Parkinson’s disease (PD) is a complex neurodegenerative condition with a multifactorial origin. To date, approaches to
drug discovery for PD have resulted in symptomatic therapies for the motor manifestations and signs associated with
neurodegeneration but have failed to identify preventive or curative therapies. This failure mainly originates from the
persistence of major gaps in our understanding of the specific molecular basis of PD initiation and progression. New
approach methodologies (NAMs) hold the potential to advance PD research while facilitating a move away from animalbased research. We report a workshop involving NAM experts in the field of PD and neurodegenerative diseases, who
discussed and identified a scientific strategy for successful, human-specific PD research. We outline some of the most
important human-specific NAMs, along with their main potentials and limitations, and suggest possible ways to overcome
the latter. Key recommendations to advance PD research include integrating NAMs while accounting for multiple levels of
complexity, from molecular to population level; learning from recent advances in Alzheimer’s disease research; increasing
the sharing of data; promoting innovative pilot studies on disease pathogenesis; and accessing philanthropic funding to
enable studies using novel approaches. Collaborative efforts between different stakeholders including researchers,
clinicians and funding agencies are urgently needed to create a scientific roadmap and support a paradigm change towards
effective, human-specific research for neurodegenerative diseases without animals, as is already happening in the field of
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