Approaches for Effective Clinical Application of Stem Cell Transplantation

Curr Transplant Rep. 2018;5(3):244-250. doi: 10.1007/s40472-018-0202-0. Epub 2018 Aug 2.


Purpose of review: This review highlights problems related to translation of advanced therapy medicinal products (ATMPs) from bench to bedsite. Regenerative medicine within the current regulatory frame reveals common hitches in the course of development, translation, and clinical application. This paper suggests outlining a path from the few examples of successfully approved vs unsuccessful advanced therapies.

Recent findings: In the multitude of ongoing studies, few of them achieved positive results with a final treatment available to patients; this result was possible due to multidisciplinary teams working together from the beginning of the development and during the hard route to standardization and clinical application.

Summary: The root of success of an advanced therapy requires not only the inescapable scientific and biological knowledge but also requires several contributions as regulatory, ethical, medical, and bio-engineering expertise, from the real beginning. A strong scientific rationale and an integrated network of expertises would contribute to a successful investment of available resources in advanced therapy medicinal products and to a greater confidence in future medicine.

Keywords: ATMP; Cell therapy; Clinical trial; Gene therapy; Stem cell; Translational medicine; Valley of death.

Publication types

  • Review