Promising Results With Gene Therapy for Refractory Angina, a Phase I/IIa Clinical Trial

Published 7 August 2017

Technology platform ATMP
Institute National Virus Vector Laboratory
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Adenoviral intramyocardial gene therapy

A clinical Phase I/IIa trial in Kuopio University Hospital Heart Center in collaboration with EATRIS Finland Centers National Virus Vector Laboratory (NVVL) from University of Eastern Finland and Turku PET Center shows promising results. An adenoviral gene transfer of novel vascular endothelial growth factor family member VEGF-D∆N∆C was injected to the hibernating myocardium of refractory angina patients. The hibernating but viable myocardium to target the therapy to was identified with PET perfusion imaging and electromechanical catheter mapping methods.

The number of patients enrolled to this study was 30, randomized 5:1 ratio to treatment and control groups, respectively. The therapy was shown to be safe, feasible and well tolerated. In addition, statistical significant increase in the myocardial perfusion reserve in the treatment area was detected both 3 and 12 months after the injections. Further, a new potential biomarker, plasma Lp(a), was identified which may be used to screen the patients who benefit the most of this new therapy.

An article describing the study and comprehensive one year follow up results is published in the European Heart Journal (Hartikainen J, Hassinen I, Hedman A, Kivelä A, Saraste A, Knuuti J, Husso M, Mussalo H, Hedman M, Rissanen TT, Toivanen P, Heikura T, Witztum JL, Tsimikas S, Ylä-Herttuala S.  Adenoviral intramyocardial VEGF-D∆N∆C gene transfer increases myocardial perfusion reserve in refractory angina patients. A phase I/IIa study with one year follow-up. European Heart Journal 2017; 0: 1-9. doi: