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Jerry Cahill’s CF Podcast: Stem Cell Research with Dr. Hans-Willem Snoeck

In this feature of The Path Forward with CF series, Dr. Hans-Willem Snoeck, Professor of Medicine (Microbiology and Immunology) at CUMC, sits down to discuss stem cell research as it relates to CF.

Because lung cells regenerate and repair themselves regularly, researchers believe that – some day – stem cell technology could be a one-time therapy to cure cystic fibrosis. Research is ongoing, but in the meantime, scientists can currently use human pluripotent stem cells to create lung organoids (tiny, 3-D structures that mimic features of a full-sized lung), introduce various mutations, and apply technologies to learn more about those mutations’ characteristics.

This video was originally published on JerryCahill.com

Vertex Employees Donate $1M to CF and Other Communities via Matching Gift Program

By Carolina Henriques

Vertex Pharmaceuticals employees have raised more million $1 million  using  the Vertex Foundation‘s matching gift program in a show of commitment to causes that include the cystic fibrosis (CF) community, a company press release states.

The dollar-for-dollar matching gift program is being run through the nonprofit Vertex Foundation, established by the company in November 2017 as part of it’s charitable giving goal of donating $500 million to qualified nonprofits and other causes worldwide over 10 years.

To date, more than 500 Vertex employees have used the program to support 753 charities around the globe working to advance work in areas that include healthcare, human services, education, and disaster relief.

Vertex’s charitable commitment has four primary goals: supporting CF patients and caregivers worldwide, including enabling access to Vertex’s medicines; helping underserved students and young women with STEAM (science, technology, engineering, arts and math) education; supporting young doctors and scientists; and strengthening and fostering innovation in local communities through health and wellness programs.

“Giving back is in our DNA at Vertex, and our employees have a long history of going the extra mile to improve the lives of patients, students and their neighbors,” Jeffrey Leiden, president, chairman and chief executive officer of Vertex, said in the release. “I’m proud that The Vertex Foundation is able to help extend the impact of our employees’ giving and look forward to seeing the reach of these investments in the causes they care about most.”

Also as part of its 10-year commitment, Vertex awarded $400,000 in scholarships to eligible CF patients and their family members in May as part of its second “All in for CF” scholarship program. In total, 80 scholarships worth $5,000 each were awarded for the upcoming academic year.

Vertex, which specializes in cystic fibrosis, has three approved CF therapies: Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor), and Symdeko (tezacaftor/ivacaftor).

The company is also testing potential triple combination treatments for CF.

For the rest of this article, click here.

How to Beat Brain Fog

By Wendy Caroline

As I sat down to write my column for this week, a massive wave of writer’s block hit me. This has been plaguing me for a while. Great timing, right?!

I started five different column pieces, each of which I would get a solid start on, and then nothing. I stopped understanding where the piece was going and didn’t like any of the words that I was writing. A few years ago, I started developing chronic fatigue. It started slowly, then quickly snowballed. In the past year, it has developed into brain fog. Very frustrating. I love using my brain for writing, reading, problem-solving, and critical thinking. It increasingly feels like cystic fibrosis is taking all these parts of my personality away from me. So here I am today, compiling a list of some tips and tricks that I use to combat brain fog and feel a little more like myself.

1. Don’t force it.

I can’t tell you how many times I have blocked my own brain by trying to force my way through brain fog. “Just do it.” How many times have we heard that? Unfortunately, at least for me, if I “just do it” and try to force myself to work through the brain fog, it just puts up more blocks. Then I get frustrated, and all I can think about is how I can’t think. It just spirals from there.

Cool, so how do you get past brain fog and still make your deadlines (because if you’re forcing it, that’s often why)? Be ahead of the game. I am guilty of this not being a natural instinct. In fact, I’m still learning how to do this. I used to be a procrastinator. I would thrive under the stress. My brain fog does not. Now I start writing and drafting at least a week before.

2. Get inspired.

One of the best ways to get inspired is to look to others. CF News Today columnist Brad Dell’s latest piece about brain fog directly inspired this post. It got me thinking about my own experience with brain fog, and how I have been insanely frustrated by its hold on my life. Find a piece of other people’s lives or the world around you that makes you think about your own. Capture it and go. I find that making a note of these inspirations in a note-taking app on my phone really helps because brain fog causes forgetfulness.

