News

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

Oct

CFFC Facebook Posts – October 2020 – Part 1

10/01/2020 - What Are Your Remaining Medical Needs Post-Trikafta? - Gunnar Esiason gives us reality check in the euphoria associated with the success of CFTR modulators. And he is asking for some information to help him describe the post-Trikafta CF world. 10/02/2020 - Oral antibiotics torpedo Pseudomonas as effectively as IVs – The study cited in this article dealt with the initial Pseudomonas infection. 10/03/2020 - Potential Gene Therapy, SPIRO-2101, Named Rare Pediatric and Orphan Disease by FDA – Further evidence that researchers are not abandoning those in the CF community who cannot benefit from CFTR modulators. 10/04/2020 - Raising an Adult (in Training) – Learning to manage your own care starts at an early age. 10/05/2020 - Study: Screening for Hearing Loss in CF Patients Feasible With Tablet-based Tests – Hearing loss can be a side effect of antibiotic treatments. This study suggests a path to earlier detection. 10/06/2020 - Disability is a Reinvestment, Not a Defeat – The power of a word; the unfortunate power of a misunderstood word. This blog may help you combat misplaced ableism. 10/07/2020 - Drink coffee after breakfast, not before, for better metabolic control – CF-related diabetes is a significant issue in the CF community. This study indicates that those with CFRD may want to consider the timing of your morning coffee. 10/08/2020- Research in focus: Antimicrobial resistance in cystic fibrosis – The report linked in this article describes the efforts funded by Cystic Fibrosis Trust (the UK equivalent of Cystic Fibrosis Foundation) to overcome resistant infections. 10/10/2020 - How Do You Decide What Is Worth the Risk of COVID-19? - This blogger shares her approach to re-entering the world in the era of COVID. She even included a decision tree. 10/11/2020 - Lenabasum Fails to Lessen Exacerbations in CF Patients in Phase 2 Trial – It is important to understand that not all potential treatment are successful. 10/12/2020 - Despite the Detours, I Live a Full ...

Sep

CFFC Facebook Posts – September 2020 – Part 2

09/16/2020 - Researchers discover a specific brain circuit damaged by social isolation during childhood – While this article is geared toward the isolation associated with the COVID-19 response, you can probably see the connection to the isolation that may be experienced by children with CF. 09/17/2020 - Vertex to Seek Expanded FDA Approval for Trikafta After Positive Child Safety Data – This development is profound. Imagine mitigating infections before lung tissue is damaged. 09/18/2020 - My Return to In-Person Clinic Visits – If you have had an in-person clinic visit recently, you can compare your experience. If not, this blog gives you a peek at what to expect. 09/19/2020 - With CF Clinics Reopening, 10 Tips for a Productive Visit – Unlike yesterday's post, Gunnar Esiason's reflection on his first “inside” clinic visit post-pandemic are recommendations that apply to every clinic visit. 09/20/2020 - Next-gen organoids grow and function like real tissues – Advances in methodology have allowed researchers to grow more realistic organ tissue from stem cells. This, in turn, will produce better models for drug testing – and maybe transplantation. 09/21/2020 - Have Our Backs and Push Against Ableism With Us – The general population may be unaware that people with “invisible” disabilities are susceptible to discrimination. 09/22/2020 - Having a Newborn With CF During COVID-19 – While the circumstances are not common (thankfully), the reaction to the CF diagnosis is all to familiar. 09/23/2020 - Money and budgeting at university – former students share their tips – While some of these recommendations are specific to the UK, these students have some tips you may find helpful – in or out of college. 09/24/2020 - Moderna, Vertex Team Up to Develop Gene-editing Delivery Techniques – Not only does this research open new options for treating CF, it is also an important step in serving the CF population with mutations that do not benefit from CFTR modulators. 09/25/2020 - Why I Chose to Go Back to School in Person – ...

Sep

Clinical Trial Alerts – September 2020

Cystic Fibrosis Foundation issued the following clinical trial alerts in the month of September. September 8, 2020 Study to evaluate the effects of CFTR modulators in infants and young children (BEGIN Part A) Status: Enrolling Description: This two-part observational study will look at the effects of CFTR modulators on growth in young children with CF. These drugs are intended to help CFTR protein function closer to normal. Age: 0 Years to 4 Years Mutation: No Mutation Requirement Fev1% Predicted: No FEV1 Limit Number of Visits: 6 Length of Participation: 3 years ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT04509050 Study to evaluate the effects of CFTR modulators in infants and young children (BEGIN Part B) Status: Enrolling Description: This two-part observational study will look at the effects of CFTR modulators on growth in young children with CF. These drugs are intended to help CFTR protein function closer to normal. Age: Less than 6 Years Mutation: No Mutation Requirement Fev1% Predicted: No FEV1 Limit Number of Visits: 6 Length of Participation: 2 years ClinicalTrials.gov link: https://www.clinicaltrials.gov/ct2/show/NCT04509050 September 30, 2020 Study of VX-445 Triple Combination in Teens and Adults With Cystic Fibrosis (CF) Who Have One Copy of F508del and One Copy of a Gating or Residual Function Mutation Status: Completed with results Description: This study evaluated the safety and effectiveness of VX-445 (elexacaftor), tezacaftor, and ivacaftor in combination called TRIKAFTA®. This study was for people with CF 12 years and older with one copy of the F508del mutation and one copy of a gating or residual function mutation. Age: 12 Years and Older Mutation: One Copy F508del Fev1% Predicted: 40 to 90% Number of Visits: 8 Length of Participation: 20 weeks ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT04058353