Exploring Omaveloxolone efficacy in Ataxia-telangiectasia
Research Project information
Principal researcher: Professor Paola Giunti (pictured) and Dr Rosella Abeti
Institute: University College London (UCL), UK
Cost: £24,914.62 over 4 months
Start Date: 23rd of September 2024
What are the researchers proposing to do?
One of the processes happening in people who have A-T is increased oxidative stress. Oxidative stress is an imbalance of free radicals and antioxidants in your body that leads to cell damage. The team at UCL will study a recently approved drug called Omaveloxolone (Omav). This drug is known to defeat oxidative stress, and the team will investigate whether it could help overcome the adverse effects of oxidative stress in A-T cells. Omav activates a natural defence system in our cells that fights off damage caused by stress. To test this, they will use skin cells from A-T patients and see if Omav can lower the levels of reactive oxygen species that are generated in patients’ cells, leading to oxidative stress. They will then compare the levels of reactive oxygen species in A-T patients to healthy volunteer lines to verify that Omav is effective.
Why?
Recently, Omav has been approved for the treatment of Friedreich’s ataxia, a different degenerative condition which is characterised by oxidative stress. It is important to now investigate whether Omav has the potential in reducing oxidative stress in A-T patients as it could offer a promising therapeutic intervention and a fast routing toward clinical trials.
How will the research be done?
The team will first obtain the A-T cell lines from the University of Birmingham depository in collaboration with Professor Grant Stewart. These cell lines will be expanded and cultured in their laboratory. Then, they will measure the levels of oxidative stress with and without Omav. Lastly, they will perform protein quantification analysis to verify the activation of the antioxidant pathway in A-T patients’ fibroblast lines under treatment with Omav.
How could it make a difference in the lives of those affected by A-T?
If it proves to be efficacious in A-T cells, their study may lead to a potential new treatment from which A-T patients could benefit in a relatively short time.
