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

Telavancin Promising Potential Treatment Option for MRSA in Cystic Fibrosis Patients

By Kristi Rosa

Responsible for several issues ranging from skin infections and sepsis to pneumonia and bloodstream infections, methicillin-resistant Staphylococcus aureus continues to plague patients in the health care and community setting, as well as the providers who treat them.

When acquired in patients with cystic fibrosis, clinical outcomes are known to be even worse, affecting several organs—primarily the lungs—and resulting in an increased rate of declined respiratory function as well as infections that can have severe, and sometimes deadly, consequences.

Now, however, for the first time, investigators have found that telavancin—a drug that is currently used to treat skin infections and hospital-acquired pneumonia—has potent in vitro activity and low resistance development potential when used against S aureus isolates in patients with cystic fibrosis, making it a promising potential treatment option for this population.

“Telavancin (TLV) is a lipoglycopeptide antibiotic approved by the US Food and Drug Administration in 2009 for the treatment of complicated skin and skin structure infections and in 2013 for the treatment of cases of nosocomial pneumonia, however its application for the treatment of CF-MRSA pneumonia infections was not known, so our studies are contributing to extending the application of TLV for CF treatment,” Adriana E. Rosato, PhD, associate professor in the department of Pathology and Genomic Medicine at Houston Methodist Research Institute told Contagion®. “We were also inspired by the fact that CF patients have a short life time—until 40 to 50 [years]—so our priority is to contribute to better treatment in this patient population.”

Dr. Rosato and her team hypothesized that TLV might be a promising treatment option for CF-patient-derived MRSA and MSSA infections, as in vitro studies have shown that TLV has activity against MRSA.

To prove this, the investigators screened a total of 333 strains of CF patient-derived S aureus of the wild-type or small-colony-variant phenotype, collected from both adults and children at 3 different cystic fibrosis centers: Houston Methodist Research Institute, UW Health and the Center for Global Infectious Disease Research. TLV was found to display activity against all 333 strains collected.

When testing the activity of the drug against 23 MRSA strains, the investigators observed intermediate resistance to ceftaroline (CPT)—a new beta-lactam antibiotic that targets PBP 2a in MRSA—in 20 of the strains, and high-level resistance to CPT in 3 of the strains. The authors note that although high levels of resistance to CPT is rare, intermediate resistance is more common in patients who have chronic infections.

“Among all strains, the TLV MIC90 was 0.06 mg/liter, i.e. 8-fold lower than the daptomycin (DAP) and CPT MIC90 and 25-fold lower than the linezolid (LZD) and vancomycin (VAN) MIC90,” the authors write.

Using time-kill experiments, the investigators assessed the in vitro effectiveness of TLV compared with DAP, VAN, and CPT. They found that TLV showed activity against all tested strains and displayed rapid bactericidal activity as well. The activity profile for the drug at a free serum concentration of 8 mg/liter showed that TLV performed better than VAN (16 mg/liter), LZD (10.4 mg/liter), and CPT (16 mg/liter).

The investigators also set out to determine the fate of mutation selection that could be projected by the potential prolonged use of TLV in patients with cystic fibrosis. To do this they looked at 3 specific strains: AMT 0114-48, WIS 664, and TMH 5007. They found that due to the ease of mutation selection which had been noted in control strains, TLV mutant resistance is independent of the CF patient background of the strains.

“We demonstrated that TLV has bactericidal activity against the S aureus strains tested, including those against which CPT and LZD displayed reduced activity, which might provide TLV a significant advantage over the drugs currently used to eradicate those strains and prevent future exacerbations,” the study authors write.

A clinical trial is currently underway to assess the pharmacokinetic profile of TLV in patients with cystic fibrosis, who usually need dose adjustment because of an increase in the volume of distribution and clearance.

“[The next step for our research is] to perform in-vivo analyses studies that could lead to translational application/clinical trial,” Dr. Rosato added. “However, we are limited in research funds to continue our investigations.”

Original article here.

Monitoring Pulmonary Exacerbation in Cystic Fibrosis: The Hunt for Urine-based Biomarkers Begins

By Michele Wilson PhD

The buildup of mucus in the lungs is an ongoing challenge faced by people with cystic fibrosis, and knowing whether they should seek medical attention is not always clear.

