Scientists aim for treatment that blocks toxic protein in Parkinson’s
3-year project to stop alpha-synuclein spread 'offers hope for a cure'
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- Parkinson's disease progression is linked to alpha-synuclein protein clumps in the brain.
- Scientists are developing therapies to block these toxic aggregates.
- The project aims for testing-ready treatment candidates by 2029.
Scientists in Denmark are embarking on a three-year project aiming to develop a Parkinson’s disease treatment that blocks the spread of toxic alpha-synuclein protein in the brain.
“This breakthrough project positions Denmark at the forefront of Parkinson’s disease research, with a bold strategy that offers hope for a cure for patients living with this terrible disease,” project leader Simon Glerup, PhD, associate professor at Aarhus University and co-founder and chief scientific officer of Draupnir Bio, said in a company press release.
Toxic clumps of alpha-synuclein in the brain are a molecular hallmark of Parkinson’s and are thought to play a central role in driving the disease. The project, funded by a 26.7 million DKK (just over $4 million) investment from Innovation Fund Denmark, aims to develop therapies that can target alpha-synuclein in the space around nerve cells. The goal is to stop the spread of these toxic protein clumps that are thought to drive Parkinson’s progression.
The project, dubbed DESYNA (Degradation of Extracellular alpha-SYNuclein Aggregates) will combine research into alpha-synuclein’s role in Parkinson’s done at Aarhus with protein-destroying technology from Draupnir. Researchers hope to design therapies that can grab onto the alpha-synuclein protein and drag it to lysosomes, compartments that act as cellular garbage disposals to destroy molecular waste.
“Our therapy would be the first in the world to specifically remove and prevent the spread of a toxic protein build-up in the brain that is proven to sustain disease progression,” said Glerup.
Scientists hope to have testing-ready candidates by 2029
The project’s goal is to develop both biologics — complex molecules such as antibodies produced in living cells and usually given by injection — and small-molecule therapies that can be taken by mouth. Researchers hope to have preclinically validated candidates ready for further testing by 2029.
“We know that aggregation of [alpha]-syn is central to the progression of Parkinson’s disease,” said Daniel Otzen, PhD, Aarhus professor and DESYNA project partner. “So, by finding new ways to target this process, we aim to go beyond managing symptoms and instead change the course of the disease itself. This approach has the potential to open the door to entirely new treatments and, importantly, to give people living with Parkinson’s disease, and their families, real hope for the future.”
