Deferiprone is a drug already used to remove excess iron in the body in people who have certain blood disorders. Deferiprone easily crosses the human brain’s protective layer (the blood-brain barrier) crossing from the bloodstream into the brain, and in so doing it removes the excess iron which may be harmful to brain neurons.

For this reason, researchers have been wondering if deferiprone could be useful in Parkinson’s. Deferiprone is called an iron chelator. The drug works by attaching itself to iron molecules which the body can then excrete.

Why do researchers think this drug is a potential treatment to slow, stop or reverse Parkinson’s?

It is believed that high levels of iron in the substantia nigra of people with Parkinson’s causes ‘oxidative stress’ in neurons resulting in their demise. Oxidative stress can cause cell damage or death. For many years, oxidative stress and mitochondrial dysfunction have been considered as one of the main mechanisms involved in cell death in Parkinson’s and oxidative stress is exacerbated by excess iron. Chelation (removal) of this free iron has powerful antioxidant effects known to dramatically increase cell survival, and it is hoped that decreasing the levels of iron in the brain will improve symptoms and decrease the rate of disease progression.

Deferiprone – the trial

Dr David Devos has led a Europe-wide consortia of neurologists and Parkinson’s specialists to secure funding from the EU’s Horizon 2020 programme to run a clinical trial through 28 centres across Europe to understand the efficacy of deferiprone in people with Parkinson’s.

It is hoped that this trial will demonstrate for the first time in a large phase ll, multicentre, parallel-group, placebo-controlled, randomly selected trial that iron chelation with the drug deferiprone will slow down the progression Parkinson’s. A nine month treatment period will be followed by a one month post-treatment monitoring period in order to assess the disease-modifying effect in the absence of deferiprone versus placebo. The Movement Disorders Society – Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) will be used which encompasses measurement of movement, cognition behaviour and activities of daily living.

We expect this trial to observe a significantly lower MDS-UPDRS score relative to the control group. This will enable a demonstration of the efficacy of iron chelation as a treatment to slow the progression of Parkinson’s. We hope that the findings of this trial will be widely disseminated in order to promote and support the clinical development of deferiprone and other selected iron chelators for the treatment of Parkinson’s.