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Be Involved in a Meeting with FDA on CF!

On October 29th, individuals with cystic fibrosis and their families will have a unique and pivotal opportunity to share their experiences with representatives of the FDA during a live-streamed interactive meeting.

CFRI is very honored to host an Externally-Led Patient-Focused Drug Development Meeting on Cystic Fibrosis with the FDA on Monday, October 29th. This is an amazing and singular opportunity to share the patient experience with FDA representatives. They want and need to know the impacts and burden of the disease, your hopes for new therapies, and what you are willing to go through to find these new drugs.

PLEASE register to participate in this free live-streamed meeting! We need your input and participation You will have the opportunity to participate in live polling, and to email and call in to share your experiences. Those who do not have CF/a family member with CF should also feel free to join us.

You can register and log in for any or all of the day’s presentations and discussions. The day begins at 9:45 am. Please note: all times listed are East Coast time, as the meeting will be held at the College Park Marriott Hotel and Conference Center in Hyattsville, Maryland.

Here is the link:  http://cfri.org/advocacy/advocacy-events/

Speakers/Panelists
Jen Caruso
Lise-Courtney D’Amico
Boomer Esiason
Gunnar Esiason
Joseph Klausing, JD
Emily Kramer-Golinkoff
Robert Lim, MD
Jane Mitchell
Anna Payne
Kat Quinn Porco, MS
Tejashri Purohit-Sheth, MD
Arek Puzia, CPA, MBA
Emily Schaller
Isa Stenzel Byrnes, LCSW, MPH
Ahmet Uluer, DO, MPH
James Valentine, JD, MHS

Thank you for having an impact upon those who are assessing the safety and efficacy of new CF therapies, and making recommendations for their movement to market. 

Your voices matter!

The Cystic Fibrosis Reproductive & Sexual Health Collaborative (CFReSHC) Invitation to Participate

The Cystic Fibrosis Reproductive & Sexual Health Collaborative (CFReSHC) is a patient engagement project that provides women with CF the opportunity to be equal partners with healthcare professionals to shape the future of CF research.  CFReSHC offers women with CF the opportunity to participate on the Governance Board, Research Advisory Panel and Patient Task Force as Patient Partners to develop research projects that directly respond to the questions faced by women with CF.  CFReSHC holds monthly virtual meetings on topics that impact the lives of women with CF like contraception, family building, hormone influences on CF, incontinence and menopause.  As we enter our third year, CFReSHC is looking for women with CF who are passionate about sexual and reproductive health research to join our collaborative who can commit 4-6 hours a month and we provide  a small honorarium for your time commitment.

CFReSCH is looking for a:

  Social Media Strategist who has connections in the CF community or who is willing to engage with the CF community on our behalf.  CFReSHC currently has a social media presence on Facebook, Twitter and Instagram and the applicant would need to be able to post 2-3 times per week as well as check the platforms regularly and make responses as needed.  The applicant would need to attend 3 paid monthly meetings often held during work hours.

Grant writer and Coordinator who has connections in the CF community or is willing to connect with the CF community and engage it to seek out funding opportunities for potential grants.  The applicant would need to maintain a database of potential grant opportunities.  An applicant who has experience writing applications is preferred.  The applicant would need to attend 3 paid monthly meetings often held during work hours.

Women with CF are welcome to send a letter of interest to: cfreprohealth@gmail.com.  Together, we will respond to the health needs of the CF community by providing data for healthcare professionals to pave the way for improved sexual and reproductive health resources, healthcare, and knowledge.

Hooray! I Celebrated My Fifth Anniversary – Guest Blog by Kathy Russell

October 3rd, 2018, I celebrated five years since having bilateral mastectomies because of cancer. I have had no recurrence of those cancers and no chemo or radiation therapy. Since I am 74 years old and have CF, I feel very fortunate.

My surgeon would have done just a lumpectomy on the left side, where the initial tumor was found, but I opted for removal of both breasts. I didn’t want to have any more surgeries. If I could have had reconstruction surgery at the time of my mastectomy, I might have opted for that. However, since my surgeon and the plastic surgeon both said that was not an option, I chose to get rid of the whole possibility of future trouble by removing both breasts. Continue reading Hooray! I Celebrated My Fifth Anniversary – Guest Blog by Kathy Russell

Livestreaming of the North American Cystic Fibrosis Conference (NACFC)

