Phase II study of itMSC (stem cells) in heart failure demonstrates IV delivery is feasible- CardioCell

CardioCell has announced data from its Phase IIa clinical trial "Safety and Efficacy of Intravenous Infusion of Ischemia-Tolerant Allogeneic Mesenchymal Stem Cells in Patients With Non-ischemic Cardiomyopathy" using itMSC stem cells in heart failure patients. Data illustrates statistically significant improvement in 6-minute walk test, quality-of-life scores as assessed by Kansas City Cardiomyopathy Questionnaire (KCCQ) and favorable immune modulatory benefits. This represents the first clinical trial to study the effects of intravenous (IV) administration of ischemia-tolerant mesenchymal stem cells (itMSCs) in patients with chronic heart failure (HF).

The study demonstrates that the systemic effects of intravenously delivered itMSCs can be measured and is associated with improved patient outcomes. Results show that an IV injection strategy is safe and well-tolerated. There are no major differences in any of the safety endpoints, including clinical events like all-cause mortality, all-cause hospitalization and adverse events, and there are no alterations in pulmonary function, liver function or arrhythmias. In addition, IV itMSC injections exhibit improvements in several clinical-efficacy endpoint measurements, including statistically significant improvement in 6-minute walk test; KCCQ Clinical Summary score and trend to improvement in KCCQ Functional Status score.

The study also demonstrates that intravenously administered itMSCs suppress inflammation, which is noteworthy because inflammation is believed to importantly contribute to HF progression. There was a statistically significant reduction in natural killer (NK) cells, the magnitude of which correlated with the magnitude of improvement in left ventricular ejection fraction (LVEF). Overall, there was no difference in cardiac structure and function. In an exploratory data analysis before the 90-day crossover, there was a statistically significant improvement in the left ventricular end systolic and diastolic volumes in the itMSC-treated groups, whereas this was not seen in the placebo-treated group.

It was previously assumed that large amounts of stem cells must be delivered directly to the myocardium to improve patient outcomes. However, this delivery mechanism � either via catheter or by surgery � is not practical for such chronic conditions as HF, where multiple treatments may be needed. The results of this study suggest that IV itMSC administration could potentially provide a more practical alternative that offers health benefits. Results were presented at the European Society of Cardiology (ESC) Congress.