TY - JOUR
T1 - Genetically targeted clinical trials in parkinson’s disease
T2 - Learning from the successes made in oncology
AU - Sjögren, Magnus
AU - Huttunen, Henri J.
AU - Svenningsson, Per
AU - Widner, Håkan
N1 - Publisher Copyright:
© 2021 by the authors. Licensee MDPI, Basel, Switzerland.
PY - 2021/9/28
Y1 - 2021/9/28
N2 - 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.
AB - 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.
KW - Antiparkinson Agents/therapeutic use
KW - Clinical Trials as Topic
KW - Humans
KW - Molecular Targeted Therapy/methods
KW - Parkinson Disease/drug therapy
KW - Precision Medicine/methods
UR - http://www.scopus.com/inward/record.url?scp=85116142618&partnerID=8YFLogxK
U2 - 10.3390/genes12101529
DO - 10.3390/genes12101529
M3 - Review
C2 - 34680924
AN - SCOPUS:85116142618
SN - 2073-4425
VL - 12
SP - 1
EP - 8
JO - Genes
JF - Genes
IS - 10
M1 - 1529
ER -