Current gene therapy trials for vascular diseases.

Introduction: In the previous gene therapy trials for vascular diseases, safety of the therapies has been demonstrated with some evidence for clinical benefits. In the future, it will be important to also test the potential clinical benefits of the treatments in randomized and controlled trials with sufficient numbers of patients.

Areas Covered: This review covers 15 currently ongoing cardiovascular gene therapy trials that aim to treat coronary artery disease, heart failure and peripheral artery disease. This review summarizes current trials and their main features in the cardiovascular field.

Expert Opinion: In the gene therapy trials for vascular diseases, some limiting factors are still present. The trials have enrolled mainly elderly and severely affected patients who might not have the capacity to respond optimally to the therapies. Also, major cardiac adverse events, major amputations, mortality and other very demanding hard clinical end points have been used in relatively small patient populations. Therefore, there is an urgent need to enroll less severely affected patients and to use more informative surrogate end points in the forthcoming clinical trials.