Anaerobic bacteria cultured from CF airways correlate to milder disease-a multisite study

Anaerobic and aerobic bacteria were quantitated in respiratory samples across three cystic fibrosis (CF) centres using extended culture methods. Subjects, ages 1–69 years, who were clinically stable provided sputum (n=200) or bronchoalveolar lavage (n=55). Eighteen anaerobic and 39 aerobic genera were cultured from 59% and 95% of samples, respectively; 16/57 genera had a ≥5% prevalence across centres. Analyses of microbial communities using co-occurrence networks in sputum samples showed groupings of oral, including anaerobic, bacteria whereas typical CF pathogens formed distinct entities. Pseudomonas was associated with worse nutrition and F508del genotype, whereas anaerobe prevalence was positively associated with pancreatic sufficiency, better nutrition and better lung function. A higher ratio of total anaerobe/total aerobe colony forming units was associated with pancreatic sufficiency and better nutrition. Subjects grouped by factor analysis who had relative dominance of anaerobes over aerobes had milder disease compared to a Pseudomonas-dominated group with similar proportions of subjects being homozygous for F508del. In summary, anaerobic bacteria occurred at an early age. In sputum producing subjects anaerobic bacteria were associated with milder disease suggesting that targeted eradication of anaerobes may not be warranted in sputum producing CF subjects.

Full article here.

£11 million-a-year US pharmaceutical boss is accused of trying to ‘blackmail’ Theresa May into buying cystic fibrosis drug

By Stephen Adams and Glen Owen

The boss of a US pharmaceutical giant was last night accused of trying to ‘blackmail’ Theresa May into buying a groundbreaking drug at an eye-watering annual cost of £100,000 per patient.

Thousands of people with the deadly lung condition cystic fibrosis (CF) have been pleading with NHS bosses to strike a deal with Vertex over Orkambi, a new treatment that could extend their lives by years.

The talks collapsed acrimoniously ten days ago, after which Vertex boss Dr. Jeff Leiden wrote to the Prime Minister threatening to scale back the firm’s investment in the UK unless NHS England agreed to buy Orkambi on its terms.

In an extraordinary outburst, Dr. Leiden accused Britain of placing ‘a lower value on the life of a CF patient than other countries’ which have purchased Orkambi, and warned that British jobs relied on his benevolence.
The row comes amid growing anger among health chiefs about how the ‘profiteering’ of drugs companies is heaping pressure on the NHS budget.

The starting price for Orkambi negotiations is thought to be £104,000 per patient per year.

Tory MP Bob Stewart said: ‘Drug companies have every right to turn a profit to fund their vital research and development work, but it is completely immoral to overcharge for a life-saving drug. If the CEO is trying to blackmail the Prime Minister that is utterly disgraceful.’

In his letter to Mrs. May, Dr. Leiden, who received £11 million in salary and stock options last year, wrote: ‘Vertex is heavily committed to the UK with its international HQ in London, an R&D site near Oxford and runs many clinical trials in the NHS. Since 2006, Vertex has invested over £1.2 billion in the UK and now employs 250 people, of whom over 110 are scientists.’

But he added: ‘We are questioning this ecosystem as [the UK] is unable to value life-changing medicines for the patients that need them.’ Without major changes to how new drugs were assessed, he pointedly warned that ‘any future biotech investment in the UK is at significant risk’.

In 2014, Dr. Leiden, who owns a string of waterfront homes in Massachusetts and Florida, faced a shareholder revolt over his ‘exorbitant’ pay package of almost £35 million. Orkambi is the only current therapy that slows the inevitable progression of CF. It could benefit up to half of the 10,500 Britons with the condition.

CF causes lungs to produce too much mucus, reducing their effectiveness. Around half of sufferers die before the age of 47, but Orkambi has been shown to boost lung function by 42 percent.

Catherine Upstone, from Brackley, Northamptonshire, whose daughter Cerys, 14, is one of those who could benefit from Orkambi, said: ‘The two sides need to get back around the table.’

