October, 2019
Fall finally feels like it is here. We set record high temperatures for October with 94 degrees on the 1st and 93 on the 2nd. Once again we are excited to raise funds for the Cystic Fibrosis Foundation and set some “new highs” in our 42nd fundraiser!
The most exciting news we want to share with you is about the new drug, Trikafta, that has just (as of October 23) been approved by the FDA. So we are going to open our CF news this year with what has been happening in research…
Background: The CF “problem” - chloride ions can’t move freely at the cell surface so mucous is thick, causing infections, poor digestion and other symptoms. There are several ways to attack the “CF” problem. Many of them work on improving the “cystic fibrosis transmembrane conductance regulator (CFTR) gene which affects production of the CFTR protein. Let’s talk about one of these ways to attack “the problem” – the CF “modulator”. There are three basic types of CF modulators:
Potentiators act like gatekeepers. The CFTR protein is shaped like a tunnel or channel. Potentiators help keep the “gate” to the CFTR protein channel open longer so chloride can flow through.
Correctors are like construction engineers. Correctors help the CFTR protein (tunnel) form the right shape so chloride can make it to the cell surface.
Amplifiers act like the amplifier in your sound system. Sound system amplifiers take the signal from your MP3 player or CD player (probably a few thousandths of a watt) and increase it to “many” watts to a sound level you can hear. Instead of sound amplification, CFTR amplifiers aim to help the cell make more CFTR protein. Some CFTR mutations produce insufficient CFTR protein. The “Amplifiers” job would be to increase production of CFTR protein.
Using CFTR modulators, The best “fixer” would 1) Keep the “gate” for chloride ions open (Potentiator), 2) make the right CFTR “tunnel” shape (Corrector), and 3) increase the “volume” (Amplifier) of CFTR protein – all to make the mucous slippery instead of thick. Research scientists have made progress – READ ON!!
In June, 2019, a two-drug combination (containing one Corrector and one Potentiator) called “Symdeko” was approved by the FDA for children with CF (aged 6-11) with two copies of the most common mutation (F508del) or with a single copy of one of 26 specific mutations. This drug was first approved in 2018 for people with CF 12 and older. The latest approval allows an additional 2,000 children to qualify for the drug.
In October, 2019 (Yes, This Is Breaking News!), a three drug combination (containing 2 Correctors and one Potentiator) called “Trikafta” was approved by the FDA for people with CF (age 12 and older) having at least one copy of the F508del mutation. This means more than 90% of people with CF (including Brent and Derik) could have access to a drug that could improve lung function by an average of 14%! Brent participated in the trial for Trikafta.
There are some caveats that go along with these drugs:
The Drugs are not a cure for CF. They improve the function of the CFTR protein but must be taken regularly.
The drugs will not repair damage already caused by CF.
You’ll notice I defined Amplifiers but did not mention them in any of the drugs. Amplifiers are being developed and tested, but are not yet available.
Family Update:
Jim and Linda – We do not lead the retired “rocking chair” life (far from it). With grandchildren arriving on our doorstep 5 days a week at 6:30 am to our involvement with musical groups, we are constantly looking at our calendars! Linda has added into her life involvement with a non-profit called My Very Own Blanket. She makes fleece blankets that are given to kids as they enter the foster care system. Her goal this year was to make 100 blankets and she just completed her 116th. Linda got Jim involved too with some computer creations at their workshop location. We both are very involved in church activities and working with kids in several elementary schools. Linda did most of the family traveling with trips with her high school friends to Cleveland and a sisters retirement trip to Indiana. At the end of May, Jim’s stepmom, Jean (93) passed away in Florida. We made a trip to Florida in March and shared many stories of our lives shared together over the last 48 years.
Brent and Christine – They continue to enjoy life in Chicago (3 years now). They play in a shuffleboard league weekly and are working with the Chicago area Cystic Fibrosis Foundation on upcoming events. Brent’s job with BP working on renewable fuels includes trip to interesting places and working on challenging problems. Christine began working for a consulting firm over the summer as a non-profit IT specialist and is currently embedded at a large philanthropy organization in downtown Chicago. They are looking forward to a fall and winter of fireside nights and cuddles with their pup Leia. Their new rooftop deck has a spectacular view of the city! Brent’s health has been good this year with no hospitalizations. The battery vest continues to make a difference in his life and so many other CF patients.
Derik, Misty, Zach, and Jeffery – With two very active boys, Derik and Misty’s lives are busy with karate and school activities. Both boys continue to advance in their classes and enjoy their friends at karate. Jeffery has added in piano lessons, too. First grader Jeffery likes to read Dogman and Captain Underpants. Fourth grader Zach has lots of homework. Grammy likes to study his science and social studies with him (she is learning lots more than when she taught 2nd grade!). Misty continues her 3rd grade teaching in a charter school and with seniority comes more responsibility! ...and also a new hairdo! Derik bounces out of bed very early for his job as a corporate chef at the Electric Company Distribution Center. Since he finishes work in the mid afternoon, he is the karate chauffeur while Grammy and Papa do homework supervision. Last Christmas, two kittens were gifts to the boys (Midnight and Bamboo). Derik’s CF health has been good this year with few infections and no hospitalizations.
This year we are dedicating our fundraising efforts to Jean Pace (Jim’s stepmom) and also the loss of one of our supporters, Rusty Slack. Many CF patients and families have benefited from the generosity of these two people over many years. We will miss them on our team!
So now it is your turn to step up and join our efforts. So many of you have been there beside us for many years. We welcome all our “newbies” too. Together we can make a difference so continue reading below on how to donate. We would like to turn all our donations in by December 15, 2019. That day is when we began this journey in 1978...let’s fill it with hope and joy for the future. Many blessings to each of you.
Love,
Jim, Linda, Brent, Christine, Derik, Misty, Zach, and Jeffery Pace
The easiest way to donate is to Click here (or type “http://jimpace.eml.cc” 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 15, 2019