Hope for cystic fibrosis with range of novel treatments in development

New kinds of treatments in development may help turn the life-threatening condition into a manageable, chronic one

According to the Cystic Fibrosis Foundation, more than 70,000 worldwide suffer from cystic fibrosis (CF), a genetic condition that causes the lungs to become clogged with mucus, making it difficult to breathe.

People living with cystic fibrosis (CF) have a significantly reduced life-expectancy; median age of death is just 28 years.

Quality of life is also extremely poor due to high treatment burden and susceptibility to chronic lung infections that result in frequent hospitalisations.

Patients face considerable unmet needs due to the lack of curative therapies, the limited choice of mucolytic therapies and the constant fight against lung infection requiring the development of novel classes of antibiotics.

CTFR modulators

Recent months have been encouraging for cystic fibrosis treatments, with the introduction of disease-modifying cystic fibrosis transmembrane conductance regulator (CFTR) modulators.

Pavan Kottamasu, Healthcare Analyst for GlobalData, explains: “Until recently, R and D strategies in the cystic fibrosis space have targeted organ-specific sequelae of the underlying disease.”

“This approach has mainly resulted in the development of new formulations of antibiotics that can be administered via inhalation for the management of chest infections, and therapies that enhance airway mucociliary clearance.”

CFTR modulators can potentially turn this life-threatening condition into a chronic one.

“However, during the last decade, advanced technologies have yielded a shift in research development towards therapies that target the underlying cause of the disease by enhancing CFTR function.”

“There is currently enormous excitement for CFTR modulators that can modify the course of the disease and potentially turn this life-threatening condition into a chronic one.”

Bronchospheres

In addition, Enterprise Therapeutics, a UK-based drug discovery company for respiratory disease therapies, will identify new drug mechanisms for the treatment of CF.

Using funding won from the Cystic Fibrosis Trust, the research will leverage Enterprise Therapeutics’ bronchosphere technology platform.

Bronchospheres are a miniaturised model of the human airway. This model can be used to support high-throughput drug and target discovery and will be used to facilitate the development of new classes of therapeutics for the treatment of cystic fibrosis.

The spheres are cultured in a 3D matrix and form ball-like structures. The ciliated cells line the interior of the structure and beat to propel the mucus gel, a vital component of the mucociliary clearance system.

Bronchospheres also contain the mucus producing goblet cells. Each bronchosphere can be considered a small segment of airway. This enables study of the key airway cells, in the context of a fully functioning epithelium.


Together, these developments could mean significant market increases for the cystic fibrosis space. GlobalData predict a tripling from $2.1bn in 2015 to $7.6bn by 2025, across the seven major markets of the US, France, Germany, Italy, Spain, the UK, and Canada.

“The majority of novel therapeutic agents for cystic fibrosis are being developed by small companies. Licensing activity and new partnerships will steer smaller companies to continue researching novel compounds,” Kottamasu said.

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