February 24, 2021 at 3:05 pm #16297Jenny LivingstonKeymaster
I’ve talked about clinical research on multiple occasions here on the forum. Not to sound like a broken record, but I’m currently enrolled in a new clinical trial that I’m rather excited about!
Without saying too much (because I’m not sure how much I can share at this point) this is a therapy that has the potential to help many people. It is not dependent on a person’s genetic mutations which means that it could benefit everyone. Of course, this all just possibility and prospect right now, but it’s gotten me thinking…
The landscape of CF treatment has changed so much over the years. When my oldest sister was diagnosed, digestive enzymes were the only CF specific medication available to her. When it came onto the market, Pulmozyme was a huge breakthrouh! (Speaking of clinical research, my sisters were part of the trials for Pulmozyme.) I can remember when things like inhaled TOBI and Cayston came out, and of course, CFTR modulators have been the most groundbreaking medications in the world of CF.
It’s crazy to think of how much things have changed in my lifetime and to imagine the progress that might still be made. What are some treatment breakthroughs that have happened in your lifetime? How much have treatment options changed for you through the years?
February 26, 2021 at 12:21 pm #16307RustyParticipant
Where do I begin? As a 64 year old person with CF I have seen many treatment options change, some good and some bad. The bad would be many of the antibiotics that used to be effective no longer are. The good, indeed the best, have been the CFTR modulators. I used to work in a major medical college hospital that is also a CFF center and as such I was involved in several clinical trials. I had the second gene transplant in the US which “cured” a small portion of my nasal passage. I was slated to be first but some sinus issues precluded that from happening as the symptoms could mask a potential side effect from the modified cold virus that was being used as the vector for the normal genes being transplanted. Unfortunately the gene transplants did not pan out but maybe somewhere down the road? I was also in all three phases of clinical trials for Kalydeco. It was all double blind but in very short order I knew beyond a doubt that I was receiving the real drug. I thought Kalydeco was the ultimate breakthrough and that I would never see anything better. Then along comes Trikafta and I am still amazed at how much improvement that has provided over and above Kalydeco. Trikafta got early FDA approval but I was still enrolled in clinical trials for it as they wanted to gather more data and actually complete the trials but covid put an end to that.
Almost forgot; the way the IV antibiotics are administered has changed for the better as well. They used to use short IV catheters in the arm or hand that had a tendency to infiltrate rather quickly. If you had one that lasted three of four days before it had to be pulled and a new one inserted you felt very lucky.
Now with the longer cath’s the many painful pokes are a thing of the past. Also, I can have a pump at home and administer my own antibiotics without having to be an inpatient. A vast improvement!
I’m sure I could think of more but that is enough history for today.
February 26, 2021 at 1:43 pm #16308RustyParticipant
Oops, a couple more changes/improvements. The use of Cayston every other month is a boon. No more painful artery sticks to get a blood gas – just put out your finger for a quick pulse oximetry. No more being paired with another CF patient when hospitalized. Getting a flutter valve to help with airway clearance. (I have never used the vest but that is also a change for the better). I am done now. I guess.
March 1, 2021 at 2:44 am #16313Paul met DebbieParticipant
As a small encore, I would like to mention the discovery of the cftr gene in 1989. Many of the modern treatments are based on this, and many future developments will be. And it also made it possible to scan for carriers and for new born patients.
July 1, 2021 at 7:40 pm #17129Tim BlowfieldParticipant
Ah! So much wonderful progress! And rethinking of the issues! Discovery of the CFTR gene was a game changer as is the use of Creon and most recently the Potentiators/Correctors.
What next? Do we continue to concentrate on CF as a problem of mucous and mucous membrane containing organs?
It is now apparent that the faulty CFTR gene affects other cells and organs. Heart disease, Adrenal issues, osteoporosis and other issues occur in organs where mucous are not produced as is also with sweat glands. Urgent research is needed here especially in PWCF who have had lung transplants and those who receive gene therapy targeting the respiratory epithelium.
July 25, 2021 at 9:35 pm #17220Tim BlowfieldParticipant
Much research and rethinking continues to be needed. Why are so many PWCF on Trikafta (T) having headaches, insomnia and other neuro signs?
I suspect this is because T is affecting electrolyte levels in the brain cells. Our researchers need to look closely into this and not just dismiss it as a ‘side effect’. Also the effect of T on other cells – heart, Adrenals & so on.
We need clinicians and researchers looking at CF with out the blinkers that it is ‘a respiratory disease’. We need much lateral thinking, Vertex not excluded.
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