MADRID, 20 Abr. (EUROPA PRESS) –
An international study, led by a group of Spanish researchers, has demonstrated the feasibility and safety of introducing gene therapy (viral vectors) into the brain of primates in a focal and non-invasive manner, which could be a breakthrough for patients with neurodegenerative diseases such as Parkinson’s.
The results obtained in parallel with Parkinson’s patients open the door to new scenarios and represent a starting point for future trials with application in other neurodegenerative diseases and brain tumors. “In this study we have shown that the delivery of gene therapy is safe, without visible damage to the brain,” says one of the authors of the study, Dr. José Ángel Pineda-Pardo.
The new findings have just been published by the scientific journal ‘Science Advances’, in an investigation led by Professor José A. Obeso, whose main authors have been researchers from HM CINAC (from HM Puerta del Sur in Móstoles), Dr. Javier Blesa and doctor José Ángel Pineda-Pardo and belonging to the Network Biomedical Research Center on Neurodegenerative Diseases (CIBERNED), dependent on the Carlos III Health Institute (ISCIII). Scientists from the University of Kyoto (Japan) have also participated in the study.
This new non-invasive therapy is produced thanks to the temporary opening of the blood-brain barrier by means of low-intensity ultrasound (LIFU) in regions relevant to patients with Parkinson’s disease (substantia nigra and striatum).
The viral vectors were injected through a peripheral vein and entered the brain by opening the blood-brain barrier. “It is important to highlight that, as it is a focal opening, the viral vectors only reach the region in which they have acted, avoiding interaction with the rest of the brain structures, which will ultimately allow the design of personalized therapeutic strategies “, explains Dr. Pineda-Pardo.
In particular, intravenous administration of type 9 adenovirus to brain regions affected in Parkinson’s disease was achieved, with no negative side effects or tissue damage observed. The study suggests that this administration could be generalized to other viral vectors used in gene therapy.
This new discovery is especially implicated in Parkinson’s disease but the researchers say “it could be applied to other diseases such as Alzheimer’s disease, Huntington’s disease or Amyotrophic Lateral Sclerosis”. “It must be taken into account that there are numerous molecules that could reach the brain more efficiently with this type of procedure,” says Dr. Blesa.