Recently, Mologic – a developer of personalized diagnostics – have developed a tool which they hope will help guide people with cystic fibrosis so they can avoid unnecessary stays in hospital.

The app-embedded algorithm converts data collected from a urinary test to a traffic light result, which indicates whether a patient is stable or in need of medical intervention.

Recently, Mologic, announced that they are launching a clinical trial to assess the company’s urine-based diagnostic tool, ‘HeadsUp’.

To learn more about how this point-of-care diagnostic tool could help improve healthcare for people with cystic fibrosis, we spoke with Gita Parekh, Head of R&D at Mologic.

How do you define pulmonary exacerbation, and why is it important that it is monitored in people with cystic fibrosis? Continue reading Monitoring Pulmonary Exacerbation in Cystic Fibrosis: The Hunt for Urine-based Biomarkers Begins

Patients Will Test Digital Platform to Manage CF-Related Diabetes (CFRD)

By Janet Stewart

Attain Health will partner with DarioHealth, a digital health and big data solutions company, to test its Dario Engage platform to monitor blood sugar levels in cystic fibrosis (CF) patients with CF-related diabetes (CFRD). Attain Health provides integrative health coaching for CF patients.

CFRD is an unusual form of diabetes estimated to affect some 30,000 CF patients in the United States and 70,000 worldwide. Experts say that the hyperglycemia (high blood sugar levels) seen in CFRD patients results in higher rates of bacterial lung infections, and an increased risk of death.

“Effective diabetes management in cystic fibrosis patients is critical, as there is a sixfold increase in mortality among cystic fibrosis patients who have diabetes as compared to those who don’t. The increased risk of mortality from lung infections is correlated with hyperglycemic events,” Kat Quinn Porco, founder of Attain Health, said in a press release.

The three-month pilot study will include real-time tracking of 12 patients with CFRD using the Dario Engage digital platform, which includes a blood sugar monitoring device that transmits readings to the clinic. Attain Health will pay for access to the Dario Engage Dashboard to monitor participants.

The app is designed to help clinics detect blood sugar trends that could lead to disease progression. Disease management is also expected to be improved by DarioHealth’s ability to provide health education content via the app.

“We are very excited to move forward with DarioHealth in exploring the benefits of digital health solutions for patients living with cystic fibrosis. We chose to work with DarioHealth because of their platform’s patient-centric approach, ease of use, real-time actionable data, and their very favorable reputation in the diabetes market,” Porco said.

The companies plan to jointly present preliminary findings at the North American Cystic Fibrosis Conference Oct.18-20 in Denver. Final results are expected in December.

Attain Health plans to obtain grant money for continuous use of the DarioEngage program with up to 200 patients a year.

“By piloting this study in partnership with Attain Health, DarioHealth is taking a leadership position in addressing CFRD by deploying what we believe are the best digital health management tools on the market today. This agreement and pilot study mark DarioHealth’s foray into chronic disease treatment markets that overlap with and expand beyond the treatment of diabetes, our company’s first treatment indication,” said Erez Raphael, president and CEO of DarioHealth.

Original article here.

Pushing Through the Fear – Guest Blog By Andrea Eisenman

By Andrea Eisenman

So many fears, where do I begin. Let’s start with my impending trip to Seattle from NYC. I like to travel but it gets complicated. How much room in my suitcase do I have to pack my myriad of machines and meds? And how much will I forget, despite my thorough list? I learned I had to put obvious things on my list like a hairbrush after I forgot that a few times. But when it is easily purchased at a drug store, no biggy. When it is my immune suppressants or a nebulizer, that is harder to replace.

I now have a lot more machinery to tote around when I leave home. I have my CPAP, my percussor and my inhalation machine and a facial steamer for my sinuses, plus my Neti pot for nasal lavage. These things become cumbersome and traveling light is not an option, I have to check my bag. So, planning is key for several days prior to take off. I am in that phase now. Packing it all. I bring enough meds for twice my travel time. My last trip to Seattle happened during 9/11. I could not fly home for a week. Luckily, I had an extra 10 days of medications to cover me.

