First-in-human Trial to Assess the Safety and Tolerability of PD01, the First Alpha-synuclein-based AFFITOPE® Parkinson's Vaccine

Objective/Rationale:
Parkinson’s disease (PD) is characterized by the presence of protein aggregates called Lewy bodies, with a major component of the protein alpha-synuclein. This fact and comprehensive scientific data strongly suggest that alpha-synuclein plays a central role in the pathogenesis of PD. Hence it is assumed that a reduction of alpha-synuclein in the brain has the potential to modify the pathophysiology underlying the disease. The AFFITOPE® vaccine candidate PD01 has been designed to target alpha-synuclein. Its application to pre-clinical models was found to reduce accumulation of alpha-synuclein and, consequently, to ease alpha-synuclein-triggered pathology, both on a neuropathological and a functional level. The objective of this project is the evaluation of PD01 for the first time in human beings with regard to its safety/tolerability profile, but also with respect to its immunological and clinical activity.

Project Description:
The novel anti alpha-synuclein vaccine candidate PD01, identified by the AFFiRiS’ AFFITOME® technology, has passed all pre-clinical development stages including toxicity- and proof-of-concept studies. During this project, PD01 will be for the first time applied to human beings. Within this prospective clinical trial, PD patients will be vaccinated four times by injection of PD01. This treatment period will be followed by an observational period. Patients will be assessed primarily for safety/tolerability of the immunizations but also with regard to the immunological and clinical activity of PD01.

Relevance to Diagnosis/Treatment of Parkinson’s Disease:
According to the researchers' knowledge, AFFITOPE® PD01 is the first alpha-synuclein-targeting active vaccine to be introduced into the phase of clinical development. Most importantly and in contrast to currently approved PD drugs, it aims at modifying disease pathology and, thus, the course of the disease. If successful, this would signal a new era in the treatment of PD.

Anticipated Outcome:
The project will inform investigators on the safety- and toxicity profile of PD01. In addition, data on the immunological- and clinical activity will provide the basis for further clinical development of PD01.