CF Annual Fundraiser for 2022 – Please donate by December 1, 2022

Join us on our “Set the PACE campaign” to raise money for Cystic Fibrosis. It is our 44th year to raise funds.  This year we are dedicating our efforts to the memory of four special friends who passed away this year:  Sharon Herlan, Debbie Dhuyvetter, Diane Whitt, and Dave Russell.  They were all past supporters of our efforts to raise funds for the Cystic Fibrosis Foundation.  

 

Pace Family Update

The Senior Paces:  Jim hasn’t played his trombone seriously for 50 years, but he made the decision to join the Westerville Community Band (Linda is a charter member, 1989).  So now we go to band together on Monday evenings.  Jim is still involved in home computer tech assistance to anyone who calls his name!  Singing with American Harmony is “on hiatus” but will begin again in November.  We both are volunteering this year in our daughter-in-law’s 3rd grade classroom and we continue  involvement with our church where we have been members for 51 years.  Linda also walks regularly (Jim is getting better at the regular routine) and does Jazzercise, too.  And don’t forget the 6am alarm that takes Linda to Blacklick to put grandson Jeffery on the bus.  

 

The Derik Paces:  Derik continues to do well on Trikafta and his overall CF health continues to be positive.  In September he spent a week at OSU Hospital for a bowel blockage (luckily surgery was not required).  This year’s challenge has been managing his CF induced diabetes.  He is now being treated by adult doctors associated with OSU, including his endocrinologist, but when he has checkups he still goes to Children’s Hospital and the doctors come to clinic.  Derik continues to work in the corporate cafeteria at Hikma Pharmaceuticals.  Misty has busy roles of mom and teacher.  She manages homework and keeps everyone on track.  Zach (12) is almost a teenager (turns 13 in December) and is involved in basketball, band, and art.  He “gets” to clean the litter boxes and that is a big job with four cats in the family. Jeffery will hit his first double digit birthday (10) in November.  He continues with piano lessons and enjoys baking.  Both boys spent a week at an overnight camp and finally (after two years of COVID) got to go to Washington DC.  Their favorite thing was the Lincoln Memorial.  This trip almost didn’t happen because Misty and Jeffery got COVID (last day in isolation was right before we left for DC)  No one else came down with it.  The Pace family is also involved with a new church in their neighborhood.  

 

The Brent Paces:  Right now they call Chicago home, but in mid November home will change, once again, to Charleston, SC.  You read that right- Brent, Christine, and dog Leia are moving back to the city where they fell in love.  They have bought their first house after living in condos.  After six years in Chicago and more cold and snow then they want to see again, they are heading south.  Work-at-home has made this move possible for both Brent and Christine. They will both continue their jobs at BP and the McArthur Foundation. Brent just finished participating in a three year study on Trikafta.  The purpose of the study was to test the long-term safety and benefits of the drug after FDA approval.  Every 3 months he had tests (blood, salt, spirometry are some) and each month had a call with the study nurse.    

 

Many of you get mailings (probably seems like almost daily at times) from the National Cystic Fibrosis Foundation.  We want to share some valuable history and facts from next year’s CF calendar:

 

 

Development of Modulators

 

CF modulators modify the defective “CF chloride channel” to work better. (Read our 2019 letter for information about how modulators work and many of our letters back to 2007 on the development of modulators)

2011 – Kalydeco (ivacaftor, also known as VX770) – 10% of people with CF were eligible

2015 – Orkambi (lumacaftor/ivacaftor) – up to 50%

2018 – Symdeko (ivacaftor/tezacaftor and ivacaftor – regimen of two different pills) – up to 60%

2019 – Trikafta (elexacaftor/tezacaftor/ivacaftor)  – up to 90%

 

An Anecdote

 

39 years ago an Asian Mom noticed her baby had granules of salt on her head after she would sweat. She received a sweat test (test to diagnose CF). The test was inconclusive but her dad remembers the technician running the test say, "Asians don't get CF".  She had continual bouts of asthma and bronchitis growing up. In college, she had frequent sinus infections. When she showed her college Student Health Clinic records to her home pediatrician, the doctor didn't believe she had chronic sinus infections because they were diagnosed by a nurse practitioner, not a doctor. She was referred to an allergist, then to an ear, nose, and throat (ENT) doctor. She had 3 sinus surgeries over 4 years and continued to have sinus infections.

