Alzheimer’s Symptoms Reduced in Gene Therapy Trial

Researchers at Imperial College London used a modified virus to deliver the PGC-1 gene to brain cells in animal studies. Previous studies suggest the PGC-1 alpha gene — responsible for regulating sugar and fat metabolism in the body — could prevent the formation of amyloid-beta peptide – a precursor to Alzheimer’s.

Dr Magdalena Sastre, from the department of medicine at Imperial and senior author, said: “Although these findings are very early they suggest this gene therapy may have potential therapeutic use for patients. There are many hurdles to overcome, and at the moment the only way to deliver the gene is via an injection directly into the brain. However this proof of concept study shows this approach warrants further investigation.”

The modified virus used in the experiments was a lentivirus vector, commonly used in gene therapy. It was injected into the mice’s hippocampus and cortex, the first two areas to develop amyloid plaques in Alzheimer’s. The hippocampus is responsible for short-term memory and orientation, while the cortex is responsible for long-term memory, reasoning, thinking and mood, according to the Labarotary News website.

The research showed mice that received the gene treatment had less amyloid plaques than those that did not. No loss of brain cells in the hippocampus was observed in mice that received the injection. Scientists, unexpectedly, also noticed a reduced number of glial cells in these mice. This is important as these cells release inflammatory substances that further cell damage in Alzheimer’s sufferers.

Although these injections were given directly to the area of treatment, researchers hope that there is a way to turn these trials into human treatment. Dr Sastre said: “In a disease that urgently needs new options for patients, this work provides hope for future therapies.”