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Genetically targeted clinical trials in parkinson’s disease : Learning from the successes made in oncology

Sjögren, Magnus ; Huttunen, Henri J. ; Svenningsson, Per and Widner, Håkan LU (2021) In Genes 12(10).
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... (More)

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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author
; ; and
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Clinical trials, Genetic targets, Neurodegenerative disorders, Oncology, Parkinson’s disease, Precision medicine
in
Genes
volume
12
issue
10
article number
1529
publisher
MDPI AG
external identifiers
  • scopus:85116142618
  • pmid:34680924
ISSN
2073-4425
DOI
10.3390/genes12101529
language
English
LU publication?
no
additional info
Publisher Copyright: © 2021 by the authors. Licensee MDPI, Basel, Switzerland.
id
058594df-6639-4455-95d8-096142452b91
date added to LUP
2021-10-20 15:10:59
date last changed
2024-06-15 18:30:28
@article{058594df-6639-4455-95d8-096142452b91,
  abstract     = {{<p>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.</p>}},
  author       = {{Sjögren, Magnus and Huttunen, Henri J. and Svenningsson, Per and Widner, Håkan}},
  issn         = {{2073-4425}},
  keywords     = {{Clinical trials; Genetic targets; Neurodegenerative disorders; Oncology; Parkinson’s disease; Precision medicine}},
  language     = {{eng}},
  number       = {{10}},
  publisher    = {{MDPI AG}},
  series       = {{Genes}},
  title        = {{Genetically targeted clinical trials in parkinson’s disease : Learning from the successes made in oncology}},
  url          = {{http://dx.doi.org/10.3390/genes12101529}},
  doi          = {{10.3390/genes12101529}},
  volume       = {{12}},
  year         = {{2021}},
}