Biological therapies for inherited diseases: social and bioethical considerations. Hemophilia as an example

Abstract

Introduction: In hemophilia, advanced therapies are warranted from a conceptual and methodological standpoint. Current advanced therapy strategies are centered on the use of adeno-associated viral vectors, although problems related to immunogenicity and hepatotoxicity still remain.

Areas covered: Future clinical trials will have to scrupulously observe international bioethical standards in terms of patient selection, particularly children. Patient recruitment rates are likely to remain low due to the stringent exclusion criteria usually imposed on the trial population regarding their hepatic and immunological markers and the presence of viral coinfection; and to the existence of an optimal palliative treatment.

Expert opinion: Accordingly, the results obtained are likely to be of low statistical significance, which could hinder their application to clinical practice. Another important issue is the degree to which society embraces these new emerging therapies. The unfamiliarity of society with these new methods, together with the many unresolved questions about them that remain, may threaten their acceptance not only by society at large but also by health-care professionals, which would limit their translational application to clinical practice.

Keywords:

• advanced therapies
• cell therapy
• gene therapy
• hemophilia
• social and bioethical

Declaration of interest

The author has been supported by funding from a grant from the Victoria Eugenia Royal Hemophilia Foundation, and Octapharma S.A. A Liras is Principal Researcher in a preclinical project, but not a clinical trial, with adipose- and decidua-derived mesenchymal stem cells and gene/cell therapy protocols for the treatment of hemophilia. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Notes

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