But NHS England boss Simon Stevens recently told MPs that Vertex must first drop its price.

‘Right now, the company is a very long way from acting responsibly,’ he said. ‘The implication of the kinds of prices that are being talked about would either be to deny patients those drugs or to rip off British taxpayers, and neither of those situations is acceptable.’

Vertex’s chief commercial officer Stuart Arbuckle said the firm had made NHS England ‘the best offer in the world, which provides all eligible patients immediate access to our current and future medicines. However, the NHS wants our existing medicines and those coming down the line in the next year for no extra money – this is essentially asking for our medicines for free.’

NHS England said: ‘If Vertex believes they are offering a reasonable deal, they should waive their confidentiality clause and let patients and taxpayers judge whether it is fair.’

Original article here.

Positive Data from the CARE CF 1 Clinical Study of Oral Lynovex in Cystic Fibrosis Exacerbations

NovaBiotics Ltd (“NovaBiotics”) announces that its oral therapy for cystic fibrosis (CF), Lynovex®, has met the study objectives of the CARE CF 1 clinical trial.

CARE CF 1 assessed the effects of two weeks of Lynovex treatment as an adjunct to standard of care therapy (SOCT) in CF, compared to placebo plus SOCT. This trial was designed to determine whether the inclusion of Lynovex capsules alongside SOCT lessened the clinical impact of exacerbations in adults with CF, as measured by symptom severity and levels of bacteria and inflammatory mediators in sputum and blood.  CARE CF 1 was a 6-arm study with the primary objectives of determining the optimal dose and regimen of Lynovex in patients with exacerbations of CF-associated lung disease and to further evaluate the safety and tolerability of Lynovex in exacerbating CF patients.  Continue reading Positive Data from the CARE CF 1 Clinical Study of Oral Lynovex in Cystic Fibrosis Exacerbations

Omega-3 Compound Reduces Inflammation in Cystic Fibrosis Patients in New Pilot Study

By Jennifer Prince

A marine omega-3 compound comprising a docosahexaenoic acid (DHA) sn1-monoacylglyceride (MAG-DHA) may act as an anti-inflammatory for subjects with cystic fibrosis, according to a new pilot study1 published in the journal Marine Drugs. In the study, MaxSimil (Neptune Wellness Solutions; Laval, QC, Canada) increased omega-3 red blood cell levels, helped moderate the ratio of arachidonic acid (AA) to docosahexaenoic acid, and reduced key inflammatory biomarkers in subjects with cystic fibrosis. Continue reading Omega-3 Compound Reduces Inflammation in Cystic Fibrosis Patients in New Pilot Study

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

Toothpaste ingredient may bust up cystic fibrosis biofilms

By Chris Waters and Sarina Gleason

A common antibacterial substance in toothpaste may combat life-threatening diseases such as cystic fibrosis when combined with an with an FDA-approved drug, researchers report.

Researchers have found that when triclosan, a substance that reduces or prevents bacteria from growing, combines with an antibiotic called tobramycin, it kills the cells that protect the CF bacteria, known as Pseudomonas aeruginosa, by up to 99.9 percent.

CF is a common genetic disease with one in every 2,500 to 3,500 people diagnosed with it at an early age. It results in a thick mucus in the lungs, which becomes a magnet for bacteria.

These bacteria are notoriously difficult to kill because a slimy barrier known as a biofilm, which allows the disease to thrive even when treated with antibiotics, protects them.

“The problem that we’re really tackling is finding ways to kill these biofilms,” says Chris Waters, lead author of the study and a microbiology professor at Michigan State University.

According to Waters, there are many common biofilm-related infections that people get, including ear infections and swollen, painful gums caused by gingivitis. But more serious, potentially fatal diseases join the ranks of CF including endocarditis, or inflammation of the heart, as well as infections from artificial hip and pacemaker implants.

Waters and his coauthors grew 6,000 biofilms in petri dishes, added in tobramycin along with many different compounds, to see what worked better at killing the bacteria. Twenty-five potential compounds were effective, but one stood out.

