Study Links CF Patients’ Airway Bacteria with Disease Outcomes

By: Diogo Pinto

Researchers have linked variations in the mix of microorganisms in cystic fibrosis patients’ airways to their disease outcomes.

The findings in the journal PLOS One were in an article titled “Fluctuations in airway bacterial communities associated with clinical states and disease stages in cystic fibrosis.

CF patients typically have particular strains of bacterial and fungus in their airways. The usual bacteria suspects include PseudomonasAchromobacterBurkholderiaHaemophilusStaphylococcus, and Stenotrophomonas.

Other bacteria and fungi also inhabit CF patients’ airways, however. These include anaerobic species that do not need oxygen to grow and spread.

Not only do the microbial communities in CF patients’ airways vary by type of microorganism, but also in the relative abundance of each species.

Researchers decide to see if the prevalence and relative abundance of typical CF pathogens and anaerobic microorganisms play a role in the severity of patients’ disease and their lung function.

They analyzed 631 sputum samples collected over 10 years from 111 patients.

The team classified the stage of patients’ disease on the basis of their lung function scores. The yardstick they used was forced expiratory volume in one second, or FEV1. They considered an early stage of the disease to be an FEV1 score higher than 70, an intermediate stage a score of 40 to 70, and an advanced stage a score lower than 40.

Researchers classified disease aggressiveness — mild, moderate or severe — on the basis of change in FEV1 relative to age.

They discovered a link between variations in the prevalance of the six typical CF pathogens, plus nine anaerobic species, and changes in a patient’s disease stage and lung function.

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Cystic Fibrosis Podcast 180: The Pre-Transplant Process

In Jerry Cahill’s latest podcast series, The Path Forward with Cystic Fibrosis, we hear from Dr. Selim Arcasoy from Columbia University Medical Center. He discusses the pre-transplant process by covering the following topics:
  • When should a CF patient consider a lung transplant?
    • When lung function decreases to 30% or below
    • When there is an increased infection resistance
    • When exacerbations resulting in ICU hospital stays become frequent
    • When a patient experiences frequent lung bleeds and collapse
  • What is the transplant listing process?
  • What is the transplant evaluation process?
  • What are some testing and evaluation obstacles, both mental and psychosocial?
  • What is dual listing?
  • What happens when you are actively listed?

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

FDA approves Proteostasis’s triple combination program for CF

Singapore — Proteostasis Therapeutics, a clinical stage biopharmaceutical company dedicated to the discovery and development of ground-breaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the Company’s triple combination program for the treatment of cystic fibrosis. The Company’s proprietary triple combination includes a novel cystic fibrosis transmembrane conductance regulator (CFTR) amplifier, third generation corrector and potentiator, known as PTI-428, PTI-801 and PTI-808, respectively. The Company announced in January that the protocol for its triple combination clinical study, which the Company plans to initiate in the current quarter, has received endorsement and a high strategic fit score from the Therapeutics Development Network (TDN) and the Clinical Trial Network (CTN), the drug development arms of the Cystic Fibrosis Foundation (CFF) and the European CF Society (ECFS), respectively.

“Fast Track designation represents another positive step for the development of our triple combination therapy and underscores the serious unmet need that remains for the vast majority of CF patients,” said Meenu Chhabra, president and chief executive officer of Proteostasis Therapeutics.

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. An investigational drug that receives Fast Track program designation is eligible for more frequent communications between the FDA and the company relating to the development plan and clinical trial design and may be eligible for priority review if certain criteria are met.

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A Dutch Company on the Quest Against Cystic Fibrosis

An interview by:  Clara Rodríguez Fernández

Daniel de Boer founded ProQR in 2012 following a strong determination to improve the lives of people with cystic fibrosis. We started ProQR Therapeutics for a very personal reason,” he told me. “Eight years ago, my son was born, and diagnosed with cystic fibrosis. At that time, I was a serial entrepreneur in IT. I decided to make a career switch and start a company to develop drugs for cystic fibrosis, but then also for other genetic diseases.”

One would think that a person without a background in biotech might have it difficult to succeed, but de Boer is not the only to have so far successfully undertaken this endeavor. Over in France Karen Aiach built Lysogene to treat her daughter’s rare genetic disorder, while in the US the story of John Crawley and his company Amicus Therapeutics, founded to help his two children’s diagnosis, went so far as to inspire a movie. The determination and motivation of these parents seem to overdrive any challenges they might have faced because of their limited experience.

De Boer set out to create a business plan for his new company and found out that there was already quite a lot of activity, especially in approaches using small molecules or gene therapy.“We decided that we really wanted to add something new to the space, and take a completely novel approach.”

So he started looking for a new technology, and he found it. “Around that time, I met for the first time with some people in biotech, including the CEO at Alnylam, John Maraganore, and we talked about how they used RNA approaches for genetic diseases,” says de Boer.

