Parkinson’s disease drug hunters think outside the α-synuclein box - Nature Biotechnology

Parkinson’s disease is the fastest growing neurological condition, with no disease-modifying treatments to offer patients. At the center of the disease are neurotoxic clumps of the α-synuclein protein, so it is no surprise that most drugs in pipeline target α-synuclein with either small molecules, antibodies, peptide vaccines or gene therapies.

Choosing α-synuclein as a target for potential disease-modifying drugs is grounded in almost two decades of scientific findings. The protein is present throughout the body and ubiquitously in neurons. But in PD, it is abundant and forms aggregates of misfolded proteins contained in Lewy bodies, which are the hallmark of the disease.

Despite the uncertainties and the setbacks, “α-synuclein remains a viable and important target,” says Greenamyre. Those pursuing α-synuclein include Roche, who has initiated a fresh phase 2 trial of prasinezumab with a primary endpoint of improved motor signs, despite the negative results from the PASADENA trial.

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