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Cell therapy in dilated cardiomyopathy: from animal models to clinical trials
C Del Corsso1, A C Campos de Carvalho
1Programa de Terapia Celular e Bioengenharia, Instituto de Biofísica Carlos Chagas Filho, Universidade Federal do Rio de Janeiro, Rio de Janeiro, RJ, Brasil.
Insights
Cell therapies show promise for dilated cardiomyopathy, a severe heart condition. Research in animal models and early clinical trials suggests potential, but challenges remain in translating findings to widespread patient use.
Area of Science:
- Cardiology
- Regenerative Medicine
- Cell Therapy
Background:
- Dilated cardiomyopathy (DCM) is a severe heart condition, often idiopathic, leading to heart failure and sudden death.
- Current treatments for DCM have limitations, highlighting the need for novel therapeutic approaches.
- Cell therapy offers a potential regenerative strategy for damaged heart muscle.
Purpose of the Study:
- To review current data on cell therapy applications in animal models of dilated cardiomyopathy.
- To discuss preliminary findings from clinical trials of cell therapy for patients with DCM.
- To identify challenges and future directions for translating cell therapy research into clinical practice.
Main Methods:
- Summarizing existing literature on cell therapy in genetically manipulated, chemically induced, and infectious agent-induced animal models of DCM.
- Analyzing published clinical trials investigating cell therapy for dilated cardiomyopathy patients.
- Evaluating the impact of specific animal models and cell types (autologous vs. allogeneic) on clinical translation.
Main Results:
- Various cell therapy approaches have been explored in preclinical animal models of DCM with promising results.
- Early-phase clinical trials show feasibility and some potential benefits of cell therapy in DCM patients.
- A significant gap exists between animal model findings and clinical application, partly due to the predominant use of autologous cells in trials.
Conclusions:
- Cell therapy holds potential for treating dilated cardiomyopathy, supported by preclinical and early clinical data.
- The translation of cell therapy from animal models to effective clinical treatments for DCM requires further research.
- Wider adoption of allogeneic cell use in clinical trials may facilitate the translation of genetic DCM models.
Abstract:
Dilated cardiomyopathy can be the end-stage form and common denominator of several cardiac disorders of known cause, such as hypertensive, ischemic, diabetic and Chagasic diseases. However, some individuals have clinical findings, such as an increase in ventricular chamber size and impaired contractility (classical manifestations of dilated cardiomyopathy) even in the absence of a diagnosed primary disease. In these patients, dilated cardiomyopathy is classified as idiopathic since its etiology is obscure. Nevertheless, regardless of all of the advances in medical, pharmacological and surgical procedures, the fate of patients with dilated cardiomyopathy (of idiopathic or of any other known cause) is linked to arrhythmic episodes, severe congestive heart failure and an increased risk of sudden cardiac death. In this review, we will summarize present data on the use of cell therapies in animal models of dilated cardiomyopathies and will discuss the few clinical trials that have been published so far involving patients affected by this disease. The animal models discussed here include those in which the cardiomyopathy is produced by genetic manipulation and those in which disease is induced by chemical or infectious agents. The specific model used clearly creates restrictions to translation of the proposed cell therapy to clinical practice, insofar as most of the clinical trials performed to date with cell therapy have used autologous cells. Thus, translation of genetic models of dilated cardiomyopathy may have to wait until the use of allogeneic cells becomes more widespread in clinical trials of cell therapies for cardiac diseases.
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