Cystic Fibrosis News Today Weekly Forum Digest

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Moderator Introduction

Sep 30, 2019 06:00 am | Jenny Livingston

Replies: 0

Hey, everyone! My name is Jenny Livingston and I’ve recently become a forum moderator here. A little bit about me? I’m 32 years old with cystic fibrosis. I also have two older sisters with CF (my family certainly did not win the genetic lottery). I live in Utah with my fiance, my 10-year-old daughter, two dogs, some bunnies, a horse, and a hedgehog.
I’ve been blogging for ten years (you can find some of my writing at lungsnroses.blog). I write about life with CF, motherhood, mental health, and more. I recently graduated with my Bachelor’s degree in Psychology, so it probably makes sense that I am especially interested in the social and mental/emotional aspects of living with CF.
I am actively involved in fundraising and advocacy efforts in a variety of ways.
I’m a firm believer in the power of human connection. I have found so much support, knowledge, and understanding in the online CF community.
I’m thrilled and honored to be here. If you have any questions, please don’t hesitate to ask me or @luisa-palazola (the senior forum moderator). I look forward to connecting with, learning from, and exchanging information with each of you!

Blocking This Protein Complex Could Help Treat P. aeruginosa Infections

Sep 30, 2019 05:06 am | Cystic Fibrosis News Moderator

Replies: 1

A study suggests that inhibiting a pro-inflammatory protein complex called the NLRP3 inflammasome can treat chronic P. aeruginosa infections in people with CF. Read more here.
What do you think of this story?

Travel Nebulizers

Sep 30, 2019 05:05 am | Luisa Palazola

Replies: 1
Traveling with CF can be a pain, especially with all the equipment we have to lug around. To make our lives a bit easier there are several small compressors available on the market and I wanted to share them with you. I have tried several of these, and will note my personal experiences if applicable.

The Pari E-Flow: This one I think was originally approved for the inhaled antibiotic Cayston, but has since expanded to various medications, including Pulmozyme and HyperSal. It’s a bit pricey, we’re looking at about $1000 and I believe you may need a prescription for it. In terms of effectiveness, it’s pretty dang quick (5-7 minute treatment times) and can be both battery operated or plug-in. But, the downfall I’ve found is that you need to immediately sterilize the handsets, or the medication residue will clog the handset and interfere with the speediness. Also, new handsets are pretty pricey as well. Insurance will cover in most cases.
The Pari Trek: I’ve had this one for years and am on the fence about it. On one hand, it’s pretty small and can be taken anywhere. However, it takes a long time for a treatment to be done (about 20 minutes) and it’s not nearly as effective as other nebulizers. It feels as though medication delivery is dimmed down quite a bit. I will say, it did get me through backpacking through Costa Rica, though. Another downfall is that you do have to sterilize your neb kits as well or bring new ones. Not my favorite.
Phillips Innospire Go: This one is PHENOMENAL. It recently just came out and it’s small, has only two connecting piece, it’s simple to clean, portable and treatments last about 6 minutes. I recently used it on my last two trips, and loved it. I’m not sure about it’s approval with medications, but I used it with HyperSal — and, man I was surprised at how well it delivered the medication. It’s a little pricey, about 200$. I found a site that had it at 180 with a prescription. Not sure if insurance would cover it, but worth a try.
DevilBliss Traveler: This one was recommended by a friend, but I have no personal experience with it. If you do, let us know in the comments below It’s a little bit more affordable, so perhaps worth a try.

What’re your CF Tattoos?

Sep 30, 2019 05:01 am | Luisa Palazola

Replies: 1

Last year I got my first real tattoo. I scheduled an appointment with an artist I had followed on IG and drove 3 hours to get a my piece done by him. After him postponing my original appointment and then kinda forgetting I was coming on our rescheduled date, a resilient best friend, and some coffee: I got my piece.
I won’t leave out the special details of accidentally (or cosmically?) meeting a Brazilian man, who felt like home, and who also helped me keep this tattoo clean that night. Sometimes, things fall apart, to make way for beautiful encounters and to unveil deep connections that have always been there

Here’s that special tattoo

View this post on Instagram

Ahhhh

A post shared by Luisa Palazola (@ladilla93) on Aug 3, 2018 at 3:39pm PDT

This topic was modified 1 week, 5 days ago by Luisa Palazola.
This topic was modified 1 week, 5 days ago by Luisa Palazola.
This topic was modified 6 days, 20 hours ago by Marta Ribeiro.
This topic was modified 6 days, 20 hours ago by Marta Ribeiro.