Technologies targeting RNA are quite new compared to those that target DNA such as gene therapy. But RNA-based treatments have started to gain traction in the last few years. There are multiple ways that RNA can be used as a therapeutic, but its distinctive advantage over gene therapies and the likes is that it does not permanently change our genetic makeup, making it possible to reverse its effects.

Today, RNA technology is being tested in multiple rare diseases caused by genetic mutations, such as hemophilia, porphyria, or iron overload disorders. I thought, ‘if you can do that for all these other genetic diseases, why not for cystic fibrosis?’” says the Boer. “With that in mind, we started ProQR.”

For the rest of this article, please click here.

Potential Nitric Oxide Treatment for Resistant Bacterial Infections Gets Patent

A possible inhalable treatment for antibiotic-resistant bacterial infections in people with cystic fibrosis due to Pseudomonas aeruginosa now has a U.S. patent and is being readied for a first clinical trial, Novoclem Therapeutics announced.

The patent (No. 9,850,322) was issued to the University of North Carolina (UNC) at Chapel Hill where the potential therapy, BIOC51, was discovered, and covers a technology known as water-soluble polyglucosamine compositions that release nitric oxideContinue reading Potential Nitric Oxide Treatment for Resistant Bacterial Infections Gets Patent

Supercharged antibiotics could turn tide against superbugs

An old drug supercharged by University of Queensland researchers has emerged as a new antibiotic that could destroy some of the world’s most dangerous superbugs.

The supercharge technique , led by Dr Mark Blaskovich and Professor Matt Cooper from UQ’s Institute for Molecular Bioscience (IMB), potentially could revitalise other antibiotics. Continue reading Supercharged antibiotics could turn tide against superbugs

Steps in the Journey: CFTR mutation to sweat chloride concentration to survival

Associations between “salty” sweat and early mortality can be found in the scientific literature dating back to the 17th century [1], hundreds of years before a comprehensive medical description of cystic fibrosis (CF) [2]. Insightful observation of excessive dehydration and deaths among children during a 1948 New York City heat wave suggested that salt homeostasis was a fundamental cellular problem in CF [3], with identification of supranormal sweat chloride concentrations remaining fundamental to the diagnosis of CF today. Since identification of the mutated gene associated with CF (the cystic fibrosis transmembrane conductance regulator; CFTR) [4], pieces of the CF puzzle seem to have, for the most part, fallen into place. Continue reading Steps in the Journey: CFTR mutation to sweat chloride concentration to survival

Clinical Trial Opportunity for Phase IV Airway Clearance System

Med Systems is sponsoring a Phase IV clinical study to measure the
effectiveness of the Electro Flo 5000 Airway Clearance System for
people who have been diagnosed with cystic fibrosis. The goal of the
study is to provide health insurers and Medicare with comprehensive
information regarding the system’s performance. The study is designed
to measure the efficacy of the system, which includes the FDA510K
(K031876) device under current indications. The study will last 30 days
and involve using the system for lung clearance and recording the
results in a digital journal. The study should take about 10 minutes per
day to record measured results in the morning after waking. You will
also be asked to use a spirometer and a digital pulse oximeter to
evaluate your lung function after using the Electro Flo 5000 Airway
Clearance System.

Interested participants must be:
 Between the ages of 18-55 years of age
 Diagnosed with cystic fibrosis
 Prescribed chest physical therapy for airway clearance
 Able to perform self-treatment- having manual dexterity
 Residing in the United States

Contact- Dr. Leigh Mack: CFtrial@mackbio.com or Phone 888-935-
8676 ext. 706

Vitamin D3 deficiency and its association with nasal polyposis in patients with cystic fibrosis

This trial ascertained if the deficiency of vitamin D3 (VD3) correlated with the presence of nasal polyposis (NP) in patients with cystic fibrosis (CF) and patients with chronic rhinosinusitis (CRS). VD3 deficiency appeared to be related to the presence of nasal polyps in the patients with CRS and in the patients with CF in a similar manner. It was inferred that the lower the level of serum VD3, the more severe the mucosal disease was disclosed in the imaging studies and the more frequent microbial colonization of the patients with CF and the patients with CRS. Continue reading Vitamin D3 deficiency and its association with nasal polyposis in patients with cystic fibrosis

CYSTIC FIBROSIS WIND SPRINT 67: CIRCUIT TRAINING 2

For people with cystic fibrosis, getting “back” into shape is a common occurrence. Because of the nature of the disease, patients often experience set backs in both their health and fitness routines. But, exercise is an important and essential part of remaining compliant with treatments and medications in order to live a longer, healthier life with CF.  Continue reading CYSTIC FIBROSIS WIND SPRINT 67: CIRCUIT TRAINING 2