Planning advanced therapy trials for Huntington's Disease: ethical considerations.

Huntington's Disease (HD) is a relentlessly progressive genetic disorder characterised by cognitive, behavioural and physical impairments. The lack of effective treatments, early onset of symptoms and high rates of suicide reflect a disease of significant human suffering. The ethical imperative to pursue disease modifying treatments with gene and stem cell therapies - collectively referred to as advanced therapeutic medicinal products (ATMPs) - is therefore sound. Recent gene therapy trials have generated international attention and widespread hope in the HD community, however gene and stem cell therapies are fraught with ethical challenges and complexity. Genetic discrimination, trial design challenges (especially the use of surgical placebos) and the risk for therapeutic misconception need careful consideration whilst broader concerns relating to justice and equity must be recognised by researchers and funders alike. This paper will provide those involved in gene therapy trials and other advanced therapy research a comprehensive overview of the ethical issues.