Epilepsy Drug Valproate May Treat Spinal Muscular Atrophy

Filed in archive Drugs, Vaccines and Therapeutics , Genomics, Proteomics and Bioinformatics on May 8, 2006

In laboratory as well as preliminary clinical trials, researchers from the Institute of Genetics at the University of Cologne found that valproate, a drug currently being used to treat epilepsy, may have potentials as therapy against spinal muscular atrophy (SMA).

Valproate seems to be able to stimulate the expression of the survival motor neuron (SMN) gene, a gene which is underexpressed in SMA patients. Increased levels of SMN proteins were observed in the blood as well as brain tissues of patients treated with the drug. It is however still unclear whether there is an equivalent increase in the SMN expression in motor neurons, thus increasing muscle strength and alleviateing SMA symptoms.

Professor Brunhilde Wirth said: "The long-term outcome could be both improved therapy to enable a better quality of life for SMA patients, and also the introduction of neonatal screening so that therapy could be started before the first symptoms appear."

Dr Fred Kavalier, of the British Society of Human Genetics, said: "This work shows that it may be possible to influence the behaviour of genes with drugs.

Not yet a treatment, but a small step. Read the full article from BBC News: Self-repair gene therapy promise.