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The AFFITOME® technology, in particular its safety features, allows to introduce immunotherapy against diseases caused by self-molecules. Alongside Alzheimer’s disease, Parkinson’s disease is one of the most serious neurodegenerative conditions, similarly associated with advancing age. Due to demographic trends, experts predict that the number of Parkinson patients in the western world will rise from today’s 4 million to about 9 million in 2030. Currently, only symptomatically acting drugs are registered and available, which are for example based on the compensation of the deficiency of the neurotransmitter dopamine. A causal treatment, however, is lacking. AFFiRiS already started the preclinical programme as part of the development of a novel vaccine against Parkinson’s disease. Relevant patent applications have been submitted.
The self protein α-synuclein is the main constituent of the protein deposits found in degenerating dopaminergic cells in Parkinson’s patients. Analogous to ß-amyloid in Alzheimer disease, α-synuclein forms aggregates which deposit within neurons as so called Lewy bodies and lead to the cells’ demise. The typical symptoms of Parkinson’s disease arise from progressive nerve cell loss in specific brain regions: progressive immobility (akinesis), stiffness (rigor) and trembling (tremor). In general, it is assumed that a reduction in cerebral α-synuclein concentration has a beneficial effect on disease progression. The vaccine developed by AFFiRiS, known as PD01, is currently undergoing preclinical efficacy testing (proof of concept). The Parkinson’s vaccine PD01 which is targeting the harmful α-synuclein protein, is designed to exert a profound and sustained therapeutic effect. PD01 is expected to induce disease modifying effects and should not provide symptomatic treatment only. It must be noted, however, that α-synuclein is one of the body’s own proteins, belonging to a protein family whose members exhibit extensive structural similarities.
Other members of this protein family, namely ß-synuclein and γ-synuclein, are important to the healthy body. Any treatment aimed at decreasing α-synuclein must be carefully developed in order to take the fine structural differences between these related proteins into account. AFFITOME® is just the right technology for dealing with such issues.
