Cystic Fibrosis Letter, 2023 Please donate by December 1, 2023
Dear Friends and Family,
The glorious days of fall color are surrounding us here in Ohio. The importance of family and friends is on our hearts as we hear the news and the tragedies here in our country and overseas. We hold you all in our thoughts and prayers. You all are a blessing to us.
Our fundraising efforts this year are in memory of our supporters in CF who passed away this past year: Carrie Pierson, Bill Peck, Bob Willke, Matthew Russell, Melody Schreiber, and Nancy Slack.
We are happy to report that life “in the world of Cystic Fibrosis” holds many positive outlooks in our family and in research. Brent (48) has had a year full of change. He and wife Christine made a permanent move to Charleston, S.C. about a year ago. They purchased their first home and are again exploring their favorite city with dog, Leia. Having a yard for the first time has been fun and they have become expert gardeners. Brent continues into his 28th year of working for BP. but this time all remote. For the first time in approximately 15 years Brent is not taking a daily antibiotic as part of his CF medications. This is all thanks to the drug, Trikafta.
Derik (45) has had a much improved health year. He continues to work with doctors at The Ohio State University on regulating his CF induced diabetics. One concern in the Pace household is the availability of some medicine. ADHD meds (Derik and sons) are hard to locate and lately Derik has also had trouble locating his CF enzymes. This is stressful and sometimes requires 30 minute car trips to pick up meds and with no guarantees for the next refill. Derik continues in his 6th year employed by SODEXO. Misty is in her 9th year of teaching elementary school and Jim and Linda both volunteer in her 3rd grade classroom (as well as another Columbus Public School 1st grade). Zach (13) is in 8th grade and continues to have interests in art and basketball. Jeffery (10) is in 5th grade and enjoys piano playing and computer programming. He and Linda (30+ years older than the other adults) recently returned from school overnight camp at a YMCA camp. The hike on day one was challenging to a 75 year old but not a 10 year old! FLASH: if you think you see a groundhog in your yard it may be a guinea pig! An animal first identified by mom Misty as a groundhog but by Jeffery as a guinea pig was truly a guinea pig living under their raised air conditioner. It now resides inside in its own cage with 4 cats in the house!
Jim and Linda both continue to be involved in music. Jim is singing and playing trombone in the Westerville Community Band along with church music involvement. Linda is in her 35thyear playing flute in the band. Linda and her walking friend, Margaret, are trying to walk at every Metro Park (we have been to half). To celebrate her 75th birthday, Linda and her childhood friends took a cruise to Canada and Maine just a few weeks ago. While Linda was gone, Jim was exploring the construction site near our condo and fell on some rebar - 9 stitches in his hand! We remodeled our kitchen this year and are enjoying the new layout. Jim continues his volunteering on the condo board and lots of areas at our church… and in his spare time he runs his small computer business.
Jim’s research report (rather technical but interesting):
In our letters for 1997, 2002, and 2005, I mentioned clinical trials to correct the CFTR gene defect that causes CF using a modified virus introduced into the lungs. The virus DID correct the CFTR defect, but also caused an inflammatory response in the lungs.
The lungs make a convenient place for clinical tests, but the body has all kinds of natural defense mechanisms against germs and other foreign bodies.
So, what do you do?
GenexGen Inc. is developing a drug designed to temporarily “turn-off” the body’s immune system response to a “cold-like” virus.. This would allow the virus to carry the corrected CFTR gene into the lung and fix the CFTR defect (but without the cold!).
Definition: Extracellular vesicles (EV) - cell “pimples” that naturally “break off” from cells to form tiny particles. They have many biological functions – some good, some not so good. The most important function here is the ability to transfer proteins, DNA, and/or RNA.
Carmine, Inc. plans to use some cell “pimples” (the EVs) to deliver healthy CFTR genes into lungs. The expectation is this delivery method will not trigger an immune system response in the lungs.
Definition: Nonsense mutation – a change to an area of DNA or RNA that interrupts the instructions to make a protein. The protein is incomplete and doesn’t work right.
Specific Biologics Inc. is testing a gene-editing approach to correct the 3 most common CF nonsense mutations. Hopefully, this approach will work on any mutation, and on other diseases, as well.
Definition: Bacteriophage (or “phage”) – a small virus that attacks bacteria. Used since the late 20th century as an alternative to antibiotics in the former Soviet Union, Central Europe, and France.
Gensaic Inc. is exploring how to make “phages” to fix the CFTR gene defect. Researchers believe phage-derived particles might be effective because they’re unlikely to trigger an immune response.
Other Research Notes:
Trikafta is a three-drug combination taken in two doses per day. It was approved in 2019 and was a game-changer for over 90% of people with CF (including Brent and Derik). In April, 2023, Trikafta was approved for children with CF 2yrs and older. Work is ongoing to develop similar three-drug combinations that can be taken once per day instead of twice.
Sionna Therapeutics is developing drugs that target the region of the gene (called NBD1) where the most common CF mutation (F508del) resides.
As you can see, researchers haven’t stopped looking for better outcomes for people with CF. And much of their research will improve ideas and techniques for other diseases.
As we embark on our 45th fundraising effort for The Cystic Fibrosis Foundation, we are in awe of our long time supporters and those of you who have just joined our efforts. “It takes a village” and that village includes ALL of you and whatever you can donate. We thank you in advance for joining us in an effort that began in 1978 when Derik was diagnosed with CF in December with Brent’s diagnosis following before the New Year. It was a very emotional time for a couple who had been married seven years and thought they would just be adjusting to having their second child. We had and have such support that has enriched all aspects of our lives (when you get lemons...you make lemonade and there has been lots of lemonade throughout the years.) Read below about how you can donate.
The
easiest way to donate
from
your web browser is to Click
here (or
type
“https://cf.glenabby.org/” into
your web browser) to go to the “CF Letters from the Pace’s” web
page and click on one of the pictures at the bottom of the page!
You can also mail your contribution to us at:
CFF
Annual Fund
c/o James M. Pace
6251 Abby Gate
Ct
Westerville, OH 43081
Checks should be payable to “Cystic Fibrosis Foundation”
Please donate by December 1, 2023
Love,
Jim, Linda, Brent, Christine, Derik, Misty, Zach, and Jeffery Pace