Gene therapy does nothing about the other problem - Iron, which...

Gene therapy does nothing about the other problem - Iron, which by tending to that it apparently deals with the aggregation problem as well.

PD is caused by the death of specialized neurons in the region of the brain called the substantia nigra. This is the only part of the brain where iron, dopamine (a neurotransmitter) and the alpha synuclein protein are all present at high concentrations. In PD, iron binds to dopamine, preventing it from functioning normally, and creating toxic free radicals. Iron also binds to alpha synuclein, causing it to aggregate. The aggregation of this protein is a well-established pathological feature of PD, and a target for new disease-modifying therapies.

PBT434 prevents alpha synuclein from aggregating and also prevents the toxic consequences of iron combining with dopamine.

In a further sign of the potential of PBT434 as an effective treatment, its therapeutic benefits were seen to be dose-dependent. Increasing increments of the drug resulted in increased preservation of neurons and increased improvement in motor function.

.....
"What we have known for some time is that dopamine and iron, together in the brain, form a combustible mix and this drives alpha synuclein aggregation and toxicity," said Associate Professor Finkelstein.

"What we've seen with PBT434 is two beneficial modes of action -- it prevents cell death by inhibiting the interaction between dopamine and iron and it also stops this accumulation of alpha synuclein."

This is the first molecule designed to inhibit the neurotoxic build-up of alpha synuclein in the brain and PBT434 could support the "next generation for PD therapies," Associate Professor Finkelstein also said.

http://www.marketwatch.com/story/pbt434-drug-candidate-shows-potential-as-next-generation-disease-modifying-treatment-in-parkinsons-disease-2013-06-20