In pursuing this goal, the Fondazione per la Ricerca sulla Fibrosi Cistica ( FFC ) Onlus has chosen to invest in the excellence "Made in Italy" by involving as a protagonist of the task force the Department of Drug Discovery and Developement of the Istituto Italiano di Tecnologia, a research institute at the forefront of the discovery of innovative medicines with Dr. Tiziano Bandiera and Prof. Daniele Piomelli. Other primary actor of the project is the Laboratory of Molecular Genetics of the Istituto G. Gaslini in Genoa, historically expert in CFTR pharmacology and high-throughput screening, with Dr. Luis Galietta and Dr. Nicoletta Pedemonte.
"We are very proud of this project and the fact that FFC Onlus has decided to support our efforts by promoting an initiative of strategic importance." - says Dr. Tiziano Bandiera, scientific project leader at Istituto Italiano di Tecnologia: "Within the TFCF project our Institute will provide the collection of chemical compounds necessary for the initial screening and will deal with the synthesis of new molecules able to improve the functionality of the defective CFTR protein. We are very enthusiastic. Achieving this goal would be an unprecedented turning point in the history of this serious disease. "
FFC Onlus, which has already contributed to a considerable extent in the field of treatments of the basic deficiency, as well as in the one of the treatments for the lungs infection and inflammation of cystic fibrosis, has committed to fund the first three stages of the study (2014-2017) with a total contribution equal to € 1,250,000 out of the overall cost of the project amounting to € 1,615,000.
The detailed steps of the project Task Force for Cystic Fibrosis follow:
2014 | Phase 1 ( 12 months)
- Screening of a collection of more than 11,000 compounds and identification of active compounds ( hit identification)
- Selection of the most active compounds on the mutant protein
- Validation of the active compounds on bronchial cells from primary cultures (from the lungs of CF patients undergoing transplantation ) , biological conditions closer to the patient .
2015 | Phase 2 ( 12 months)
- Screening of the most promising chemical compounds and their chemical modification in search of the most effective parent molecules ( hit to lead ) .
2016-2017 | Phase 3 ( 18 months)
- Optimization of the parent molecules (lead optimization) and identification of a final compound
- Studies of the pharmacokinetics and safety for the development of a potential drug for the treatment of cystic fibrosis to be subsequently tested in vivo.