Study Rationale: In many neurodegenerative disorders, including Parkinson’s disease (PD) and (MSA), the protein alpha-synuclein is structurally altered and forms aggregates in the brain. These accumulated clumps impair the function of nerve cells and cause neurodegeneration. To diagnose these disorders and to measure the effects of experimental therapies aimed at reducing aggregate accumulation in the brain, a diagnostic test that allows measurement of these protein aggregates is urgently needed. This detection can be accomplished by imaging with radioactively labeled chemical compounds, such as MODAG-005, that bind specifically to alpha-synuclein aggregates in the brain.
Hypothesis: We have demonstrated that MODAG-005 binds specifically to aggregated alpha-synuclein in pre-clinical models of PD and MSA. In this study, we propose to confirm that MODAG-005 administration is safe in humans and allows the measurement of alpha-synuclein aggregates in people with PD or MSA.
Study Design: The safety of MODAG-005 will be assessed in healthy volunteers and individuals with PD or MSA. To this end, we will clinically monitor the well-being of all study participants by physical and neurological examinations and standardized questionnaires. We will also analyze vital signs and blood samples. Furthermore, we will assess whether we can visualize alpha-synuclein aggregates in the brains of these individuals by positron emission tomography (PET) imaging using radioactively labelled MODAG-005.
Impact on Diagnosis/Treatment of Parkinson’s Disease: The successful use of radioactively labeled MODAG-005 as a tracer for PET imaging to detect alpha-synuclein aggregates in PD and MSA would allow the early and differential diagnosis of these diseases and facilitate monitoring the progression and effect of potential disease-modifying therapies in people with the disorders.
Next Steps for Development: If successful, the next step would be to apply MODAG-005 to more patients. Further confirmation of MODAG-005’s ability to visualize alpha-synuclein aggregates in the human brain would eventually allow this novel PET tracer to be made available to all patients worldwide in daily clinically routine.
Trial Phase: 1