Genetically Targeted Clinical Trials in Parkinson's Disease: Learning from the Successes Made in Oncology

• Published in: Genes Vol. 12; no. 10; p. 1529
• Main Authors: Sjögren, Magnus, Huttunen, Henri J, Svenningsson, Per, Widner, Håkan
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI AG 28.09.2021; MDPI
• Subjects: Antiparkinson Agents - therapeutic use; Biomarkers; Clinical trials; Clinical Trials as Topic; Dopamine receptors; Drug development; Genetic targets; Humans; Kinases; Medicin och hälsovetenskap; Molecular Targeted Therapy - methods; Movement disorders; Mutation; Neurodegeneration; Neurodegenerative diseases; Neurodegenerative disorders; Oncology; Parkinson Disease - drug therapy; Parkinson Disease - genetics; Parkinson's disease; Precision medicine; Precision Medicine - methods; Review; precision medicine; clinical trials; genetic targets; neurodegenerative disorders; Parkinson’s disease; oncology
• ISSN: 2073-4425; 2073-4425
• DOI: 10.3390/genes12101529

Clinical trials in neurodegenerative disorders have been associated with high rate of failures, while in oncology, the implementation of precision medicine and focus on genetically defined subtypes of disease and targets for drug development have seen an unprecedented success. With more than 20 genes associated with Parkinson's disease (PD), most of which are highly penetrant and often cause early onset or atypical signs and symptoms, and an increasing understanding of the associated pathophysiology culminating in dopaminergic neurodegeneration, applying the technologies and designs into the field of neurodegeneration seems a logical step. This review describes some of the methods used in oncology clinical trials and some attempts in Parkinson's disease and the potential of further implementing genetics, biomarkers and smart clinical trial designs in this disease area.