End-stage heart failure is the common result of an acute or chronic injury to the heart. Currently there is no effective treatment for these patients and they will subsequently require a left ventricle assist device or a heart transplant. Now researchers are spotlighting a large study showing that cell therapy might one day be the answer for these patients.
Amit Patel, who is the director of Cardiovascular Regenerative Medicine at the University of Utah School of Medicine, and his team published their work earlier this week in the journal The Lancet.
Cell therapy, which has raised its share of doubt over its use in the clinic for heart failure, finally appears to show promise in this study. "For the last 15 years everyone has been talking about cell therapy and what it can do. These results suggest that it really works," said Patel.
The study, known as IxCELL-DCM, included 126 patients spanning 31 centers who received cell therapy treatment in a double-blinded Phase IIb clinical trial. Patients that received the therapy had a type of end-stage heart failure called ischemic heart failure.
In the study, investigators extracted two types of stem cells (mesenchymal stem and M2 macrophages cells) from the bone marrow of individual patients. They selected and grew up these cells in the lab and administered the two cell types into the heart using a minimally invasive catheter-based procedure.
The two cell types, combined in a therapy dubbed ixmyelocel-T, were chosen for their beneficial cardiac remodeling, increase in heart size and anti-inflammatory behavior--all of which were demonstrated in preclinical work.
At regular time points, up until 12 months after the treatment, patients appeared to show few side effects as well as fewer deaths than the control placebo group. Heart failure related hospitalizations were also lower and there was a 37% reduction in overall cardiac events in the treatment group compared to the control group, which arrived as statistically significant.
Of note, the benefit from the cell therapy did not extend beyond one year (the duration of the study). There was also no, or very few, differences in heart function using conventional imaging such as echocardiography between groups, as well as exercise tolerance tests which can unmask patients that have improved cardiac function due to the therapy, and those that do not.
Based on the lower cardiac events in the treatment group this could be seen as a breakthrough for heart failure treatment since, to date, all previous cell therapy that includes a single stem cell population has failed. They will continue with Phase III clinical trials.
"This is the first trial of cell therapy showing that it can have a meaningful impact on the lives of patients with heart failure," says Patel.