Scientists may have developed a gene therapy treatment for the most common form of motor neurone disease (MND).
Cells are lost in the brain
In lab tests on mice the therapy slowed onset and progression of Amyotrophic Lateral Sclerosis (ALS). It also extended life expectancy by 30%.
Writing in the journal Nature, the research team at biopharmaceutical firm Oxford BioMedica stressed the work is at an early stage.
MND affects about 5,000 people in the UK and there are 1,000 new cases a year.
The disease is caused by the death of cells - called motor neurones - that control movement in the brain and spinal cord. There is currently no known cure.
ALS is a form of the disease which affects adults, leading to paralysis and death within five years for most patients.
The new treatment - called MoNuDin - essentially consists of a gene which triggers production of a chemical called a vascular endothelial growth factor (VEGF).
The gene is injected into the muscles, but stimulates VEGF production in the nerve cells of the spine.
ALS has been linked to reduced levels of VEGF in both mice and humans. It is thought that the chemical plays a key role in protecting nerve cells from damage.
Tests on mice showed that a single shot of the new therapy was enough to produce a significant beneficial effect.
Professor Alan Kingsman, Oxford BioMedica chief executive, 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."
Dr Brian Dickie, of the MND Association, welcomed the findings.
He 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."
The Oxford BioMedica team worked on the treatment in collaboration with the Center for Transgene Technology and Gene Therapy in Belgium.