A Pilot Clinical Trial With the Iron Chelator Deferiprone in Parkinson's Disease

Parkinson's disease (PD) is a common neurodegenerative disease affecting movement. Although drug treatments for PD are available they only treat the symptoms of the disease, fail to halt neuronal loss, and are associated with long term side effects and loss of efficacy. There is a chronic need to develop neuroprotective therapies. Increased iron and oxidative stress have been heavily implicated in the neurodegenerative process in PD, hence removal of excess iron by iron chelation represents a potential drug target. Iron chelators are extensively utilised to treat peripheral iron overload disorders (e.g. thalassaemia) and recently the investigators have demonstrated iron chelators such as Deferiprone can enter the brain removing excess iron and are neuroprotective in PD animal models. Although good tolerability and efficacy to remove brain iron has also been shown in a pilot study with the iron chelators Deferiprone in young patients with Friedreich Ataxia, where iron accumulates in the dentate nucleus, no studies have been conducted in aged individuals affected by PD. Hence the aims of this study are 1) to assess whether Deferiprone is well tolerated in PD patients, 2) whether Deferiprone can remove the excess iron levels found in the brain area affected by PD, the substantia nigra, as assessed by Magnetic resonance imaging (MRI) and 3) whether Deferiprone has any direct effect on the clinical symptoms of PD. Three groups of 12 (total 36) early stage drug free PD patients will be treated with 20 or 30mg/kg/d Deferiprone or Placebo for 6 months. Over the 6 months patients will receive serial MRI scans, neurological examinations not only to assess PD symptoms but also psychological state, plus blood test to monitor for potential side effects. Positive results from this pilot will help support larger clinical trials to evaluate whether Deferiprone can slow down/halt PD.