Journal of Stem Cell Research & Therapy

Clinical proof of the safety and efficacy of a new method delivering Ad5FGF-4 to patients with refractory angina

6^th International Conference and Exhibition on Cell and Gene Therapy

March 27-28, 2017 Madrid, Spain

Gabor M Rubanyi

Angionetics Inc., USA

Posters & Accepted Abstracts: J Stem Cell Res Ther

Abstract:

One of the main hurdles of effective gene therapy is delivery of the product. We have developed a practical method of intracoronary artery infusion of Ad5 vector in pigs based on observations that both short term ischemia and nitric oxide augment Ad5 transfection in hearts. The new technique uses two consecutive transient (3 min) coronary artery occlusions by an inflatable balloon catheter combined with intracoronary infusion of nitroglycerin, which increased Ad5Luc expression in the pig heart by more than 100-fold. In order to prove the safety and efficacy of the new delivery method, we have initiated the ASPIRE clinical study. Eleven patients (7 who received a single intracoronary injection of 6x10e9 vp Ad5FGF-4 and 4 with standard of care) completed the study. None of treated patients showed any clinical signs of untoward effects due to the product delivery, and there was no elevation of blood troponin I levels. The treated patients showed a statistically significant (p<0.01) improvement in myocardial perfusion assessed by SPECT eight weeks after product administration. In conclusion, the new Ad5FGF-4 delivery method proved to be safe and well tolerated, and resulted in significant improvement in the primary efficacy endpoint in patients with refractory angina. This pilot study formed the basis for the start of a pivotal Phase III clinical trial in the US.

Biography :

Email: grubanyi@gmail.com