José López Barneo
Professor of Physiology at the University of Seville and head of the Cellular Neurobiology and Biophysics team at the Institute of Biomedicine of Seville (IBiS)
The work is very interesting because it demonstrates that by applying ultrasound in a focused way, the blood-brain barrier (BBB) can be temporarily opened and drugs (and viruses for gene therapy) can be delivered directly into the brain. This technology has previously been tested in mice, but is now being developed in primates and in three patients with Parkinson's disease (PD). In addition, precise measurements are carried out to demonstrate that the applied molecules reach the predetermined target and that the procedure is safe.
This technique represents an important step forward in the development of gene therapy for application in humans suffering from neurodegenerative diseases. Normally, the BBB isolates the brain from blood circulation. Therefore, in gene therapy trials where therapeutic viruses need to be introduced into the brain, they are administered by injection with a needle. In the studies that have been carried out so far, the injection causes significant tissue (brain tissue) damage, which limits its use in patients. This limitation disappears completely with the non-invasive therapy described here.
The work is of high technical quality and is mostly carried out in Spain and led by Spanish scientists. Before its routine application in patients, further controlled trials will be needed to confirm that the technique has no relevant side effects.