Understanding dexamethasone action in Ataxia Telangiectasia


Research Project Information

Principal researcher: Professor Luciana Chessa
Institute: Department of Clinical and Molecular Medicine, University La Sapienza, Italy
Grant award: £78,445.00
Project completion date: 31st December 2016

Project Overview
Recent studies have shown that the steroid drug, Dexamethasone (Dexa), can improve some of the neurological symptoms experienced by patients with Ataxia Telangiectasia (A-T). But why Dexa works and how it affects A-T cells is unknown. A better understanding of how, and why, the drug works could help to improve existing treatment or identify targets for new ones.

In order to understand the exact mechanism of Dexa, Professor Luciana Chessa and her team undertook a series of intricate experiments which looked at different processes within A-T cells and compared them to healthy cells.

Project Outcome
Overall, A-T cells showed significant differences in their response to Dexa, compared to healthy cells. The beneficial effect seems to be a result of a reduction in damage to cells, caused by an increase in molecules that protect the cells from harmful molecules called reactive oxygen species.

Chessa L, Micheli R, Molinaro A. Focusing New Ataxia Telangiectasia Therapeutic Approaches. J Rare Dis Diagn Ther. 2016, 2:2.

Biagiotti, S. et al. Dexamethasone improves redox state in ataxia telangiectasia cells by promoting an NRF2-mediated antioxidant response. The FEBS Journal. 2016.

Menotta, M. et al. Nano-Mechanical Characterization of Ataxia Telangiectasia Cells Treated with Dexamethasone. Cell Biochem Biophys (2017) 75:95–102.

What Next?
The team hope to use their findings to work towards new and improved ways to treat A-T in the future.