Project 171430

Ultrasound-targeted gene therapy for heart failure

171430

Ultrasound-targeted gene therapy for heart failure

$662,450
Project Information
Study Type: Other Drug_Development
Therapeutic Area: Cardiovascular
Research Theme: Biomedical
Disease Area: heart failure
Data Type: Canadian
Institution & Funding
Principal Investigator(s): Leong-Poi, Howard M
Co-Investigator(s): Connelly, Kim A; Parker, Thomas G
Institution: Unity Health Toronto
CIHR Institute: Circulatory and Respiratory Health
Program: Operating Grant
Peer Review Committee: Cardiovascular System - B: Heart and Circulation
Competition Year: 2008
Term: 5 yrs 0 mth
Abstract Summary

Despite advances in medical and surgical therapies, the morbidity and mortality of patients with chronic coronary artery disease and heart failure remains high. For patients who fail to improve with conventional treatments and whose heart function continues to deteriorate, novel therapies to improve cardiac pumping function are needed. Promising new techniques involves either genes, or special cells called "progenitor" cells. These cells are derived from the blood or bone-marrow, and have the ability to develop into new vessels or heart muscle cells. We have developed novel ultrasound techniques using microbubbles that can be targeted to the heart, and can be used to deliver gene or cell therapy, without the use of direct injections. A non-invasive means of delivery would facilitate an easy and safe method for repeated delivery in patients. This present research, if successful, will lead to a non-invasive method of delivering novel treatments to promote new vessel formation, limit heart muscle death and improve cardiac function in patients with severe symptomatic heart failure.

Research Characteristics

This project includes the following research characteristics:

Digital Health
Knowledge Translation Focus
Safety Focus
Biomarker Endpoints
Study Justification

"This present research, if successful, will lead to a non-invasive method of delivering novel treatments to promote new vessel formation, limit heart muscle death and improve cardiac function in patients with severe symptomatic heart failure."

Novelty Statement

"We have developed novel ultrasound techniques using microbubbles that can be targeted to the heart, and can be used to deliver gene or cell therapy, without the use of direct injections."

Methodology Innovation

developing ultrasound-targeted microbubbles for non-invasive delivery of gene and cell therapy to the heart

Keywords
Chronic Ischemia Gene Delivery Heart Failure Progenitor Cells Targeted Microbubbles Therapeutic Angiogenesis