North American Cystic Fibrosis Conference
Opportunity: Register Now for the Livestream Content
Dates: NACFC will be held October 18-20
The North American Cystic Fibrosis Conference (NACFC) brings together researchers and health care professionals from around the world to discuss the latest in cystic fibrosis research, care, infection, treatment, and drug development.
You can explore conference sessions along with the experts! Join us online for three livestreamed plenaries, follow-up Facebook Live Q&As with the speakers, and additional livestreamed workshops and symposia that were hand-selected by members of the CF community, including members of Community Voice. You will find the schedule for these sessions below. The plenary sessions are the major keynote sessions that are presented to all attendees. Each plenary will be followed by a Facebook live Q&A session with the plenary presenters.
During the third plenary session, keep an eye out for Community Voice Melanie Abdelnour, who will be sharing about the importance of partnering with her care team.
Additionally, you will have access to view the content from all other sessions where speakers have consented to share their presentations through the sync-to-slide feature 6 weeks after the conference. Stay tuned for more details!
Register now to livestream the plenaries, workshops, and symposia.
Thursday, October 18, 2018
  • Advancing Basic Science Toward a ‘One-Time’ CF Cure – 09:45 am – 11:05 am MT
  • Pro/Con: Current Debate in Pulmonary Treatments – 09:45 am – 11:05 am MT
  • Controversial Practices – Helpful or Harmful – 02:00 pm – 03:25 pm MT
  • Healthy Habits: Promoting Physical & Mental Health Through Sleep, Exercise, & Nutrition – 02:00 pm – 03:25 pm MT
  • Plenary 1: Improving Outcomes of Infections in the Age of CFTR Modulators – 04:30 pm – 06:00 pm MT (6:30-8 ET)
  • Facebook Live: Infection Research: Mission Possible,  6:30 pm MT (8:30 pm ET) 
Friday, October 19, 2018 
  • Plenary 2: Anti-inflammatories & Mucociliary Clearance Therapies in the Age of CFTR Modulators – 9:00 – 10:00 am MT (11:00 – 12:00 pm ET)
  • Facebook Live: Glad You Brought It Up: Mucus and Inflammation, 12:15 pm MT (2:15 pm ET)
  • The Changing Face of Pulmonary Exacerbation Treatment – 10:30 am – 11:55 am MT
  • Progress & Promise of the CFTR Modulator Pipeline – 10:30 am – 11:55 am MT
  • Novel Approaches to Modulate CFTR – 02:00 pm – 03:20 pm MT
  • Nutrition Research – 02:00 pm – 03:20 pm MT
Saturday, October 20, 2018 
  • Plenary 3: Partnering: The Oldest New Idea to Improve CF Care – 9:00 – 10:00 am MT (11:00 – 12:00 pm ET) 
  • Facebook Live: Howdy, Partner! A Live Q&A, 12:15 pm MT (2:15 pm ET)
  • CF Airway Inflammation – 10:30 am – 11:50 am MT
  • Progress in CF Pulmonary Disease – 10:30 am – 11:50 am MT
  • Pain Management in CF – 02:30 pm – 03:55 pm MT
  • ‘Adulting’ With CF – 02:30 pm – 03:55 pm MT
If you have any questions, please email communityvoice@cff.org.
Best,
The Community Voice Team

I’m Drowning – A researcher-patient’s plea for broader inclusion in cystic fibrosis trials

By: Ella Balasa

I’ve always known cystic fibrosis (CF) is a progressive disease; it destroys lung cells, tightens the small airways in the bottom of my chest, and each day takes me closer to the time when it will have ravaged my lungs. I had never really questioned if there was some way this process could be altered. I accepted that it couldn’t.

Recently, however, this has changed. The epicenter of new CF research is the development of medications that will slow, stop, and hopefully even reverse the effects and damage that CF inflicts on the body. The possibility of the cells in my lungs functioning to their full potential — with CF transmembrane conductance regulator protein function restored and working correctly, expelling chloride out of my cells, hydrating the surface of my lungs, and halting the thick sticky mucus that has caused my airways to be enveloped in a suffocating cloak for all these years — is like a feeling of being rescued when you are drowning.

Unfortunately, I am still drowning.

“I’m very sorry, Ms. Balasa, but you will not be able to be a participant in this clinical trial.” This was the response I received during one of my searches for these drug trials. Excited by the possibility of participating, finding one recruiting at my local adult clinic, I reached out to study coordinators and was informed that I met all but one criterion to participate in the studies. This specific criterion has prevented me from prior trial participation involving other investigational medications treating the symptoms of CF, including anti-infectives and anti-inflammatories.

Most CF studies, including phase I, II, and III trials, require a lung function minimum of at least 40% FEV1 (forced expiratory volume in one second). My FEV1 is 25%, so I am excluded from these trials. Many patients face a similar situation. The 40% threshold biases samples toward a young patient population, as this degenerative condition causes steadily decreasing lung function with time. Furthermore, as CF treatment has rapidly progressed and increased patients’ life expectancies, there are now more adults with CF in the U.S. than children, according to the CF Foundation Patient Registry.

