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Proof-of-Concept Trials for Lung Stem Cell Therapy

¹ Novartis Institutes for Biomedical Research, Cambridge, Massachusetts

Correspondence and requests for reprints should be addressed to David M. Rodman, M.D., Translational Medicine Head, Respiratory Novartis Institutes for Biomedical Research, 400 Technology Square, Cambridge, MA 02139. E-mail: david.rodman{at}novartis.com

ABSTRACT

Proof-of-concept (PoC) trials are an important component of the new therapeutic development paradigm, contributing to the goal of accelerated progression from bench to bedside. Developing a simplified PoC strategy for lung stem or progenitor cell therapy can be helpful in focusing initial efforts and identifying methodologic hurdles in humans at a relatively early stage of development. In this article, the use of lung radiation injury as a model of epithelial injury and regeneration is proposed as a potential PoC strategy. Strengths of the approach include well-understood biology, predictive preclinical and translational models, a tractable human trial design, and the potential to improve outcome in an area of high unmet medical need. While significant hurdles remain, this approach could provide the first interpretable results within 4 years of the decision to proceed, thus greatly accelerating the translation of lung stem cell therapy to humans.

Key Words: radiation • fibrosis • inflammation