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.

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