Research Project information
Principal researcher: Professor Luciana Chessa
Institute: The Sapienza University of Rome, Italy
Grant award: €78,445
Start Date: January 2015
Duration: 12 months
This research project in partnership with Sparks and the A-T Society is aimed at understanding how specific types of drugs called steroids, affect symptoms experienced by those who have A-T.
Summary of research project
Recent small scale trials have shown a positive effect of dexamethasone, a type of steroid, on neurological symptoms experienced by A-T patients. However it is currently not understood why it has this effect. The two most likely mechanisms are the known anti-inflammatory properties of steroids or that the drug permits creation of a shortened but active version of the ATM protein which is missing or not functioning completely in A-T. The team led by Professor Luciana Chessa at the Sapienza University in Rome, Italy, will use a new technique to study cells from A-T patients to try and understand how the drug acts and why it affects patients differently. The team aim to fully understand the mechanisms of action of steroids in A-T and consequently be able to help develop effective therapies for individual A-T patients in the future.