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Another study involving valproate will be presented by Professor Aurora Pujol, of the Medical and Molecular Genetics Centre- IDIBELL and the Institució Catalana de Recerca i Estudis Avançats (the Catalan Research Agency) in Barcelona, Spain. Dr. Pujol and her team are trying to develop new therapies for X-linked adrenoleukodystrophy (X-ALD). This disease involves a single gene on the X chromosome (the ALD gene) and is the most common inherited single-gene disease involving damage to the myelin sheath, which insulates nerve cells in the brain.
Using a mouse model of the disease, Dr Pujol and colleagues found that a protein called ALDR (very similar to the ALD protein mutated in the disease) could, if over-expressed, compensate for the loss of the ALD protein and prevent the development of neurodegenerative symptoms. This striking prevention of the disease lasted during the whole 2 year life span of the mouse.
"Knowing that a class of drug called histone deacetilase (HDAC) inhibitors was able to stimulate the expression of ALDR in the mouse and in human cell lines, we became interested by the work of Professor Wirth's laboratory on valproate, which belongs to the HDAC class and enters the brain ", said Professor Pujol.
The scientists tested valproate and found that it could indeed induce ALDR gene expression in human cell lines and rat and human brain slices. "We then treated 8 X-ALD patients with oral valproate over 6 months", said Professor Pujol. "We collected samples before treatment, and at 3 and 6 months, and on analysing the amount of ALDR in their white blood cells, we found that half of them showed increased levels."
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