Parkinson’s disease ranks amongst the most common neurodegenerative disorders. European researchers are working on the first therapeutic vaccines that could improve disease outcome.
Parkinson’s disease (PD) and multiple system atrophy (MSA) are two neurodegenerative disorders associated with a progressive decline in motor and cognitive functions as well as behavioural changes. The neuropathological hallmark of these diseases are so called Lewy bodies. These are primarily composed of aggregates of the protein alpha-synuclein. Currently there are no therapies available only treatment of symptoms.
The scope of the EU-funded SYMPATH (Reach α-synuclein-dependent neurodegeneration: clinical development of therapeutic AFFITOPE vaccines for Parkinson’s disease and multisystem atrophy) project is to develop therapeutic vaccines. Given the role of alpha-synuclein in disease pathophysiology, the consortium aims to target the protein as a disease-modifying strategy.
The vaccine candidates are peptide-protein conjugate vaccines that were designed to elicit neutralising antibodies against alpha-synuclein. Scientists used the novel AFFITOPE® technology, which relies on short peptides to mimic parts of the native sequence or structure.
Pre-clinical evaluation of these vaccine candidates demonstrated their disease-modifying activity in various models associated with lower cerebral levels of aggregated protein, less severe neuropathological alterations and improvements of functional deficits.
During the SYMPATH project, the production of these vaccines will be optimised for clinical application. The efficacy and safety of these two vaccines in early stage PD or MSA patients will take place in two trials in Austria and France. In addition, the consortium is working on novel methods for assessing alpha-synuclein specific antibody responses in humans and alpha-synuclein in the cerebrospinal fluid.
On another level, study partners are generating a unique collection of data and biological material from patients suffering from synucleinopathies and undergoing immunotherapeutic intervention. This information should prove valuable for future studies.
Considering the novelty of the SYMPATH work, the project has attracted extensive media attention. The initial press release itself generated more than 300 press reports worldwide.
The outcome of the project has tremendous clinical significance and is anticipated to revolutionise therapy of neurodegeneration.