VEGF Gene Therapy Improves Survival In Murine ALS Model

NEW YORK (Reuters Health) - Delivery of the vascular endothelial growth factor (VEGF) gene with a lentiviral vector can safely improve the survival of mice with amyotrophic lateral sclerosis (ALS), according to a report published in the May 27th issue of Nature.

"VEGF treatment increased the life expectancy of ALS mice by 30% without causing toxic side effects, thereby achieving one of the most effective therapies reported in the field so far," lead author Dr. Mimoun Azzouz, from Oxford BioMedica in the UK, and colleagues note.

Recent reports have implicated low levels of VEGF in the pathogenesis of ALS in mice and humans, suggesting that VEGF has a neuroprotective role. In the current study, the investigators decided to explore the potential therapeutic effects of VEGF gene therapy in a murine model of ALS.

A single IM injection of VEGF-expressing lentiviral vector delayed the onset and slowed progression of ALS in test mice. Moreover, this therapy was still useful even when delayed until after paralysis onset.

"We believe that this approach may have potential as a safe and practical treatment for many of the motor symptoms of human ALS," the researchers state.

Source: Nature 2004;429:413-417. [ Google search on this article ]

MeSH Headings: Biological Therapy : Genetic Engineering : Genetic Techniques : Investigative Techniques : Therapeutics : Gene Therapy : Endothelial Growth Factors : Analytical, Diagnostic and Therapeutic Techniques and Equipment