“It’s well known that triclosan, when used by itself, isn’t effective at killing Pseudomonas aeruginosa,” says coauthor Alessandra Hunt, a postdoctoral associate of microbiology and molecular genetics. “But when I saw it listed as a possible compound to use with tobramycin, I was intrigued. We found triclosan was the one that worked every time.”

Triclosan has been used for more than 40 years in soaps, makeup, and other commercial products because of its antibacterial properties. Recently, the FDA ruled to limit its use in soaps and hand sanitizers due to insufficient data on its increased effectiveness and concern about overuse. Clear evidence has shown, though, that its use in toothpaste is safe and highly effective in fighting gingivitis, and it is still approved for use.

“Limiting its use is the right thing to do,” says coauthor Michael Maiden, a graduate student in medicine. “The key is to avoid creating resistance to a substance so when it’s found in numerous products, the chances of that happening increase.”

Tobramycin is currently the most widely used treatment for CF, but it typically doesn’t clear the lungs of infection, Waters says. Patients typically inhale the drug, yet still find themselves chronically infected their whole lives, eventually needing a lung transplant.

“Most transplants aren’t a viable option though for these patients and those who do have a transplant see a 50 percent failure rate within five years,” he says. “The other issue is that tobramycin can be toxic itself.” Known side effects from the drug include kidney toxicity and hearing loss.

“Our triclosan finding gives doctors another potential option and allows them to use significantly less of the tobramycin in treatment, potentially reducing its use by 100 times,” Hunt says.

Within the next year, Waters and his colleagues will begin testing the effectiveness of the combination therapy on mice with hopes of it heading to a human trial soon after since both drugs are already FDA approved.

Just brushing your teeth with toothpaste that has triclosan won’t help to treat lung infections though, Maiden says.

“We’re working to get this potential therapy approved so we can provide a new treatment option for CF patients, as well as treat other biofilm infections that are now untreatable. We think this can save lives,” he says.

The research appears in the journal Antimicrobial Agents and Chemotherapy.

The National Institutes of Health, Cystic Fibrosis Foundation, and Hunt for a Cure in Grand Rapids, Michigan funded the research.

Source: Michigan State University

Calling All Students! LMK Scholarship deadline June 30th!

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.

Nancy Wech, Lauren’s mother, established this scholarship in Lauren’s name and memory. In Nancy’s own words:

Lauren Melissa Kelly was an extraordinary woman. Laughing, gregarious, spontaneous, fun, talkative, driven, thoughtful, smart, kind and loving — all descriptive terms for Lauren, who lost her battle with CF late in her senior year at the University of Georgia. In 1992, Lauren was chosen as one of ten Senior Leaders at University of Georgia. She had distinguished herself as a member of the Golden Key Honor Society, Mortar Board, Phi Upsilon Omicron, Gamma Beta Phi, the Tate Society and Delta Gamma sorority.

Lauren felt the most significant opportunities of her college career included participation in the reconstruction and formation of organizations, which will serve the university in the future. She acted as one of the re-founding members of the Phi Kappa Literary Society and was significant in the metamorphosis of the Z Club into the William Tate Society. Her other activities included Greeks Advocating Mature Management of Alcohol (GAMMA) in which she served as Secretary and Special Events Chair. She was also a member of the Women’s Glee Club for more than two years.

In recognition of her academic performance, Lauren’s degree of Bachelor of Science in Family and Consumer Sciences was awarded posthumously. At the time of her death, Lauren was engaged to be married and living off campus in an apartment. She lived life to the fullest!

Walt Disney said, “Don’t cry because it’s over, smile because it happened. It’s not the days in life you remember, it’s the moments.” As Lauren’s mother stated, “I smile because she happened to me. Now, I want you to smile because she has happened to you.”

Please visit our website for application and full scholarship criteria and details. http://www.cfroundtable.com/community-outreach/united-states-adult-cystic-fibrosis-association-scholarship/

Send any questions to scholarships@usacfa.org.