Anxiety associated with being denied necessary medication

Sep 30, 2019 04:59 am | Seamus Conlan

Replies: 0
My son Zack is currently on NG tube feeding in Temple Street hospital, Dublin, Ireland and will have a PEG inserted in September 2019 (this suffering would of course be unnecessary if the EMA allowed him access to Kalydeco as is the case in the US and Australia). Zack has the R117H gating gene alteration.
The European Medicines Agency (EMA) choose to deny access to Kalydeco for R117H children in Europe and treat these children with contempt. This lack of empathy and compassion for children with a chronic illness is shameful behaviour by the EMA. This assertion is based on below:
1. The R117H Kalydeco to children application was presented to the FDA back in 2014 and there was almost a unanimous decision with 13 subject matter experts voting yes and 2 voting no. Even back in 2014, the experts were able to understand that the risk of approving Kalydeco for kids is quite limited but the potential benefit is huge. Five years on and the significant amount of data that is now available on the benefit of Kalydeco to gating mutations such as R117H, the EMA still deny this life changing medicine to kids. This, despite the fact that approval is granted at 6 months in the US. It is approved at 18 years by the EMA.
2. The EMA is unique in that it believes Cystic Fibrosis behaves differently in adults and children for the R117H gene alteration even though there is no evidence to indicate this. The EMA are unable to provide a biologic plausibility whereby there would be a substantially different or opposite effect in younger kids to that experienced by adults. The EMA assertion that Kalydeco is only effective in R117H adults does not stand up. There are 2,000 + different gene alterations with Cystic Fibrosis. Why do the EMA believe that R117H is the only one that the disease is different between adults and children? There is no evidence to support this? The probability that the disease is different between adults and children is 0.0005% based on the figures above and if you take into account the testimonies of how the drug has worked for kids in the US, this probability is reduced to zero. Hence, as the EMA cannot prove that the drug behaves differently for this single alteration (as is currently the case) and there is clear evidence that the drug works for R117H children in the US, this is simply a case of age discrimination by denying access until 18 years.
3. There is clear evidence that Kalydeco works for R117H children in the US where it has been clearly demonstrated that Kalydeco benefits children when prescribed by increasing energy levels, improving digestive function and eliminating the constant stomach aches associated with CF. Children also begin to grow and gain weight when allowed access to Kalydeco with the most recent five-year study on Kalydeco showing significant improved nutritional status and weight gain. Kalydeco has tested safe for over 6 years now. The EMA choose to deny access in Europe and treat these children with contempt. This lack of empathy and compassion for children with a chronic illness is shameful behaviour by the EMA.
4. We do not get life changing medicines like Kalydeco very often and it is just beyond belief and incredulous that when we do get a drug as effective as Kalydeco for gating mutations we simultaneously get the human intervention of European regulators to block children’s access to the drug. The EMA are presiding over a situation that denies children access to a life changing medicine thus promoting unnecessary hospitalisations and suffering, permanent structural damage to the lungs and shortened life expectancies for these children.
5. The EMA are deciding that an improvement in cftr function for the G551D gating gene alteration to 48% is sufficient for Kalydeco to be approved at 6 months while at the same time saying that an improvement in cftr function for the R117H gating gene alteration to 38% is not sufficient. Do they not realise that as R117H is only 78% gating, Kalydeco cannot match the 48% cftr function of G551D as it is 100% gating? Kalydeco fixes the gating defect for both alterations in the same manner in terms of cftr function outcome. Who at the EMA is playing God by deciding that the kids with G551D can have early access to Kalydeco and live as close a normal life as possible to old age while the kids with R117H must accept shortened life expectancy and an early death sentence? The EMA are driving the development of a divide in care delivered to patients with CF, with one group of patients with gating mutations G551D having access to CFTR restorative treatments and another group R117H being denied such treatment due to their age. They should not be allowed do this.
6. It is important for the EMA to note that the ability to have to access to Kalydeco is a right to life issue. By approving the use of Kalydeco for those with the R117H mutation, you are upholding these rights. By enabling the ability to use Kalydeco for children with this mutation, before CF lung disease progresses and is outwardly visible, you are providing them with the right to live. Kalydeco has tested safe for over 6 years now. If the EMA believe Kalydeco is effective in adults, they must not deny it to children, when it can prevent their lungs from deteriorating as they age.
7. Kalydeco has the ability to reduce the Sweat Chloride levels of R117H children to a reading that is indicative of person who does not have Cystic fibrosis and every day that goes by where the EMA deny children access to this drug, the life expectancies of children are shortened as per direct relationship between sweat chloride levels and mortality. The EMA appear to accept this fact and do nothing about it.
https://www.ncbi.nlm.nih.gov/pubmed/29221674
8. There is clear evidence in the US that Kalydeco works for R117H children so why do the EMA believe it will not work in Europe until 18 years of age?
http://luckycfmom.blogspot.com/2014/10/the-kiwi-studyivacaftor-for-2-5-year.html
9. The FDA had an approval process in place back in 2014(for Kalydeco to R117H children) that was both fully transparent and allowed for a holistic approach with additional considerations for parental testimonies, CF subject matter testimonies, CF patient testimonies, in vitro data etc. The FDA noted that they were fully open to all evidence as part of the application process. Fifteen of the top subject matter experts in the US were asked to vote on the following:
Did the data support approval of ivacaftor oral tablets, 150 milligrams twice daily for the treatment of cystic fibrosis in patients age 6 and older who have the R117H mutation in the CFTR gene?
There was almost a unanimous decision here with 13 experts voting yes and 2 voting no. Even back in 2014, the experts were able to understand that the risk of approving Kalydeco for kids is quite limited but the potential benefit is huge. Five years on and the significant amount of data that is now available on the benefit of Kalydeco to gating mutations such as R117H, the EMA still deny this life changing medicine to kids. It is not good enough that the EMA do not have a holistic and transparent process and one that takes into account all available data including parental testimonies, CF subject matter testimonies, CF patient testimonies, in vitro data etc.?
In the CysticFibrosisnewstoday article “Accepting the bumps in the road” by Elizabeth Rogers, she notes that “Planning a full life around a chronic illness means accepting the bumps in the road. Learning to accept these bumps is a continuous process, but if I can do it, so can you”. This is fine when the bumps in the road are caused by environmental variables that are out of a person’s control. However, when the bumps in the road are caused by the human intervention of European Medical Regulators, this must never be tolerated. The EMA do no have the right to inflict unnecessary suffering on children with a chronic illness such as CF.

