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Delivery of Cardioactive Therapeutics in a Porcine Myocardial Infarction Model
Published on: February 10, 2023
Tailored therapeutics for cardiomyopathies
Athanasios Bakalakos1, Emanuele Monda1,2, Perry Mark Elliott3
1Institute of Cardiovascular Science, University College London, London, UK.
Insights
Cardiomyopathy, a complex heart muscle disorder, traditionally focuses on symptom management. Emerging therapies aim to target the root genetic causes, offering new hope for patients with these progressive heart conditions.
Area of Science:
- Cardiology
- Genetics
- Molecular Biology
Background:
- Cardiomyopathy encompasses diverse heart muscle disorders with varied causes and mechanisms.
- Current management primarily addresses symptoms and prevents complications like heart failure, with limited success in treating progressive dysfunction.
- Few etiology-targeted therapies exist in cardiology, making cardiomyopathies a prime area for therapeutic innovation.
Purpose of the Study:
- To review promising therapeutic approaches for cardiomyopathy.
- To discuss novel strategies targeting the underlying causes of heart muscle disorders.
- To explore the potential impact of these innovative therapies on patients and their families.
Main Methods:
- Review of current literature on cardiomyopathy pathogenesis and treatment.
- Analysis of emerging pharmacological and genetic therapies.
- Discussion of cellular and molecular pathways involved in disease progression.
Main Results:
- Identification of several promising therapeutic strategies for cardiomyopathy.
- Highlighting the potential for therapies to address the disease at its source by targeting genetic causes.
- Exploration of novel or repurposed pharmacological agents and genetic variant modification.
Conclusions:
- Cardiomyopathies present a unique opportunity for developing targeted therapies due to their often monogenic origins.
- Advances in understanding molecular pathways are paving the way for innovative treatments.
- New therapeutic approaches hold promise for significantly improving patient outcomes and quality of life.
Abstract:
The term cardiomyopathy is used to describe a large family of complex heart muscle disorders of diverse aetiology and pathophysiology. For decades, the management of individual cardiomyopathy subtypes has focused primarily on the management of symptoms and the prevention of disease-related complications, such as heart failure and sudden cardiac death. Treatment of progressive myocardial dysfunction has relied on conventional evidence-based heart failure therapies, with variable success. In contrast to other areas of medicine, cardiology is characterized by few aetiology-targeted therapies, but cardiomyopathies offer an ideal model for innovation because, in many individuals, the disorder has a monogenic cause, the expression of which is modified by complex genetic mechanisms, comorbidities and lifestyle. Elucidation of the complex cellular and molecular pathways that result in downstream tissue phenotypes has led to the investigation of new or repurposed pharmacological agents and, in parallel, therapies that modify or mitigate the effects of causative genetic variants, offering the prospect of targeting the disease at its source. In this Review, we describe some of the most promising therapeutic approaches in cardiomyopathy and discuss their potential effect on the lives of patients and relatives.
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