A Breath of Fresh Air for Biotechs Working on Cystic Fibrosis Therapies

Researchers from the University of Zurich have determined the structure of a chloride channel, which could be a target for new drugs to treat cystic fibrosis.

Researchers at the University of Zurich have found a new target for future cystic fibrosis treatments. The study, published in Nature, has uncovered the structure of a protein that could help to correct the mechanism underlying the buildup of sticky mucus in patients’ lungs. This could give rise to a new wave of therapeutics for the condition, which at the moment lacks disease-modifying treatments.

Cystic fibrosis is a severe genetic disease affecting the lungs, for which there is currently no cure. It is caused by a malfunctioning chloride channel, CFTR, which prevents the secretion of chloride by cells, leading to the production of thick, sticky mucus in the lung. The condition affects around 70,000 people worldwide, who suffer from chronic infections and require daily physiotherapy.

However, one potential approach to treat cystic fibrosis is to activate the calcium-activated chloride channel, TMEM16A, as an alternative route for chloride efflux. As TMEM16A is located within the same epithelium as CFTR, its activation could rehydrate the mucus layer. The research group used cryo-electron microscopy to decipher the structure of TMEM16A, which is part of a protein family that facilitates the flow of negatively charged ions or lipids across the cell membrane.

The changes that occur in the lungs of cystic fibrosis patients.

TMEM16A is found in many of our organs, playing a key role in muscle contraction and pain perception, as well as in the lungs. It forms an hourglass-shaped protein-enclosed channel, which when bound by positively charged calcium ions, opens to let chloride ions to pass through the membrane.

Current treatments for cystic fibrosis include bronchodilators, mucus thinners, antibiotics, and physiotherapy, which only control symptoms. However, biotechs around Europe are beginning to make progress, with ProQR completing a Phase Ib trial and Galapagos and Abbvie’s triple combination therapy entering Phase I. Antabio has also received €7.6M from CARB-X to develop a new antibiotic against Pseudomonas infections.

The identification of a new target provides patients and biotechs alike with renewed hope of new and effective cystic fibrosis treatments, or even a cure. It will be interesting to see whether small molecules or gene therapy specialists could take advantage of this information.

Original article: https://labiotech.eu/cystic-fibrosis-treatment-target/

Home Spirometers: A Useful Tool in Tracking CF Symptoms and Progress

Guest Blog By: Meranda Honaker

Over the last several months my health has continued to decline despite being compliant and diligent with my healthcare routine. In July I developed a fever during a trip to Boston to speak to a biotech company about my journey with cystic fibrosis. I developed a fever and by the end of my visit, I was unable to walk up steps without severe dyspnea. I was so exhausted from feeling sick I would return to my hotel room to lay in the bed for hours to rest. My chest pain and shortness of breath became so severe in the coming days that I could no longer take a deep breath. I checked my SpiroPd home spirometer which displayed a definite decline in my lung function. I immediately contacted my CF clinic to see my CF doctor. Initially, despite feeling bad, I assumed I was feeling poorly and decided not to rush to CF clinic. Sometimes I have a bad day or few bad days health wise and begin to improve on my own. Once I saw my lung function had declined I knew I needed to be seen in CF clinic rather than waiting it out.
Continue reading Home Spirometers: A Useful Tool in Tracking CF Symptoms and Progress

Phase 1a study for drug to treat CF regardless of CF Mutation

http://www.businesswire.com/news/home/20171018005403/en/Synspira-Announces-Patient-Dosed-Phase-1a-Study

Synspira Announces First Patient Dosed in Phase 1a Study of SNSP113 in Cystic Fibrosis
— First-in-class drug candidate for treatment of cystic fibrosis regardless of genetic mutation — Continue reading Phase 1a study for drug to treat CF regardless of CF Mutation

Stem cell therapy for lung fibrosis conditions

https://www.sciencedaily.com/releases/2017/08/170803091928.htm

In the journal Respiratory Research, the scientists demonstrated that they could harvest lung stem cells from people using a relatively non-invasive, doctor’s-office technique. They were then able to multiply the harvested lung Continue reading Stem cell therapy for lung fibrosis conditions

Clinical trial assessing the potential of novel anti-inflammatory investigational therapy

http://m.marketwired.com/press-release/celtaxsys-announces-full-enrollment-its-landmark-empire-cf-phase-2b-clinical-trial-assessing-2216923.htm

Celtaxsys announces full enrollment of its landmark EMPIRE-CF phase 2b clinical trial assessing the potential of novel anti-inflammatory investigational therapy, oral acebilustat, to preserve lung function in CF patients
Continue reading Clinical trial assessing the potential of novel anti-inflammatory investigational therapy

New Techniques Drastically Prolongs Life of Donor Lungs

New Cross-Circulation Technique Drastically Prolongs Life of Donor Lungs

By Mohammad Saleh
Originally published in medGadget 3/10/17

Lung transplants remain the only hope for patients suffering from end-stage lung disease. Transplants are often preceded by an extensive period on a waiting list because of a donor shortage, a reality Continue reading New Techniques Drastically Prolongs Life of Donor Lungs

Animal model with CF shows promise against infection

http://finance.yahoo.com/news/aeol-20415-protects-lungs-against-130000156.html

AEOL 20415 Protects Lungs Against Infection in Animal Model of Cystic Fibrosis

• TREATMENT WITH AEOL 20415 REDUCED INFECTION, IMPROVED BODY WEIGHT AND REDUCED PRESENCE OF WHITE Continue reading Animal model with CF shows promise against infection

Patients with Severe Chronic Rhinosinusitis Show Improvement with Verapamil Treatment

http://www.newswise.com/articles/view/668018/?sc=mwhn

A clinical trial studying the use of Verapamil (a drug currently in use for cardiovascular disease and cluster headache) in alleviating chronic rhinosinusitis (CRS) with nasal polyps revealed significant improvement in the symptoms of this subset of patients. It is the first study of its kind to explore Continue reading Patients with Severe Chronic Rhinosinusitis Show Improvement with Verapamil Treatment