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CSIC has developed and brought to preclinical stage a novel non-invasive pharmacological treatment for Niemann Pick disease Type C (NPC) and other lysosomal storage disorders where cholesterol accumulates in the brain.
Niemann Pick type C is a fatal disease caused by mutations in the cholesterol transport protein Npc1. The accumulation of cholesterol, particularly evident in lysosomes, leads to progressive neurodegeneration and to cognitive and psychiatric alterations in NPC patients. Currently, the only treatment approved for NPC is the iminosugar Miglustat, which prevents accumulation of gangliosides and delays neurological symptoms but does not cure the disease having serious side effects.
We have demonstrated that oral treatment with Efavirenz (EFV), which activates the cholesterol-degrading enzyme CYP46, prevents brain cholesterol accumulation and pathology in a mouse model for NPC (Npcnmf164mice).
We propose EFV treatment could be also suitable for many other neurological lysosomal storage disorders where brain cholesterol accumulation is a hallmark.
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Pre-clinical stage
The Spanish National Research Council (CSIC) is the largest public institution dedicated to research in Spain and the third largest in Europe. Belonging to the Spanish Ministry of Science, Innovation and Universities through the Secretary of State for Universities, Research, Development and Innovation, its main objective is to develop and promote research that will help bring about scientific and technological progress, and it is prepared to collaborate with Spanish and foreign entities in order to achieve this aim.
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