Genzyme completes gene therapy trial enrolment
Genzyme Biosurgery, a division of Genzyme Corp., has completed enrolment in its Phase I clinical trial of an investigational gene therapy treatment for severe peripheral arterial disease. The trial is part of Genzyme Biosurgery's efforts to develop new treatments for cardiovascular disease using gene and cell therapy.
In the randomised, double-blind, placebo controlled trial, 28 patients with critical limb ischemia and no other treatment options received an engineered form of the HIF-1alpha gene to promote new blood vessel growth and improve circulation in their limbs. HIF-1alpha has been shown in pre-clinical studies to turn on the expression of proteins associated with the body's response to tissue ischemia.
HIF-1alpha is a transcription factor that has been shown to activate a cascade of proteins associated with blood vessel formation. This approach contrasts with pro-angiogenic therapies employing a single protein, such as a VEGF isoform, given as a gene or as a recombinant protein. Because the Genzyme-engineered version of HIF-1alpha turns on the expression of many angiogenic proteins - including all known VEGF isoforms - Genzyme Biosurgery believes it may produce a more robust and longer-lasting pro-angiogenic effect than gene therapies based on a single growth factor. Genzyme holds an exclusive license for the use of HIF-1alpha in gene therapy for cardiac and vascular diseases from Johns Hopkins University.
In addition to the peripheral arterial disease trial, Genzyme Biosurgery is currently enrolling 20 patients in a Phase I randomised, double-blind, placebo controlled clinical trial in which a genetically engineered form of HIF-1alpha is administered as an adjunct to coronary artery bypass surgery to help grow vessels in areas of the heart that are not suitable for surgical revascularisation. The primary objective is to evaluate the safety of the therapy, but preliminary bioactivity outcomes will also be evaluated.
Genzyme Biosurgery is also preparing to begin a new clinical trial later this year using autologous cell therapy to strengthen heart muscle damaged during a heart attack, with the aim of stopping or slowing the subsequent progression to heart failure. Details of this trial are expected to be finalised later.