Demonstrate safety of gene therapy in neurodegenerative diseases

Demonstrate safety of gene therapy in neurodegenerative diseases


Researchers from the Autonomous University of Barcelona (UAB, northeastern Spain) demonstrated the safety and long-term durability of a gene therapy for neurodegenerative diseases, the University reports.

The researchers carried out a seven-year study in dogs to combat the very rare genetic metabolic disorder called “Sanfilippo A syndrome”, based on a therapy that consists of administering adeno-associated viral vectors directly to the cerebrospinal fluid of the animals.

The UAB recalls that, to date, “never has a gene therapy aimed at the central nervous system of older animals been proven safe and efficient over such a long time”.

The researchers had already managed to cure mice with “Sanfilippo A” syndrome by administering adeno-associated viral vectors that code for sulfamidase directly in the cerebrospinal fluid.

To move the therapy to a clinical application in humans, it was necessary to test its feasibility in larger animals and, to this end, the research team also showed that it was feasible to administer the therapy to the cerebrospinal fluid in dogs through a surgical procedure routine in pediatric neurosurgery, intracerebroventricularly.

The next step, published this December, was to demonstrate that this therapeutic approach is also safe, since it does not present any toxicity and maintains a sustained production of the enzyme sulfamidase in the very long term.

Apply viral vectors adenoasociados as a vehicle for gene therapy directly into the cerebrospinal fluid, which surrounds and protects the brain and spinal cord, “is a promising strategy to achieve the therapeutic agent is spread by both the central nervous system and the peripheral nervous system”, according to the University.

This administration has applications in the treatment of a wide range of neurodegenerative diseases and, in particular, lysosomal accumulation diseases such as mucopolysaccharidosis type IIIA such as “Sanfilippo A Syndrome”.

Although several studies demonstrate the viability of this strategy, there was still no knowledge about its safety and durability of therapeutic gene expression in the very long term, the University adds.