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Published on: January 21, 2018
Cell therapy in patients with heart failure: a comprehensive review and emerging concepts
Roberto Bolli1, Mitesh Solankhi1, Xiang-Liang Tang1
1Institute of Molecular Cardiology, University of Louisville, 550 S. Jackson St., ACB, 3rd Floor, Louisville, KY 40292, USA.
Insights
Cell therapy shows promise for heart failure (HF) patients, with early trials demonstrating an efficacy signal. Further rigorous Phase III trials are needed to confirm the utility of this promising regenerative medicine approach.
Area of Science:
- Regenerative Medicine
- Cardiology
- Cellular Therapy
Background:
- Heart failure (HF) remains a leading cause of morbidity and mortality worldwide.
- Current treatments for HF have limitations, driving the search for novel therapeutic strategies.
- Cell therapy has emerged as a potential regenerative approach for HF patients.
Purpose of the Study:
- To review and summarize the clinical trial evidence for cell therapy in heart failure.
- To evaluate the efficacy and safety of various cell types in HF treatment.
- To identify promising cell types and future directions for cell therapy in HF.
Main Methods:
- Systematic review of Phase I-II clinical trials of cell therapy for heart failure.
- Analysis of efficacy and safety data from studies using different cell types (e.g., mesenchymal stromal cells, cardiosphere-derived cells).
- Evaluation of trial design, endpoints, and long-term outcomes.
Main Results:
- Early-phase trials (Phase I-II) for HF show encouraging results, unlike those for acute myocardial infarction.
- An efficacy signal was observed 6-12 months post-single treatment in both ischemic and non-ischemic cardiomyopathy.
- Mesenchymal stromal cells from various sources show the most promise; embryonic stem cells are unlikely to be viable therapies. No safety concerns have emerged.
Conclusions:
- Cell therapy, particularly with mesenchymal stromal cells, demonstrates potential for treating heart failure.
- The findings support the need for larger, well-designed Phase III trials to establish definitive efficacy.
- Future research should explore optimized delivery methods and allogeneic cell sources for broader clinical application.
Abstract:
This review summarizes the results of clinical trials of cell therapy in patients with heart failure (HF). In contrast to acute myocardial infarction (where results have been consistently negative for more than a decade), in the setting of HF the results of Phase I-II trials are encouraging, both in ischaemic and non-ischaemic cardiomyopathy. Several well-designed Phase II studies have met their primary endpoint and demonstrated an efficacy signal, which is remarkable considering that only one dose of cells was used. That an efficacy signal was seen 6-12 months after a single treatment provides a rationale for larger, rigorous trials. Importantly, no safety concerns have emerged. Amongst the various cell types tested, mesenchymal stromal cells derived from bone marrow (BM), umbilical cord, or adipose tissue show the greatest promise. In contrast, embryonic stem cells are not likely to become a clinical therapy. Unfractionated BM cells and cardiosphere-derived cells have been abandoned. The cell products used for HF will most likely be allogeneic. New approaches, such as repeated cell treatment and intravenous delivery, may revolutionize the field. As is the case for most new therapies, the development of cell therapies for HF has been slow, plagued by multifarious problems, and punctuated by many setbacks; at present, the utility of cell therapy in HF remains to be determined. What the field needs is rigorous, well-designed Phase III trials. The most important things to move forward are to keep an open mind, avoid preconceived notions, and let ourselves be guided by the evidence.
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