The promise of targeting iron in the brain for Parkinson’s
Why is it important?
- Novelty 80% 80%
- Proximity 80% 80%
- Deliverability 60% 60%
This excess iron hampers neuronal survival directly, and increases the build up of a-synuclein which damages neurons as it forms clumps inside them. So reducing this excess iron using “iron chelator” drugs should be beneficial. One of these is Deferiprone, often used in blood disorders where regular blood transfusions can unintentionally cause iron overload. Since it crosses the blood-brain barrier, an important hurdle when targeting the brain, it mops up excess iron and counteracts its toxic effect on neurons. Based on this and strong evidence from animal studies, Deferiprone was prioritised by the LCT committee in 2012 as a potential disease modifying therapy.
FAIRPARK II (link below), a larger European multicenter, parallel-group, placebo-controlled, randomized clinical trial, is recruiting newly diagnosed patients to assess whether Deferiprone can slow down Parkinson’s. 388 people will take Deferiprone orally twice daily or placebo for 9 months, and will be monitored over the course of a month after the end of the treatment period. Their movement scores as well as aspects of quality of life and cognition will be assessed.
Where can I learn more?
Martin-Bastida, A., Ward, R. J., Newbould, R., Piccini, P., Sharp, D., Kabba, C., . . . Dexter, D. T. (2017). Brain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in Parkinson’s disease. Sci Rep, 7(1), 1398. doi:10.1038/s41598-017-01402-2
Devos, D., Moreau, C., Devedjian, J.C., et al. (2014). Targeting chelatable iron as a therapeutic modality in Parkinson’s disease. Antioxid Redox Signal, 21, 195-210.
Original article: Moreau, C., Duce, J. A., Rascol, O., Devedjian, J. C., Berg, D., Dexter, D., . . . group, F.-I. s. Jan 30, 2018. Iron as a therapeutic target for Parkinson’s disease.