Drug intelligence / Profile preview

cultured autologous skeletal myoblast cells

Development stage
Discontinued
Lead developer
Sanofi
Modality
Cell Therapies
Administration
Parenteral
01

Overview

Cultured autologous skeletal myoblast cells is an investigational autologous cell therapy developed with involvement from Genzyme Corporation for the treatment of ischemic heart failure and ischemic cardiomyopathy. The therapy involves harvesting muscle progenitor cells (myoblasts) from a patient's own skeletal muscle via biopsy, followed by ex vivo expansion under Good Manufacturing Practice (GMP) conditions for approximately three weeks. The resulting cells are suspended in a medium (typically Dulbecco's modified Eagle's medium supplemented with albumin) and injected directly into and around post-infarction myocardial scar tissue, often during coronary artery bypass surgery. The biological goal is to improve regional contractility and left ventricular ejection fraction (LVEF) by populating the scarred myocardium with functional muscle cells. This therapy was evaluated in the landmark Phase 2 MAGIC (Myoblast Autologous Grafting in Ischemic Cardiomyopathy) trial, which was the first randomized, double-blind, placebo-controlled study of its kind. Although the trial demonstrated the feasibility of the procedure, it failed to show a significant improvement in global heart function compared to placebo and raised safety concerns regarding an increased risk of ventricular arrhythmias, necessitating the use of implantable cardioverter-defibrillators (ICDs) in treated patients.

Other names
autologous skeletal myoblastsskeletal myoblast transplantationmyoblast autologous grafting

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