Gene Therapy Destroys Brain Cancer

Gene Therapy Destroys Brain CancerGene Therapy Destroys Brain Cancer

Technologists have developed a nanoparticle transport system for gene delivery. The system can wipe out brain gliomas, which cause a deadly form of cancer.

With the system, nanoparticles are packed with genes related to a specific enzyme that converts a pro-drug termed ganciclovir into a chemical that can wipe out glioma cells. Glioma is a tumor that begins in the spine or in the brain (the brain is the most common site.) The risk associated with this type of cancer is high, with a survival rate of less than 10%. The exact cause is unknown, although there is a probable genetic connection.

Therapies for glioma are based on delivering specific genes, depending upon the type of tumor and its location in the brain. The main delivery system involves using viruses. However, this mechanism can be unreliable.  

The new process is called gene therapy. It is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery, reports

Most types of gene therapy use DNA to encode a therapeutic gene that, when introduced into the body via a vector aims to replace the mutated gene causing the cancer.

In place of viruses, the new technology uses biodegradable nanoparticles. The objective is to use something more accurate and less harmful. Being biodegradable, the particles breakdown into harmless components and then are removed from the body.

In new studies, researchers have used an encoding gene known as HSVtk. This gene uses an enzyme that transforms the compound ganciclovir into a compound that is toxic to brain cancer cells. For the drug delivery system, a polymer ‘PBAE 447’ was found to be the optimal one for the basis of the nanoparticles.

The studies to date relate to experiments conducted using rats, where the rats were developed with brain cancer. In the studies, the life of the rats was extended. The nanoparticles entered the rats through infusion, via an injection into the site of the tumor. Over eight days a measurable reduction of the tumor was seen.

The next phase is to use the non-viral nanomedicine method on other animals, with a view to conducting trials on humans at a later stage.

Although gene therapy is a promising treatment option for a number of diseases, the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures.

The study was carried out at the US National Institute of Biomedical Imaging and Bioengineering). The research has been reported in the journal ACS Nano.