Childhood memories

Sep 30, 2019 04:24 am | Luisa Palazola

Replies: 1
Last night I was sharing my thoughts on Instagram stories, as I often do. I began reminiscing about childhood and all the things I used to innocently think. It started a dialogue with my CF friends, and we began sharing things related to how we saw CF when we were little, here are two of my absurd childhood thoughts:
1. I was diagnosed late with CF at ten years old, and for the most part — everything before that, I was relatively healthy. So, I remember when I was about 7 or 8, my younger brother had his tonsils removed, and had an overnight hospital stay. In my mind, I remember being absolutely fascinated and almost envious of his IV that he had, I thought it was so neat. I also remember him getting some neat toys in what our children’s hospital called the “Bunny Room,” which was the room right before surgery that had all sorts of toys for kids to pick from to calm their nerves.
I didn’t know that I would have my own fair share of toys from the bunny room, and plenty of experience with peripherals.
2. In fifth grade, when I was eleven, I had never had an admission. I had been diagnosed with CF and was aware of my disease, but never had an admission. So, one day, one of my classmates had been admitted to the hospital for asthma related complications. She recovered and on the day she came back to school, she brought her empty liquids bag from her IVs, for a sort of show and tell. And, I remember thinking to myself “This should be me giving this show and tell..” LOL
So, here are a few ways life has a way of coming full circle, and bringing you things that you both wanted nor never expected. Do you have any stories for your childhood? What’re they?

Professor’s Research Initiative May Lead to New Antibiotics for CF

Sep 30, 2019 03:55 am | Cystic Fibrosis News Moderator

Replies: 0
A professor of chemistry at Milligan College has launched a research initiative to encourage students to develop and test antibiotics for cystic fibrosis infections. Read more about the project here.
Had you heard of this project? What do you think of it?

I’m Breaking Up with My Clinic

Sep 30, 2019 03:52 am | Cystic Fibrosis News Moderator

Replies: 0
Click here to read Bailey Anne Vincent’s column about her experience with a not-so-great CF clinic.
Have you ever switched clinics? How did that go?

Crazy Stats

Sep 26, 2019 08:00 am | Luisa Palazola

Replies: 0
Have you seen these stats before, our team at CF News Today came up with this neat infographic of strides recorded by the Cystic Fibrosis Foundation. And, I think it’s pretty phenomenal:

For, me it’s boggling to realize that I am one of those people. I am one of those people who has not only benefitted from the newer medications and trials. BUT, the advancements across the board — from HyperSal to Pulmozyme. These are all crucial steps in getting to these numbers. It’s gotten me to be a college graduate, and later on to someone whose lung function IS above 70%, and now a woman who can have kids. How did this become my reality?
Link to infographic:

This topic was modified 4 days, 19 hours ago by Luisa Palazola.
This topic was modified 4 days, 19 hours ago by Luisa Palazola.

LCI Is Sensitive Lung Function Parameter in Patients with Mild CF, Study Says

Sep 26, 2019 04:19 am | Cystic Fibrosis News Moderator

Replies: 0
LCI is more sensitive than standard spirometry parameters (e.g., FEV1) to changes in lung function in patients with mild cystic fibrosis. Click here to read more.
What do you think of this news?