The Bellvittge Biomedical Research Institute (IDIBELL) has signed a licensing agreement with the Spanish biotechnology company Minoryx of a patent for the treatment of X-linked adrenoleukodystrophy, a rare serious neurodegenerative disease which has no effective treatment.
The Neurometabolic Diseases group led by ICREA researcher Aurora Pujol, has developed an animal model to study this disease. Studies using this model have shown that the patented product, a repositioned drug, may be effective against X-linked adrenoleukodystrophy.
The obtaining of the patent and the license has been managed by the Technology Transfer Office of IDIBELL. The license represents an opportunity not only to get this invention into the market and patients, but also to initiate a partnership of trust between the company and IDIBELL in developing new solutions to improve the quality of life of patients with adrenoleukodystrophy.
Hereditary disease
The X-linked adrenoleukodystrophy is an inherited disease that destroys the myelin (white matter that coats the nerves) at the central or peripheral nervous system and causes hormonal changes. Patients have a genetic mutation that impedes the properly degradation of very long chain fatty acids within cells. This causes the accumulation of this type of fat in plasma and tissues, especially in the brain and adrenal gland, causing irreversible damage.
This is a rare disease that affects only male subjects, although female carriers may also experience some symptoms. Its incidence is one case per 17,000 men. It is the most common type of leukodystrophy and the main peroxisome disorder. The peroxisome is the part of the cell responsible for eliminating toxic products. The disease became famous in 1992 with the film Lorenzo's oil, starring by Nick Nolte and Susan Sarandon.
Pioglitazone acts on the nuclear receptor PPAR-gamma and to a lesser extent, on PPAR-alpha. These are proteins that regulate the storage of fatty acids and the glucose metabolism and also enhance peroxisome proliferation in cells.
More studies
The drug has been successful in animal experiments, but more studies will be needed before the product can be applied to X-linked adrenoleukodystrophy patients. In this sense, the signing of the license agreement with Minoryx is an important step to use the drug for this purpose in the future.
Minoryx is a biotechnology company located in the TecnoCampus Mataró-Maresme, Barcelona, dedicated to develop therapeutic solutions to treat rare diseases, especially those affecting children and adolescents. Minoryx has two complementary strategies: drug repositioning and identification of a new drugs generation of pharmacological chaperones, through a proprietary technology platform.
The development of the patent transferred to Minoryx has been possible thanks to the support of the Spanish Ministry of Economy and Competitiveness, Instituto de Salud Carlos III and European regional development funds (ERDF). The ELA Foundation, which has a small stake in the patent, has also collaborated.