El grupo COMET-NANO full Universidad Rey Juan Carlos, led by Professor Santiago Gómez Ruiz, and the Neurometabolism Group of the National Paraplegic Hospital Research Unit, directed by Dr. Carmen María Fernández Martos, have designed a nanostructured system based on porous silica nanoparticles that have been combined with a cocktail of drugs previously studied preclinically by the group led by Dr. Fernández Martos for other neurodegenerative diseases. This mixture consists of the combination of leptin, a hormone that has recently been associated with neuroprotective effects, together with a drug used in the treatment of type II diabetes, pioglitazone, which is also involved in neuroprotective processes.
The study "Design of mesoporous silica nanoparticles for the treatment of Amyotrophic Lateral Sclerosis (ALS) with a therapeutic cocktail based on leptin and pioglitazone”, published in the scientific journal ACS Biomaterials Science and Engineering, shows that the nanomaterial studied is capable of crossing the blood-brain barrier and stopping the disease in a model with mice with ALS, observing that it reaches key tissues associated with the potential treatment of this disease, such as the spinal cord or the motor cortex. "The progression of ALS in the mice studied was much slower, extending their lifespan and showing significant improvements in motor performance, which determined their effectiveness in in vivo neurodegenerative models," says Professor Gómez Ruiz.
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