VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model

Mimoun Azzouz (), G. Scott Ralph, Erik Storkebaum, Lucy E. Walmsley, Kyriacos A. Mitrophanous, Susan M. Kingsman, Peter Carmeliet and Nicholas D. Mazarakis ()
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Mimoun Azzouz: Oxford BioMedica plc
G. Scott Ralph: Oxford BioMedica plc
Erik Storkebaum: University of Leuven
Lucy E. Walmsley: Oxford BioMedica plc
Kyriacos A. Mitrophanous: Oxford BioMedica plc
Susan M. Kingsman: Oxford BioMedica plc
Peter Carmeliet: University of Leuven
Nicholas D. Mazarakis: Oxford BioMedica plc

Nature, 2004, vol. 429, issue 6990, 413-417

Abstract: Abstract Amyotrophic lateral sclerosis (ALS) causes adult-onset, progressive motor neuron degeneration in the brain and spinal cord, resulting in paralysis and death three to five years after onset in most patients1. ALS is still incurable, in part because its complex aetiology remains insufficiently understood. Recent reports have indicated that reduced levels of vascular endothelial growth factor (VEGF), which is essential in angiogenesis and has also been implicated in neuroprotection2,3,4, predispose mice and humans to ALS5,6. However, the therapeutic potential of VEGF for the treatment of ALS has not previously been assessed. Here we report that a single injection of a VEGF-expressing lentiviral vector into various muscles delayed onset and slowed progression of ALS in mice engineered to overexpress the gene coding for the mutated G93A form of the superoxide dismutase-1 (SOD1G93A) (refs 7–10), even when treatment was only initiated at the onset of paralysis. VEGF treatment increased the life expectancy of ALS mice by 30 per cent without causing toxic side effects, thereby achieving one of the most effective therapies reported in the field so far.

Date: 2004
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DOI: 10.1038/nature02544

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