Therapeutic strategies for prion disease: a practical perspective

Brian S. Appleby; Allyson Connor; Han Wang

doi:10.1016/j.coph.2018.11.006

Therapeutic strategies for prion disease: a practical perspective

Brian S. Appleby
, Allyson Connor
, Han Wang

Research output: Contribution to journal › Review article › peer-review

19 Scopus citations

Abstract

Human prion diseases are usually rapid neurodegenerative illnesses that are invariably fatal. Despite several clinical trials, no effective treatment has been discovered in humans. Although prior clinical trials have not been successful, they provided information that is vital for the formation of future clinical trials. Among these findings is the realization that there are several prion disease-specific aspects that must be considered when conducting clinical trials. The rarity, rapidity, and clinical heterogeneity of prion disease affect study enrollment and the ability to measure treatment effects. In addition to affecting results, study methodology may also influence study enrollment. In this review, we explore several challenges to conducting clinical trials in prion disease and suggest some practical considerations.

Original language	English (US)
Pages (from-to)	15-19
Number of pages	5
Journal	Current Opinion in Pharmacology
Volume	44
DOIs	https://doi.org/10.1016/j.coph.2018.11.006
State	Published - Feb 2019
Externally published	Yes

Bibliographical note

Funding Information:
The authors would like to thank the staff of the National Prion Disease Pathology Surveillance Center (NPDPSC) and CJD Foundation for all their hard work and support of individuals affected by prion disease. This work was funded in part by the Stivison Fund for CJD Research . The NPDPSC is funded by the Centers for Disease Control and Prevention ( NU38CK000480 ).

Publisher Copyright:
© 2018 Elsevier Ltd

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10.1016/j.coph.2018.11.006

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title = "Therapeutic strategies for prion disease: a practical perspective",

abstract = "Human prion diseases are usually rapid neurodegenerative illnesses that are invariably fatal. Despite several clinical trials, no effective treatment has been discovered in humans. Although prior clinical trials have not been successful, they provided information that is vital for the formation of future clinical trials. Among these findings is the realization that there are several prion disease-specific aspects that must be considered when conducting clinical trials. The rarity, rapidity, and clinical heterogeneity of prion disease affect study enrollment and the ability to measure treatment effects. In addition to affecting results, study methodology may also influence study enrollment. In this review, we explore several challenges to conducting clinical trials in prion disease and suggest some practical considerations.",

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T2 - a practical perspective

AU - Appleby, Brian S.

AU - Connor, Allyson

AU - Wang, Han

N1 - Funding Information: The authors would like to thank the staff of the National Prion Disease Pathology Surveillance Center (NPDPSC) and CJD Foundation for all their hard work and support of individuals affected by prion disease. This work was funded in part by the Stivison Fund for CJD Research . The NPDPSC is funded by the Centers for Disease Control and Prevention ( NU38CK000480 ). Publisher Copyright: © 2018 Elsevier Ltd

PY - 2019/2

Y1 - 2019/2

N2 - Human prion diseases are usually rapid neurodegenerative illnesses that are invariably fatal. Despite several clinical trials, no effective treatment has been discovered in humans. Although prior clinical trials have not been successful, they provided information that is vital for the formation of future clinical trials. Among these findings is the realization that there are several prion disease-specific aspects that must be considered when conducting clinical trials. The rarity, rapidity, and clinical heterogeneity of prion disease affect study enrollment and the ability to measure treatment effects. In addition to affecting results, study methodology may also influence study enrollment. In this review, we explore several challenges to conducting clinical trials in prion disease and suggest some practical considerations.

AB - Human prion diseases are usually rapid neurodegenerative illnesses that are invariably fatal. Despite several clinical trials, no effective treatment has been discovered in humans. Although prior clinical trials have not been successful, they provided information that is vital for the formation of future clinical trials. Among these findings is the realization that there are several prion disease-specific aspects that must be considered when conducting clinical trials. The rarity, rapidity, and clinical heterogeneity of prion disease affect study enrollment and the ability to measure treatment effects. In addition to affecting results, study methodology may also influence study enrollment. In this review, we explore several challenges to conducting clinical trials in prion disease and suggest some practical considerations.

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UR - https://www.scopus.com/pages/publications/85057445368#tab=citedBy

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DO - 10.1016/j.coph.2018.11.006

M3 - Review article

C2 - 30508662

AN - SCOPUS:85057445368

SN - 1471-4892

VL - 44

SP - 15

EP - 19

JO - Current Opinion in Pharmacology

JF - Current Opinion in Pharmacology

ER -

Therapeutic strategies for prion disease: a practical perspective

Abstract

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