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Gene Transfer for Ischemic Heart Failure in a Preclinical Model
Published on: May 15, 2011
Non-viral vectors for gene therapy: clinical trials in cardiovascular disease
Pinak B Shah1, Douglas W Losordo
1Division of Cardiology, Caritas St. Elizabeth's Medical Center Boston, Massachusetts 02135, USA.
Insights
Therapeutic angiogenesis using gene therapy offers a promising approach to treat patients with severe vascular disease. Clinical trials explore delivering angiogenic factors to stimulate new blood vessel growth, improving blood supply to ischemic tissues.
Area of Science:
- Cardiovascular Medicine
- Regenerative Medicine
- Gene Therapy
Background:
- Growing population with end-stage symptomatic coronary and peripheral vascular disease.
- Patients often lack mechanical revascularization options and remain symptomatic despite medical therapy.
- Insufficient blood supply to muscle due to diseased vessels causes angina and critical limb ischemia.
Purpose of the Study:
- To explore therapeutic angiogenesis as a strategy to increase blood supply to ischemic tissues.
- To review clinical trial experiences with non-viral gene therapy for therapeutic angiogenesis.
- To address limitations in patients with disabling myocardial ischemia and peripheral vascular disease.
Main Methods:
- Investigating molecular mechanisms of vascular development.
- Utilizing animal models to demonstrate the efficacy of vascular development mediators.
- Delivering angiogenic cytokines (e.g., VEGF, FGF) via non-viral gene therapy (naked plasmid or viral vector).
Main Results:
- Animal studies show mediators can produce new capillaries in ischemic tissue.
- Non-viral gene therapy is being evaluated in human clinical trials.
- Focus on improving blood supply to target tissues to relieve symptoms.
Conclusions:
- Therapeutic angiogenesis holds potential for patients with limited treatment options.
- Non-viral gene therapy represents a viable delivery method for angiogenic factors.
- Clinical trials are crucial for validating this approach in myocardial ischemia and peripheral vascular disease.
Abstract:
The population of patients with end-stage symptomatic coronary and peripheral vascular disease is ever-expanding. Many of these patients no longer have options for mechanical revascularization, and despite maximal medical therapy, they remain physically limited due to angina or critical limb ischemia. The fundamental problem in these patients is insufficient blood supply to muscle due to severely diseased conduit vessels to the target tissue. Therefore, it seems logical that increasing the blood supply to ischemic tissue will relieve symptoms. One potential means to achieving this goal is via therapeutic angiogenesis. The molecular mechanisms behind vascular development are being elucidated, and animal models have shown that mediators of vascular development can be harnessed to produce new capillaries in ischemic tissue. These mediators include cytokines such as vascular endothelial growth factor (VEGF) and fibroblast growth factor (FGF). Angiogenic cytokines can be delivered in several forms including recombinant protein or via gene delivery as a naked plasmid or via viral vector. This chapter will describe the clinical trial experience to date with delivery of non-viral gene therapy for therapeutic angiogenesis in humans with disabling myocardial ischemia and peripheral vascular disease.
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