09/01/2021 - Researchers Explore Trikafta in Younger Patients – This article describes four research projects looking at extending the use of Trikafta to children, including infants. 09/02/2021 - New bipartisan legislation can encourage needed antibiotic development – Whether or not you agree with the legislation noted in this article, the discussion provides insight to the economics affecting the development of new treatments. 09/03/2021 - Why We Chose Surrogacy After My Wife With CF Had a Lung Transplant – This blog shows one path to parenthood despite the hurdles raised by CF. 09/04/2021 - Trikafta Helps Lung Function in CF Patients With Certain Mutations – There may be no major revelations in this study, but confirmation is good. One interesting point: there was improvement with Trikafta even where the individual had taken other modulators. 09/05/2021 - Questioning the Psychology of Need – Like many aspects of chronic illness, CF may provide a different analysis of need than the “normal” view. 09/06/2021 - CF Foundation Strongly Urges Universal Masking in Schools – School is open. Vigilance is the key to a healthy year. New York's recently issued guidelines are consistent with the CFF recommendations. 09/07/2021 - Research Overview – Cystic Fibrosis Sinus Disease - Management of complications secondary to CF, such as sinus disease, is important for maintaining an individual's health and quality of life. CF researchers Daniel Beswick, MD, and Anna Zemke, MD, PhD, alongside Eliza Callard, an adult with cystic fibrosis, discuss new research on this important topic including studies looking at the impact of highly effective modulators on sinus disease and sinus surgery outcomes. 09/09/2021 - Minorities With CF Have Worse Lung Function, Study Says - Like many early studies, this one identified an issue that requires more work to understand the “why.” 09/10/2021 - In cystic fibrosis, above-average height in early life tied to highest pulmonary function – Another study that identifies statistics that need further explanation, including whether there is any cause and effect to the correlation. 09/12/2021 -
Cystic Fibrosis Foundation has issued the following clinical trial alerts in September. September 7, 2021 Study to evaluate nebulized BX004-A in adults with cystic fibrosis and chronic Pseudomonas aeruginosa (BiomX Phase 1b/2a) Status: Enrolling Description: This study is testing the safety and tolerability of nebulized BX004-A, 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 (PsA) to find the best dose. Age: 18 Years and Older Mutation: No Mutation Requirement Fev1% Predicted: 40% or greater Number of Visits: 9 Length of Participation: 192 days ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT05010577 Study to evaluate nebulized BX004-A in adults with cystic fibrosis and chronic Pseudomonas aeruginosa (BiomX Phase 1b/2a) Status: Enrolling Description: This study is testing the safety and tolerability of nebulized BX004-A, 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 (PsA) to find the best dose. Age: 18 Years and Older Mutation: No Mutation Requirement Fev1% Predicted: 40% or greater Number of Visits: 11 Length of Participation: 189 days ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT05010577
Within an hour, this quiet scene from Saturday, September 11th was abuzz with golfers lending a hand to make CFFC's Living with CF Golf Tournament a success. It was an all-around great day. Great weather. Great fun. Great result for CFFC's programs. Twenty-five players competed in a closely contended 9-hole scramble. The first place team had a comfortable margin but there were ties for the second and third lowest scores. [The ties were resolved by the compared scores on the toughest holes.] Here are the final standings: First place, with a score of 28, went to Tim Meyers, Aaron Huss and Jason Hogan. Dan Sullivan, Jamie Romeo, Jim Fetzner and Kristen Shaw took second place with a score of 33. Dan also took the prize for closest-to-the-pin on the 9th hole. Dave Fitzgerald, Nathan Dederick, Jeremy Cranker and Keith Kaufmann also scored a 33 for the third place slot. A score of 33 earned the fourth place finish for P Gannon Fitzpatrick, Jeff Goldberger, Allan Mason and Steve Scofield. The closest-to-the-pin prize for the 2nd hole was claimed by Steve. Fifth place honors were earned by Mike Sellars, George J Eiff, George R Eiff and Jake French with their 34. The other score of 34 by Jackie Goldberger, Sue Smith, Carl Jutzin and Greg Smith produced a sixth place finish. Of course, there are several components in a successful event. One key element has already mentioned: the players listed above. Having players wouldn't do much good if they didn't have a place to play. We again thank Southern Meadows Golf Club for hosting our event. Club pro, Mike Clawson, acted as official scorer as well as event coordinator. Double duty was also performed by Rick Clawson with his efforts as course superintendent and lunch grill master. The Living With CF Golf Tournament is our major fund raising event so the real winners were the local CF community. We netted over $2,800 to fund our Hospital Help Packet and Clinic Baggie programs. For that, we owe a big thank you to the sponsors shown below. We appreciate the ongoing support of our repeat sponsors and welcome some new friends. It should also be noted that some of our golfers paid above the entry fee without asking for sponsor status.