2. Create the mood.

Ditch the phone and whatever other fancy gizmos that cause a distraction. Find the type of environment that makes you feel inspired and ready to work. For me, that’s open space with lots of natural light and structured seating.

Next, put on some music — music that is going to make you focus and productive. My go-to is this Classical Essentials playlist on shuffle. This helps me drown out enough thoughts that I won’t get distracted without drowning out the thoughts that I need to work. Whatever you listen to, just make sure it doesn’t have words.

It’s also important to get your brain in the right mood. My brain fixates on chores and tasks that need doing, so I always make sure to get those done before I sit down to write. Also yoga and meditation, that’s some great stuff.

4. Let it flow.

A friend of mine gave me this very useful tip. Just let the words flow out of you, even if you don’t like them. When I use this technique, I have to write everything my heart desires and then walk away. Don’t reread it right then. You will get frustrated. Instead, write all you can and come back to it after you’ve had a solid break. For me, this is usually the next day. Your rough work can have a really good base but just needs to be re-worked.

I know that these tips and tricks are focused on writing, but I use the general ideas in all aspects of my life. Whether it is work, family, friends, or hobbies, brain fog can affect every aspect of your life. It’s one of the reasons that I haven’t been very active on any of my social media platforms — Instagram, YouTube, Facebook, and even my blog. As my brain fog developed into almost a daily issue, I took a step back and prioritized what I needed to focus on first. I truly believe that as these steps of beating brain fog become a habit, I will be able to handle more. Hopefully, you’ll see me around the internet again real soon.

» Find me on the internet at The Living, Breathing Wendy «

This article was originally published on CF News Today.

This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition.

Jerry Cahill’s CF Podcast: The Pre-Transplant Process with Dr. Emily DiMango

The latest video in The Path Forward with Cystic Fibrosis series, Dr. Emily DiMango, Director of the Gunnar Esiason Adult CF Program at Columbia University Medical Center, discusses the lung transplant process through the lens of a CF doctor.

First, she reviews the importance of CF patients participating in drug trials in order to start life-changing medications sooner. She then answers the following questions:

· What does pre-transplant management look like for a CF patient?
· When is the right time to be referred to the list?
· What is the referral process like?

Finally, she reiterates the importance of well-rounded treatment that includes physical health, nutritional health, and emotional health.

This video was originally posted on JerryCahill.com

Cystic Fibrosis Disease Severity Linked to Immune Overreaction to Fungus, Study Reports

By Ana Pena

Disease severity in cystic fibrosis (CF) may be associated with an overreaction of the immune system to the fungus Aspergillus fumigatus, particularly due to a type of white blood cell called a phagocyte — which ingests and kills invading organisms — a study suggests.

U.K. researchers found that phagocytes from CF patients release higher amounts of harmful reactive oxygen species in response to Aspergillus fumigatus, a common cause of lung infection in these patients.

The study, “Aspergillus-induced superoxide production by cystic fibrosis phagocytes is associated with disease severity,” was published in the journal ERC Open Research.

Recent studies have supported the idea that the widespread environmental fungus Aspergillus fumigatus may play a critical role in CF lung disease.

Up to 58% of CF patients are colonized with this fungus, and an estimated 47.7% of adult patients are affected by either allergic reactions or infection caused by the fungus.

Persistent infections with A. fumigatus are also known to be adversely correlated with lung function and hospitalization in CF patients.

Researchers hypothesized that the anti-fungal defense mechanism in CF patients might be altered and have an impact on the progression of lung disease.

To investigate this hypothesis, the team compared the immune response of phagocytes from CF patients with those of healthy individuals used as controls, and tried to correlate them to clinical metrics of disease severity.

For original article please visit CF News Today.

Cystic Fibrosis Awareness: Sharing A Story & The Facts

Cystic fibrosis is a devastating disease. About 30,000 children and adults have cystic fibrosis (CF) in The United States. While the life expectancy for someone with CF has doubled in the last 30 years, children and adults people still lose their lives to this disease every day. There is no cure yet. It’s time to find it.

