Amyotrophic Lateral Sclerosis (ALS), the most prevalent form of motor neuron disease, causes adult-onset, progressive motor neuron degeneration in the brain and spinal cord
Oxford BioMedica 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 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 USA.
The research was led by 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.
Brian Dickie, director of research development at the MND Association, said, "These findings reflect optimism among 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 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."
