Oxford, UK: 27 May 2004 - Oxford BioMedica (LSE:OXB.L), the leading gene therapy
company, announces today that the Company's scientists, in collaboration with
scientists from VIB (the Flanders Interuniversity Institute for Biotechnology)
in Leuven, Belgium, have published the results of pioneering research that could
lead to a treatment for patients with Amyotrophic Lateral Sclerosis (ALS), the
most prevalent form of motor neuron disease. The results, published in today's
Nature magazine (Volume: 429, Issue: 6990 pp: 413-417), are based on preclinical
studies and show that using a novel gene therapy approach both onset and
progression of disease is slowed and that life expectancy is extended by 30%,
thereby achieving one of the most effective therapies reported in the field to
date.
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 patients. There is currently no known cure for motor neuron disease, a
condition that affects approximately 100,000 people in Europe and the US.
The research was led by Dr Mimoun Azzouz, Director of Neurobiology at Oxford
BioMedica, in collaboration with the VIB department for Transgene Technology and
Gene Therapy in Belgium. The novel gene therapy product, MoNuDin, delivers a
vascular endothelial growth factor (VEGF) gene, a neuroprotective factor, using
the Company's proprietary LentiVector system. The product is injected into
muscle and mediates its therapeutic effect within the nerve cells of the spine.
It has previously been reported that reduced levels of VEGF predispose mice and
humans to ALS, but this is the first assessment of its therapeutic potential.
These results show that a single injection of a VEGF-expressing lentiviral
vector into various muscles delayed onset and slowed progression in an animal
model of ALS. Treatment was also found to increase the life expectancy of mice
by 30 per cent.
Dr Brian Dickie, Director of Research Development at the Motor Neurone Disease
Association, said: "These findings reflect current optimism amongst researchers
that gene therapy represents a viable strategy for the treatment of ALS and
other neurodegenerative diseases, overcoming problems of access of drugs to the
central nervous system, which can occur with more conventional approaches to
treatment."
Commenting on the results, Oxford BioMedica's Chief Executive, Professor Alan
Kingsman, said: "Although these results published in Nature are still at a
preclinical stage, the data suggests that VEGF gene therapy could provide an
effective treatment for ALS, a debilitating disease that leads to premature
death and for which there is no current cure and current treatments are
ineffective. These results also bode well for our spinal muscular atrophy
product, which employs a similar technology." |