My dad asked if I was up to the flight, it is a longer one than I have taken in many years. My answer is, I don’t know. I am fearful as I know I have lymphedema and even though I wear compression tights when I fly, it is less than comfy and I will swell in my upper body. I do have a compression machine for upper body swelling but it is way too big to bring. Will I be ok not using it for a few days? I am hoping the answer is yes. But because I do not know these things for certain, I have anxiety. And I worry I might get sick either from the flight or anytime during my trip. I do wear a mask in flight and try to stay as hydrated as possible in order to keep well. And of course, I will wipe down the area near my seat with cleaning wipes.

But in order to live a life, I have to take some risks. I had wanted to go to Seattle for a few years. It is therapeutic to get away once in a while and I had not traveled too far from home while my mom was alive. I wanted to be near enough if she needed me. I no longer have that worry. And maybe I used that as an excuse so I am now pushing myself to go on this trip. I know I can be resourceful and my doctors are only a phone call away if I get sick. There is a great CF center there and my friend is sensitive to my CF needs. When we were in college together she gave my CPT when I let her.

I find that when I push myself beyond my fears, I feel triumphant and am happy that I conquered them. Sometimes one has to get out of their comfort zone, even if it means wearing horribly tight pantyhose for six hours on a flight! I know it will be worth it and I can bond with my friend. I will feel like I accomplished something worthwhile. Maybe my next trip will be to Europe.

Cystic Fibrosis Podcast 195 Summiting Mount Everest and Denali with Nick Talbot

In the latest CF Podcast, Nick Talbot shares his incredibly unique journey with cystic fibrosis. From being diagnosed at the age of 13 to his latest climb – a trek up Denali, the highest point in North America – he constantly challenges himself to reach for his dreams. Nick stresses the importance of pushing one’s own personal limits and never being scared to fail – because failure simply means you tried.
Tune in to learn more about Nick and his cystic fibrosis story.

This video podcast was made possible through an unrestricted educational grant from Novartis to the Boomer Esiason Foundation.

Airway Clearance Vests Fail to Show Measurable Short-term Lung Benefits in Study

A note from CF Roundtable: Please do not stop using your Vest or other HFCWO device because of this impractical study. There are important differences in this study that make it not applicable to CF and therefore, not meaningful for us. First, healthy volunteers without CF were enrolled. Too many adults with CF have experienced significant benefits with these HFCWO devices, not to mention the preventive benefits. To imply these devices worsen lung function when used by a healthy nonCF person vs one with CF – with the usual accompanying inflammation, mucus +/- bronchiectasis, etc – is not practical. Second, these healthy subjects tested all 4 HFCWO devices in one day. Even when I repeat spirometry in one day, my lung function numbers most often decline over time, even with rest periods in between. The researchers tested lung function with a rest period of 15 minutes on these healthy individuals after use of the HFCWO device then moved on to the next device. Very impractical and again – not applicable to use by individuals with CF.

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By Iqra Mumal

A clinical study into high-frequency chest wall oscillation vests — assessing their short-term impact on standard measures of lung function before and during use — challenges the view that these devices work through airflow bias in the lungs, the process responsible for mucus movement when breathing.

Findings, using established tests that include forced vital capacity (FVC), forced expiratory volume (FEV1), and forced expiratory flow (FEF25%-75%), suggest “that the concept of HFCWO vest-induced cephalad airflow bias is not supported by standard spirometry measurements,” researchers concluded. “None of the vest groups showed statistically significant increased airflow in the lungs.” Continue reading Airway Clearance Vests Fail to Show Measurable Short-term Lung Benefits in Study

Machine learning to help cystic fibrosis decision-making

By James Hayes

New research claims to have demonstrated that machine learning techniques can predict with a 35% improvement in accuracy – in comparison to existing statistical methods – whether a cystic fibrosis patient should be referred for a lung transplant.

The research, led by Professor Mihaela van der Schaar of the Alan Turing Institute at the University of Oxford, has been generated through a partnership between The Alan Turing Institute and charity the Cystic Fibrosis Trust. Continue reading Machine learning to help cystic fibrosis decision-making