How can you help? Use your voice and get involved! Participate in Great Strides walks and other events supporting CF and share the facts on social media and in your community. The Cystic Fibrosis Foundation is working hard to find a cure. Other foundations and organizations help the CF community as well. Like the CF Roundtable which supports adults with CF, there is a foundation that supports children and families living with CF: Claire’s Place Foundation.

Claire’s Place Foundation, founded by Claire Wineland, when she was just 14 years old, is a 501c3 non-profit organization designed to help children and families affected by cystic fibrosis. Claire was born with CF. In her short life, Claire has undergone more than 20 surgeries and has spent months at a time in the hospital.  She survived months in a coma on mechanical ventilation despite the odds and was also a Make-a-Wish recipient. She has 40% of ideal lung function and the doctors have estimated two years left to live. This is not, however, what characterizes or defines her. Claire has dedicated her life work to making a difference in the lives of others living with CF.

Now 21 years old as an adult living with CF, Claire’s Place Foundation is a way for Claire to give back with hope, strength, and joy.  She wants to live with intention and purpose. In her own words, “I know my life will be shorter so every day has meaning.”  She strives to find new ways to help the CF community and other children afflicted with chronic illness. Claire’s Place is making a huge impact through its two programs – The Extended Hospital Stay Fund and the Family Support Program.  These programs are in high demand and the foundation needs your donations to continue assisting young patients in need. You can meet Claire and support her foundation at her next fundraiser, “Glow Ride,” on August 18, 2018 in Hermosa Beach, CA.

To find others ways you can help bring awareness to CF find a local chapter near you.

Get involved. Donate. Come to events.

Ex Vivo Lung Perfusion for Transplant

Cystic Fibrosis Podcast 186:
In the latest edition of The Path Forward with Cystic Fibrosis, Dr. Frank D’Ovidio – the Surgical Director of the Lung Transplant Project and Director of the Ex Vivo Lung Perfusion Program at CUMC – explains exactly what the Ex Vivo program is and what its end goals are.
Because so many donor lungs are damaged at the time of death, only 20-30% of donated lungs are usable for transplantation. The ex vivo lung perfusion (EVLP) is a process of evaluating and preparing donor lungs outside the body prior to transplant surgery. In EVLP, the lungs are warmed to normal body temperature, flushed of donor blood, inflammatory cells and potentially harmful biologic factors, and treated with antibiotics and anti-inflammatory agents.
Eventually, as this process is perfected, it could expand the available donor pool by restoring and repairing donor lungs that have sustained damage and eventually create a sort of ‘ICU for organs.’

This video podcast was made possible through an unrestricted educational grant from Columbia University Medial Center and the Lung Transplant Project.

How registries can be used to advance our understanding of the CF population

By Abaigeal Jackson and Christopher Goss

Patient or disease registries were first used in monitoring health over 60 years ago [1]. For rare diseases like cystic fibrosis (CF), these early efforts were instrumental in defining the population with the disease and tracking their clinical outcomes.

People living with rare diseases today face particular challenges including treatment availability, a lack of resources, and disease severity. Special efforts are required to undertake research for better treatments and possible cures [[2], [3]]. With CF, set standards in specialised care [4] allow high-quality care to be delivered to patients through networks of specialist or accredited CF centres in many countries. CF registries work by bringing patient data together from specialist CF centres and accumulating data on a relatively rare condition over patients’ lifetimes. The strength of CF registry studies therefore originates from the large number of subjects studied, high rates of coverage by the patient registries and lengthy patient follow-up [5].

One of the more comprehensive CF patient registries is the US CF Foundation Patient Registry (CFFPR). In 1966, the CFFPR was established to study the natural history of the disease and now contains detailed data on >48,000 individuals with CF [5]. Using national birth and death data, the CFFPR estimates that it holds information on 81–84% of all people with CF in the US. The CFFPR has been used to evaluate survival and temporal changes in survival, predictors of survival, and many other clinical studies in CF [[6], [7]]. Similar advances have come from other registries. Data from the European CF Society Patient Registry (ECFSPR) was used to project the number of European CF patients in 2025 [8], and provide knowledge about differences in average year to year lung function decline among CFTR mutation classes [[8], [9]].