As a patient who works in the science field, I started to ask myself: Where does that number come from? Should this one variable be such a deciding factor? Are we getting comprehensive results from these studies if a subset of patients is omitted? Are investigators using eligibility criteria from a prior study without determining whether the exclusions are scientifically justifiable?

To continue reading, please visit MedPage Today.

Sound Pharmaceuticals to present initial data on the STOP Ototoxicity Study at Cystic Fibrosis Conference

SEATTLESept. 25, 2018 /PRNewswire/ — Sound Pharmaceuticals (SPI) is pleased to announce that its recent submission to the upcoming North American Cystic Fibrosis Conference (NACFC) Oct. 18-20 has been selected as a late-breaking abstract. This presentation will focus on the incidence and severity of ototoxicity in CF patients undergoing intravenous (IV) tobramycin treatment for acute pulmonary exacerbation. Ototoxicity (hearing loss, tinnitus, vertigo or dizziness) is a common side effect of tobramycin and other aminoglycoside antibiotics (amikacin, gentamycin and streptomycin). Currently, there are no FDA approved therapies for the prevention or treatment of ototoxicity or any other type of sensorineural hearing loss, tinnitus, or dizziness. Continue reading Sound Pharmaceuticals to present initial data on the STOP Ototoxicity Study at Cystic Fibrosis Conference

Check out Jerry Cahill’s latest Cystic Fibrosis Podcasts

Cystic Fibrosis Podcast 196: Managing CF as a Track Athlete

In Jerry Cahill’s latest video podcast, we meet Teena Mobley – a Long Island University graduate and former track athlete. Diagnosed at 9 years old, Teena’s life changed as she adjusted to her new medication schedule while staying dedicated to fitness and health. She shares how exercise helps her manger her illness, remain positive, and continue to believe in herself.
This video podcast was made possible through an unrestricted educational grant from the Allergan Foundation to the Boomer Esiason Foundation.

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Cystic Fibrosis Podcast 197: Moving from Middle School to High School

In Jerry Cahill’s latest cystic fibrosis video podcast, he spoke with Aidan Neville – a 13-year-old with CF who started 9th grade this year. He has two siblings, 22 cousins, and many more who make up his support system. While Aidan loves to stay active – surfing, playing football, and basketball – his exercise sometimes takes a backseat to his disease.
Because he is often on IVs and has had over 24 sinus surgeries, Aidan’s biggest concern about starting high school is missing class and that possibly negatively impacting his chances to go to the college of his choice.
Tune in to learn more about Aidan and his journey from middle school to high school.
This video podcast was made possible through an unrestricted educational grant from Gilead to the Boomer Esiason Foundation.

CF Foundation asking for Public Comment on Lung Transplant Referral Guidelines

Dear CF Community,

The CF Foundation seeks your input on the draft of the Lung Transplant Referral for Individuals with Cystic Fibrosis: Cystic Fibrosis Foundation Consensus Guidelines. This guideline is one in a series of guidelines for advanced stage lung disease; the other guidelines in progress on this topic include: Advanced Lung Disease Consensus Guidelines and Post Lung Transplant Consensus Guidelines.

A committee of CF health care providers, transplant healthcare providers, and an adult with CF researched and developed the consensus recommendation statements presented in the draft. It is our hope that you will add your input and feedback to ensure that our community issues actionable practice recommendations for lung transplant referral for individuals with cystic fibrosis.

To systematically collect feedback from the CF community, we have created an on-line tool which is linked below. We recommend that you review the attached document, Lung Transplant Referral for Individuals with Cystic Fibrosis: Cystic Fibrosis Foundation Consensus Guidelines PDF, and use the link below to enter your comments.

Submit comments here: https://www.surveymonkey.com/r/LTxReferralPublicComment

The deadline for comments is Wednesday, October 10th, 2018, 5:00 PM, Eastern.

We value your input. Thank you in advance for taking time to review and provide us with feedback on this very important aspect of CF care.

If you have any questions, please contact shempstead@cff.org.

Sincerely,
Albert Faro

Albert Faro, M.D.
Senior Director of Clinical Affairs
Cystic Fibrosis Foundation | National Office

Take Our Survey. We’d love to hear from you!

Please consider taking our 5-10 minute survey so we can ensure that the CF Roundtable is the best it can be!
We value our readers’ feedback and would love to hear from you.

Two more weeks to apply for our LMK Scholarship! Due October 15th.

CF Roundtable offers the Lauren Melissa Kelly (LMK) Scholarship award each semester, in honor of Lauren Melissa Kelly. The academic scholarships of up to $2500 are awarded to adults with cystic fibrosis who are pursuing career certifications, associates, and bachelor and graduate degrees. Spring 2019 applications due on October 15th, 2018.  Continue reading Two more weeks to apply for our LMK Scholarship! Due October 15th.