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Preclinical target validation using patient derived cells

Patel, Dhavalkumar (2015) Preclinical target validation using patient derived cells. Preclinical target validation using patient derived cells, 14 (3). pp. 149-150. ISSN 1474-17761474-1784


Although the annual number of new drug approvals is
trending upwards, the number of ‘first-in-class’ therapies
has remained relatively constant — often fewer than 10
per year. For such new medicines for ‘pioneer targets’,
attrition in Phase II proof-of-concept clinical studies
remains the biggest hurdle1, in large part because the
target–disease associations derived from the currently
dominant cell-line or animal preclinical models of disease
often do not translate into clinical efficacy.
It is increasingly appreciated that the use of disease
models based on human samples is critical both to
increase our understanding of pathophysiology and
to reduce clinical attrition. However, securing regular
access to well-annotated samples from patients is challenging
to organize and raises ethical issues. Here, we
propose that these issues can most easily be addressed
by creating an open-source partnership.

Item Type: Article
Date Deposited: 03 May 2016 23:45
Last Modified: 03 May 2016 23:45


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