FFC#2/2020

Lipid-based therapeutic strategies to optimize the effectiveness of innovative drugs to rescue F508del-CFTR

AREA 1 Therapies to correct the underlying defect

FFC#2/2020

Lipid-based therapeutic strategies to optimize the effectiveness of innovative drugs to rescue F508del-CFTR
€ 0 still needed
0%
€ 87.000 goal

pRINCIPAL INVESTIGATOR

Massimo Aureli (Università di Milano, Dip. Biotecnologie Mediche e Medicina Traslazionale)

Partner

Anna Tamanini (AOUI Verona, Lab. Analisi)

Researchers

10

Category

AREA 1 Therapies to correct the underlying defect

Duration

2 years

Goal

€ 87.000

Funds raised

€ 87.000

Objectives

The potentiator Ivacaftor (Kalydeco), also included in the triple combination Kaftrio or Trikafta, might display, as a side effect, the reduction in the amount of CFTR protein at the apical cell membrane. A similar reducing effect may also be caused by Pseudomonas aeruginosa infection. Such a negative effect can be compensated by two lipid compounds, namely GM1 ganglioside and cholesterol (or its analogue beta-sitosterol); they are already on the market to treat other pathologies. The proposal intends to investigate if the exogenous administration of those lipids, which are deficient in the CF bronchial epithelial cells, can increase the efficiency of correctors and potentiators and the functional recovery of CFTR-F508del, even when P. aeruginosa infections are present, with a stabilizing effect. Researchers will work in primary cells from explanted CF lungs. The approach could represent the starting point for the development of new therapeutic strategies based on the association of lipids and modulators, to improve the effectiveness of CFTR modulators themselves.

WHO ADOPTED THE PROJECT

Associazione Trentina Fibrosi Cistica dedicato a Carmen Peterlana Cainelli

€ 20.000

Delegazione FFC di Vercelli

€ 20.000

Delegazione FFC di Vigevano

€ 47.000

OTHER PROJECTS

Discover the other projects

GMRF#1/2026

Exploring the role of PKD1 in promoting CFTR stability and function at the cell surface

FFC#1/2026

Mapping proteins regulating CFTR mRNA stability to identify new therapeutic targets for nonsense mutations

FFC#2/2026

Evaluating the potential of phosphodiesterase inhibitors to enhance the efficacy of CFTR modulators and support personalized therapeutic approaches