 

In 2012, she visited a pulmonologist because she had a chronic, productive cough which had persisted for almost a year. She asked the pulmonologist if it might be CF. He responded that it might be something to consider. She had an x-ray, CT scan, bronchoscopy. The bronchoscopy cultured Pseudomonas aeruginosa, a bacteria common to the environment and a major cause of lung infections in people with CF.

 

A new sweat test returned a slightly positive result. The local adult cf clinic officially diagnosed her with CF. The Doctor said the first sweat test she had as a baby would have been a positive test for CF if it had happened today. Her ENT was disappointed with himself because he had let her ethnicity get in the way of him seeing the CF.

 

Her CF progressed to the point where she needed (and received) a double lung transplant in November 2018. If modulators had been available for her mutations she might  have been able to delay or eliminate the need for the lung transplants.

 

She has two rare mutations which contributed to the delayed diagnosis. These mutations prevented her eligibility for treatment with CF modulators.

 

WHY THIS PARTICULAR STORY?

 

The young Asian woman from the story was diagnosed when modulators were finally giving improved health to most people with CF. Unfortunately, she wasn’t able to take advantage of modulator treatments because of her particular mutations. In addition, her late diagnosis prevented her from taking advantage of regimens available to all CF people which could have delayed the progression of her disease.

It's a myth that only white people get CF. According to the 2020 CF registry, 3.5% of CF patients are Black, 0.5% Asian,  and 0.3% Native American. 9.6% of CF patients identify as Hispanic. The mutations in these minority groups make them less likely to be eligible for modulator therapy.

 

10% of the CF community have mutations that can't benefit from a modulator. Either they (1) aren’t eligible (modulator won’t work for their mutation(s), (2) can’t tolerate the modulators (side effects), or (3) there is not enough benefit.  The hope is future CFTR mRNA and/or CFTR gene delivery will help the  remaining 10% as well as all other people with CF. In addition, a number of products are coming to clinical trials to test the use of CFTR mRNA and CFTR gene delivery. These drugs would need to be taken regularly as the “corrected” cells in the lung died and were replaced by new cells.

In the eighties and nineties we hoped the discovery of the “CF gene” would give us a cure for CF within a decade. That didn’t happen. However, new technologies give us hope this might still be accomplished in the coming years and provide a healthier life for ALL people with CF!

 

Life Cycle of Clinical Trials

 

Development of drug treatments for medical conditions is a multi-year process. Once the treatment is developed, further study in the form of clinical trials is required before the drug can be approved by the FDA.

Phase 1 – Small, quick, focused on safety

Phase 2 – larger, longer, focused on safety and efficacy (ability to produce the desired effect), proper dosage

Phase 3 – even larger, provide proof of safety, efficacy, and exact dose needed for FDA approval

Phase 4 – what happens in real world after drug is released.

 

The startup of clinical trials using CFTR mRNA and CFTR gene therapy give us hope that further improvements in the treatment of CF will happen in coming years.

 

On November 11, Jim and Linda will attend an update on research happening in Columbus, Ohio.  We will share what we learn as we update you on our fundraising efforts.

 

Not long ago Linda met a friend she hadn’t seen for awhile in the grocery store.  The friend commented as we talked about the boys’ CF journey, that when we met the goal was just to get them to live to be teenagers.  With the support of family and friends, that goal has been “knocked out of the park” and replaced many times until now it is 50! Thanks for joining us as we “Set the Pace.”  Here is what you need to know to donate...

 

The easiest way to donate from your web browser 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 1, 2022

Love,

Jim, Linda, Brent, Christine, Derik, Misty, Zach, and Jeffery Pace