Genetically Targeted Clinical Trials in Parkinson's Disease: Learning from the Successes Made in Oncology
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 gene...
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Published in | Genes Vol. 12; no. 10; p. 1529 |
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Main Authors | , , , |
Format | Journal Article |
Language | English |
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Abstract | 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. |
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AbstractList | 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. |
Author | Sjögren, Magnus Svenningsson, Per Widner, Håkan Huttunen, Henri J |
AuthorAffiliation | 3 Department of Neurology, Karolinska University Hospital, 171 77 Stockholm, Sweden; per.svenningsson@ki.se 2 Department Health Sciences, Umeå University, 907 36 Umeå, Sweden 4 Department of Neurology, Lund University Hospital, 221 00 Lund, Sweden; hakan.widner@med.lu.se 1 Herantis Pharma Plc, Bertel Jungin Aukio 1, 02600 Espoo, Finland; henri.huttunen@herantis.com |
AuthorAffiliation_xml | – name: 4 Department of Neurology, Lund University Hospital, 221 00 Lund, Sweden; hakan.widner@med.lu.se – name: 1 Herantis Pharma Plc, Bertel Jungin Aukio 1, 02600 Espoo, Finland; henri.huttunen@herantis.com – name: 2 Department Health Sciences, Umeå University, 907 36 Umeå, Sweden – name: 3 Department of Neurology, Karolinska University Hospital, 171 77 Stockholm, Sweden; per.svenningsson@ki.se |
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SubjectTerms | 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 |
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Title | Genetically Targeted Clinical Trials in Parkinson's Disease: Learning from the Successes Made in Oncology |
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