In this review, we address key aspects of CF registries including 1) the origin of national CF registries, 2) the use of registries for monitoring the health of the population, 3) the power of conducting longitudinal analysis of registry data, 4) knowledge gleaned from changing demographics, 5) the evolving area of international comparisons of registries, 6) strategies for sustaining registries and 7) CF registries of the future. CF patient registries have both strengths and weaknesses. With a rare disease like CF, the strengths and opportunities for advancing CF science clearly outweigh the weaknesses inherent to observational data.

To continue reading please click here.

Call for Articles Published in CF Roundtable! Due June 15th

When disaster strikes, what is important to you??

What are the things that you know you must have with you in the event of a disaster, either natural or manmade? Where do you keep these essential items? What plans have you made for surviving when basic services are interrupted? It could be even as small of a disaster as when your air conditioning goes out in the heat of summer? How do you manage?

Please consider contributing to CF Roundtable by sharing some of the experiences of your life. In addition to the focus topic, we welcome humorous stories, articles on basic life experiences, short stories, artwork, cartoons, and poetry. We require that all submissions be original and unpublished.

With your submission, please include a recent photo of yourself as well as your name, address, and telephone number. Photos will be returned. Or email a high-resolution JPEG along with your article. Type or hand-print your submission, using a plain font, not script, and please double-space your article.

Submissions due June 15, 2018.

Mail To: CF Roundtable, PO Box 1618, Gresham, OR 97030-0519

Email To: cfroundtable@usacfa.org

Antioxidant Supplement Helps Cystic Fibrosis Patients, Study Finds

By Carolina Henriques

A special formulation of an antioxidant-enriched multivitamin helped decrease the frequency of pulmonary exacerbations in patients with cystic fibrosis (CF), according to a new study.

The findings, by researchers at Children’s Hospital Colorado and the University of Colorado School of Medicine, were published in the American Journal of Respiratory and Critical Care Medicine under the title “Effects of an Antioxidant-enriched Multivitamin in Cystic Fibrosis: Randomized, Controlled, Multicenter Trial.”

Inflammation is an important contributor to lung damage and to progressive lung function decline in CF. In the study, researchers looked at the effects of a “cocktail” of multiple antioxidants on inflammation and health outcomes in CF patients.

“Single oral antioxidant formulations have been tested previously in CF with mixed results. However, there had not been a well-designed, randomized controlled trial of an antioxidant ‘cocktail’ that included multiple antioxidants in a single formulation,” Scott Sagel, MD, PhD, said in a University of Colorado news story. Sagel is the study’s first author and a pediatric pulmonologist at Children’s Colorado.

The 16-week study (NCT01859390) was conducted from September 2013 to October 2015 at 15 U.S. CF centers affiliated with the CF Foundation’s Therapeutics Development Network.

The study included 73 pancreatic-insufficient CF patients, 10 years and older (average age 22), who could not adequately absorb important dietary antioxidants such as beta-carotene, coenzyme Q10 (CoQ10), tocopherols (vitamin E), and selenium, which help neutralize inflammation in the body.

Participants received either capsules of antioxidant-enriched multivitamins, or control multivitamins without antioxidant enrichment. The capsules were designed specifically for people with difficulties in absorbing fats and proteins, like CF patients.

The investigational antioxidant-enriched multivitamin supplement, called AquADEKs-2, contains standard amounts of fat-soluble vitamins (A, D, E, K) plus several antioxidants, including beta-carotene, mixed tocopherols, CoQ10, mixed carotenoids (lutein, lycopene and zeaxanthin), and the minerals zinc and selenium.

Sagel and his team found that the antioxidant-enriched multivitamin supplement increased the concentration of antioxidants in the bloodstream, and temporarily reduced inflammation at four weeks, even though these results were not sustained through the end of the 16 weeks of the study.

In addition, antioxidant supplementation was found safe and well-tolerated by study participants.

Researchers also observed that antioxidant treatment appeared to prolong the time to the first pulmonary exacerbation requiring antibiotics, and also to reduce the frequency of pulmonary exacerbations altogether.

For the full article, please visit CF News Today.