08/16/2021 - Depression, Anxiety Common Among CF Patients – Another study where the results come as no surprise. During our Education Day webinar, there was an interesting discussion of the unique aspects of treating depression and anxiety in the CF community. You can revisit that discussion at https://zoom.us/rec/play/TFItrBaP8TSrVGizXUCZBpsD3O4mqpmewlhC9fi39A7IHXuulVWX440iRPlF0GrvkTXNweqYl_ZRlvyc.pjkXUyebT2K70TPS?startTime=1628115940000&_x_zm_rtaid=F36rWqyeQNawMAsVOEu94w.1629115458423.0daaca1d35e2ce79e3e5242e872317c1&_x_zm_rhtaid=370 08/17/2021 - Back to School in Scotland – Although some of the details relate to the specific conditions in Scotland, the general guidance is universal – and timely. The key is establishing a working relationship with your school. 08/18/2021 - NICU Advice From a Mom of a Newborn With CF – This story is geared specifically to the NICU experience, the the writers advice is applicable to the man challenging times in life with CF. 08/19/2021 - This Is Not Alex’s Story, It’s Ours – A good reminder that we need to be careful in applying lessons from one person's life to another. 08/20/2021 - The COVID-19 Vaccine Did Not Harm My Fertility - This is the first of two stories dealing with the road to parenthood for men with CF. This blog by Gunnar Esiason addresses the intersection of IVF and COVID-19 vaccination. 08/21/2021 - Darcy’s Blog: Everything I Did to Improve Our Chances of IVF Success - This is the second of two stories dealing with the road to parenthood for men with CF. This blog by Darcy Esiason provides extensive commentary on a woman's options for promoting the success of IVF. 08/22/2021 - Way of Producing High-yield, High-purity RNA May Aid CF Treatment – We previously shared an article on a new approach to gene editing. This article reports on research that may make gene editing less expensive. 08/23/2021 - Dancing, golfing, and hiking the Ochil hills: one year of Kaftrio – This series of stories give different perspectives on the affect Trikafta (Kaftrio in Europe) has on the quality of life. 08/25/2021 - Have Fun While Supporting Our Community – There are still a couple of weeks to sign up for ...
Who says you can't enjoy yourself at the same time you are lending aid to others. That's exactly what you do when you play in the CFFC Living with CF Golf Tournament. Come join us on September 11th. You can get the details (and an entry form) in the CFFC Golf Brochure_2021. So what are the good things you help achieve with your entry fee? The money raised through the tournament supports our Hospital Help Packet (HHP) and Clinic Baggie programs. The primary component of these packages are passes for the parking garages at Strong and Highland Hospitals. The clinic baggies also include a granola bar. The parking passes in the HHP for Strong have a value of $63 while the Highland HHP passes are worth $28. The clinic baggie contents cost a little less than $5. You can figure the math on what an entry fee covers, but it is not the monetary value that matters the most. It's the knowledge that somebody understands the stress of hospitalization and clinic visits; that somebody understands the simple convenience of prepaid parking. So, grab your golf clubs and enjoy a round with us knowing that you will bring a little joy to someone facing a difficult day.
08/01/2021 - I’m Finally Falling Asleep Without Battling Anxiety – This is a good version of Joni Mitchell's lyric “you don't know what you've got til it's gone.” 08/02/2021 – Sign up – Did you know you can join in the Education Day webinar on your smartphone? It could be handy if you can't be near your computer at 6:30 on Wednesday (8/4). Be sure to download the (free) Zoom app before hand. [NOTE: Event was held 9/4] 08/03/2021 - Not Letting CF Hold Me Back From Traveling – Trikafta may be the star of this story but planning and precaution have big supporting roles. 08/04/2021 - Doctors Willing to Help Specialists in CF Infant Care, But Concerns Evident – With the differences in US and Canadian health care systems, it is unclear how well the results of this study translate to this side of the border. That said, the conclusion is relevant; that is, more education and coordination between primary care providers and CF specialists is warranted. 08/05/2021 – Another Educational Day – Don't miss this opportunity to see an engaging presentation (including responses to attendee questions) by Gunnar and Darcy Esiaison. This article includes a link to a recording of the program. 08/06/2021 - Working from home isn’t just a perk of lockdown – it’s what keeps me well at the best of times – This story has two threads: people adapting to the protocols of COVID have a glimpse of life with CF, and difficulties of dealing with CF in the workplace. 08/07/2021 - Nutritional Status of CF Children Linked to Teenage-onset Diabetes – Getting kids to eat well is always a challenge but this study may add motivation. 08/08/2021 - CF Sinus Disease Webinar – You can use this link to register for a cystic fibrosis research webinar focusing on sinus disease offered by Cystic Fibrosis Foundation on August 25th. 08/10/2021 - New CRISPR/Cas9 technique corrects cystic fibrosis in cultured human stem cells – A further advance in the technique to correct CFTR mutations. ...
CFFC held another Education Day on August 4th. It was different from previous years because it was virtual rather than in person. However, it was like our prior events in the most important way: it was very informative. Through their stories, Gunnar and Darcy Esiason gave us first person views of "Adapting to the Evolution of Care." As promised, their presentations were entertaining as well as instructional. The learning extended into the question and answer segment of the program. Our participants played a key role by posing thoughtful questions on a range of topics. We even got to see Darcy and Gunnar formulate their approach to a parenting issue. There's no need to go into detail on the presentation or the Q&A. One of the benefits of the virtual session is a full recording of the event. If you missed the live broadcast of Education Day (or you want to relive the experience), you can watch a video of the program. Just click here to access the recording. We also thank our sponsors, Viatris and Vivus, for their financial support of Education Day.
