Cystic Fibrosis Trust presents highlights from the NACFC as viewed from the other side of the Atlantic Ocean. 11/02/2020 - Advice to New CF Parents From an Adult With CF – Dealing with a child's CF diagnosis is one of the most difficult things a parent will experience. Comfort can come from others who have survived that experience; in this blog, from a someone who has grown from a child with CF into adulthood. 11/03/2020 - New Machine Learning Technologies Could Improve Disease Prediction in CF – The promise of advanced data analysis is not merely predictive; it would also allow early, preventative treatments. 11/04/2020 - Survey Opportunity: Models of Post-Transplant Care Survey – While Trikafta is changing the CF world, lung transplantation is still a significant option. This survey is gathering input on post-transplant care. 11/05/2020 - Canadian Cystic Fibrosis Community in Crisis: Latest Developments as Wait for Life Changing Drug Continues – We are tuned in to the expanding access to Trikafta in the US. This story from Cystic Fibrosis Canada reminds us that access is not universal. 11/06/2020 - My Son's Feeding Therapy Journey – Kids with CF have a lot of challenges, but issues like an eating disorder really add to the stress of parenthood. 11/07/2020 - FDA Approves Bronchitol Inhalation Powder as Add-on Treatment for Adult CF Patients – Additional help is on the way for those who do not benefit from Trikafta. 11/08/2020 - UK CF Medical Association's statement on coronavirus – It is a good practice to look at the issues we are facing at home through the perspective from another country. 11/09/2020 - Not Letting CF Dictate My Life – This is a common story but it has a perspective that is applicable to the pandemic environment. 11/10/2020 - Llama nanobodies could be a powerful weapon against COVID-19 – Okay, this isn't directly related to CF, but ...

Nov

Survey Opportunity: Models of Post-Transplant Care Survey

CFF Community Voice issued a call for survey participation regarding post-transplant care. Here is their announcement. In partnership with the CF Foundation, an international group of CF and transplant clinicians have begun work on determining best practice models of post-lung transplant CF care. To help this group learn from a broader audience about current practices, barriers, and ideal models of post-lung transplant CF care, please consider taking this anonymous, 10-15 minute survey by clicking on the button below by Monday, November 16th. The insights gathered will be used to inform the development of best practices around a shared model of post-transplant CF care. COMPLETE THE SURVEY If you know those within the CF community who have received or have experienced their loved one undergo lung transplantation and may be interested in this opportunity, please share this survey. Additionally, if you have any questions about this opportunity, please contact communityvoice@cff.org. Thank you for your time and consideration. Erin Tallarico, RNClinical Director, Advanced Lung Disease ProgramCystic Fibrosis Foundation Sarah Hempstead, MSPractice Guidelines ManagerCystic Fibrosis Foundation

Oct

CFFC Facebook Posts – October 2020 – Part 2

10/16/2020 - Tobacco Smoke Exposure Limits Symdeko Benefit in Pediatric CF Patients – We know that second-hand smoke is harmful to those living with CF. This study indicates that smoke also reduces the effectiveness of CFTR modulators. 10/17/2020 - Scientists engineer bacteria-killing molecules from wasp venom – Next time you see a wasp (a Korean yellow-jacket wasp in particular), you may want to thank it rather than swat it. 10/18/2020 - Managing Bipolar Disorder and CF – This blog is a reminder that CF does not create an exemption from other disorders that afflict the general population. 10/19/2020 - Toronto Scientist Wins Award for Work Into Alternative Way of Treating CF – This research is good news for those with nonsense CFTR mutations by looking at other protein channels. 10/20/2020 - Contemplating Our Identity – How does CF impact your identity? This blogger throws the community reaction to COVID into the mix. 10/21/2020 - Health Insurance Coverage and Open Enrollment Periods – The 2020 open enrollment period for health insurance starts on November 1. This guide from CFF Compass can help you with making the right choice for you. You can also contact a Compass case manager for assistance. 10/22/2020 - With Trikafta Turning One Today, I LOVE Seeing All the Health Anniversaries – Gunnar shares his thoughts – and thoughts of many others – on the first anniversary of Trikafta. 10/23/2020 - How Scientists Are Going to Tackle a Genetic-Based Therapy for CF – This blog reports on the opening plenary session at the North American Cystic Fibrosis Conference which dealt with the status of gene therapy. The blogger lists his four take-aways from the session but the article includes a link to a video of the session so you can draw your own conclusions. 10/24/2020 – Spreading Holiday Cheer – CFFC wants to brighten your home with a wreath or poinsettia. Check out the details in the link. 10/26/2020 - Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients ...

Oct

Spreading Holiday Cheer

CFFC's core mission is to support the local CF community. That means emotional support as well as financial support. With the COVID protocols, people living with CF may feel even more isolated than usual. CFFC wants to help relieve some of those feelings by offering to deliver a holiday wreath or a poinsettia plant to the home of someone living with CF. We want to add a bit of holiday cheer to the household as well as let them know we care about their well being. Check out the details below. Feel free to pass this message along to others who may be interested. You can share this announcement: Holiday Ad 2020 We will provide the wreath or poinsettia at no cost. However, if you want to add a personal touch by sponsoring the gift contact CFFC president Karly Schuhart at KSchuhart03@gmail.com. While we care for everyone living with CF, this offer is limited to individuals served by the Rochester CF center.

Oct

Clinical Trial Alerts – October 2020

Cystic Fibrosis Foundation issued the following clinical trial alerts in October. October 15, 2020 Study of VX-445 triple combination in children 6-11 years old with cystic fibrosis Status: Completed with Results Description: This study evaluated the safety and effectiveness of the triple combination modulator therapy, elexacaftor/tezacaftor/ivacaftor (Trikafta®) in children ages 6-11 years with CF, who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation. Age: 6 Years to 11 Years Mutation: Two Copies F508del or One Copy F508del Fev1% Predicted: 40% or greater Number of Visits: 9 Length of Participation: 32 weeks ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT03691779 Study to evaluate inhaled molgramostim in adults 18 years and older with cystic fibrosis and an NTM infection in the lungs Status: Completed with Results Description: This study evaluated the effectiveness of inhaled molgramostim (Molgradex), a drug intended to treat nontuberculous mycobacterial (NTM) infections in the lungs of people with CF. Age: 18 Years and Older Mutation: No Mutation Requirement Fev1% Predicted: 30% or greater Number of Visits: 19 Length of Participation: 72 weeks ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT03597347