New Way to Access the Brain with Gene Therapy

It's very difficult to treat brains conditions using drugs or gene therapy because the brain is surrounded by a barricade of cells called the blood brain barrier, which is designed to keep unwanted things out, which unfortunately includes many drugs and gene-therapy treatments. But Boston researcher Ferenc Jolesz has found a way to temporarily open up tiny holes in the barrier using ultrasound waves. The researchers injected into the bloodstream tiny bubbles of protein which are already used by doctors to improve the quality of ultrasound images. They then focused the beam of ultrasound waves on a specific area of the brain. The ultrasound bursts the bubbles and generates small shock waves which open up the blood brain barrier nearby, allowing large drug molecules to get in. They have even shown that a genetically-engineered virus which can be used for gene-therapy, when injected into the blood stream, gets only into those parts of the brain that have received the treatment. The next step, they say, will be to package the virus inside the protein bubbles so that it is delivered more efficiently to the parts of the brain that need it.