The following clinical trial alerts were issued by Cystic Fibrosis Foundation in August 2021. August 2, 2021 Study to evaluate inhaled AR-501 in healthy adults and adults with cystic fibrosis and Pseudomonas aeruginosa Status: Enrolling Description: This study is testing inhaled AR-501, a drug intended to treat infections in the lung. The study will measure the drug’s safety, how well it works, and how the body processes it. Multiple doses of AR-501 will be tested in both healthy adults and adults with cystic fibrosis who are infected with Pseudomonas aeruginosa to find the best dose. Age: 18 Years and Older Mutation: No Mutation Requirement Fev1% Predicted: 45% or greater Number of Visits: 11 Length of Participation: 6 weeks ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT03669614 August 23, 2021 Study to evaluate VX-121 in adults 18 years and older with cystic fibrosis Status: Completed with results Description: This study evaluated the safety, tolerability, and effectiveness of three different doses of the drug VX-121 in triple combination with tezacaftor (TEZ) and VX-561 (deuterated ivacaftor) in people with cystic fibrosis who have two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation. Age: 18 Years and Older Mutation: Two Copies F508del or One Copy F508del Fev1% Predicted: 40 to 90% Number of Visits: 10 Length of Participation: 17 weeks ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT03912233
07/16/2021 - Darcy’s Blog: Our IVF Process – Timeline, Cost, and All the Nitty Gritty – As Paul Harvey used to say, “The rest of the story.” Here is Darcy Esiason's report on their IVF experience. Her detailed and straightforward explanation is a good guide for anyone considering the process. It will also give you an idea of how great our Education Day program will be. 07/17/2021 - Acknowledging My Trauma Has Made Me a Better Advocate - Hopefully, your path to advocacy won't be as traumatic as it was for this blogger. 07/18/2021 - Researchers Probe Targets to Reduce Mucus Production – Rather than thinning mucus, this research sought to regulate the production of mucus. 07/19/2021 – Education Day Registration Reminder – We have had some early responses to Education Day registration, but we would love to have more. 07/20/2021 - A small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations – This research is another step toward the “no one left behind” goal in treating CFTR mutations. 07/21/2021 - “Government restrictions may have ended – but please keep those of us who are clinically extremely vulnerable in mind” - This item comes from the UK so some of the details differ from the condition here in New York. That said, the underlying message is relevant – and worth sharing broadly. 07/22/2021 - Education Day 2021 FAQs – Can't make up your mind about joining Education Day? Maybe this quick Q&A will convince you that it will be a “can't miss” opportunity. 07/23/2021 - The Benefits of Being Sick – It can be hard to “look for the silver lining; however, this blogger suggests that it is healthy to think about positive instances in an otherwise difficult time. 07/24/2021 - Cystic fibrosis-related diabetes (CFRD) and cognitive function in adults with cystic fibrosis – Have you felt a little off in your memory? This study explored the relationship of CF and CFRD to cognitive abilities. As with many studies, one conclusion is that more research is needed.
Q. When is Education Day? A. The event will be held on Wednesday, August 4th. The program will begin at 6:30 with introductions followed by the keynote presentation by Gunnar and Darcy Esiason. We will take a break to hear from our sponsors at 7:30. Darcy and Gunnar will be back at 8:00 to answer your questions with a wrap up by 9:00. Q. What is the theme of this year's program? A. We have titled this year's program as “Adapting to the Evolution of Care.” The simple title covers a lot of ground. The evolution in CF care includes new treatments (like Trikafta), resulting changes in life expectations, changes in an individual’s support system (like move from parent to self to significant other), and changes in the care team (like moving between centers or turnover in center staff). Cutting across all these aspects are the concepts of advocacy and mental health. Q. Where will Education Day be held? A. Wherever you are comfortable. Our 2021 edition of Education Day will be a virtual event presented using Zoom Webinar. Once you are registered, you will get a link that you can access from anywhere you have an internet connection. Q. How do I register for Education Day? A. Simply follow this link: https://zoom.us/webinar/register/WN_7e4E9Ll3TJC6hCc6pH1z7A. You will be asked to provide five pieces of information: first name, last name, email address, zip code and your relationship to CF. After completing the registration, you will receive an email with instructions for connecting to the event. You will also receive a reminder email the day before the event. NOTE: Your registration confirmation is your ticket to participate, so you must register in order to access the webinar. If additional people in your household want to participate on their own device, they should register separately. Registration will remain open right up to the start of the webinar. Q. How does a Zoom Webinar work? A. As an attendee, joining the webinar is pretty much like watching a TV show where you can see and hear the presenters, but they can't see or hear you. There are, however, a couple of differences. First, the host and panelists will be ...