Advances in Research on Treating Cystic Fibrosis

In Belgium, an antibody has been discovered that stabilizes the protein responsible for cystic fibrosis, opening up new possibilities for therapeutic experiments

Nearly 30 years after the cause of cystic fibrosis was discovered, there is still no cure for this lethal genetic disorder. With support from the Air Liquide Foundation, researchers at Université Libre de of Bruxelles (ULB) are studying the mutations of the CFTR protein provoking Cystic Fibrosis in order to identify molecular targets for future therapies.

Project Identity

Project Initiator: Fondation Université Libre de Bruxelles Year(s) of support: 2013, 2017 & 2021-2023
Support provided: Salaries for the researches, laboratory expenses, equipments Amount provided: 200 000€
Location: Brussels, Belgium R&D Manager: Jean-Christophe Richard, Medical Expert, Air Liquide Medical Systems

 

A genetic mutation causing the disease

We have known since 1989 that cystic fibrosis affects over 100 000 people worldwide. This disease is caused by a genetic mutation that changes the CFTR protein responsible for regulating the transport of chloride through cell membranes. This malfunction leads to more viscous mucus that accumulates in the respiratory and digestive tracts.

Over 100.000 people worldwide are affected by Cystic Fibrosis.

Analysing the origins of the mutations of the CFTR protein

Prof. Govaerts and his research team from ULB have been concentrating their efforts on the faulty protein that causes cystic fibrosis. Indeed, current therapeutic approaches do not apply to a number of mutations in the functional domain of the CFTR protein. Their aim is to open new therapeutic avenues for patients bearing such rarer mutations.

The Work

The research work of Prof. Govaerts' team is divided into 3 axes:

  • Studying the reasons for the mutation-induced misfolding of CFTR and whether an alternative conformation is a structural intermediate for misfolding. Indeed, this phenomenon is a physical process when the protein gains its functional tridimensional structure
  • Using nanobodies to decipher the conformational network within the CFTR functional domain and establish the relation to physiopathology, meaning a relation between the protein’s structure and the cause of the disease
  • Investigating the conformational effect of rare Cystic Fibrosis-causing mutations

The Foundation Renews Its Support

Having been involved since 2013 in the effort to identify and characterize nanobodies, the Air Liquide Foundation is supporting ULB researchers during this third phase focusing on the study of the mutations of the CFTR protein. A new grant of 100 000€ is being provided for the salaries of the researchers and the laboratory equipment for the 2021-2023 support.

ULB Foundation

Testimonial

“This project covers an undoubtedly important subject, in continuity of the work of an expert team in this field which has been benefiting from the Air Liquide Foundation’s support for several years.“

Jean-Christophe Richard, Expert Médical, Air Liquide Medical Systems