Gene therapy using a modified virus could stop Alzheimer’s disease –study
Characterized by memory loss, confusion and changes in mood or personality, Alzheimer’s disease affects around 520,000 people in the UK alone. It has no cure and current drugs only help treat the symptoms. Now, scientists believe that it could be stopped in its early stages by boosting a gene that clears out destructive sticky plaques in the memory cores of the brain via injection.
Researchers discovered four years ago that people with Alzheimer’s disease do not produce enough amounts of the protein called PGC1-alpha which is essential in preventing the build-up of amyloid beta plaques.
But scientists from the Imperial College London in the UK were able to successfully deliver a gene which produces the plaque-busting protein directly into the brains of mice affected with Alzheimer’s. They were treated with the gene therapy at the early stages of Alzheimer’s disease.
The treated mice did not develop any plaques and performed as well in memory tasks as healthy mice after four months.
Dr. Magdalena Sastre, senior author of the research from the Department of Medicine at Imperial College, said the findings could one day provide a method of preventing the disease, or halting it in the early stages.“In a disease that urgently needs new options for patients, this work provides hope for future therapies,” she added.
A harmless, modified virus, which has been edited to include the gene, was used to infect the brain cells and rewrite their genetic codes so they would produce more of the plaque-fighting protein. Injections were given in the hippocampus and cortex of the brain, which are responsible for memory formation and orientation and are the first to be affected by Alzheimer’s disease.
Professor Nicholas Mazarakis, co-author of the study from the Department of Medicine at Imperial added: “Scientists harness the way lentivirus infects cells to produce a modified version of the virus that delivers genes into specific cells.
The team believes that injections of the gene would be most beneficial in the early stages of the disease, when the first symptoms appear.
Dr. Doug Brown, director of research and development, Alzheimer’s Society, said: “This research takes a new approach to tackling the underlying causes of Alzheimer’s disease – using a technique called gene therapy to interrupt the production of amyloid protein, one of the key hallmarks of Alzheimer’s.
“So far, potential treatments that directly target amyloid build-ups in the brain have mostly had disappointing results in clinical trials, whereas this study could pave the way for a new plan of attack.”
Dr. David Reynolds, chief scientific officer at Alzheimer’s Research UK, said that the study sets a foundation for exploring gene therapy as a treatment strategy for the disease. “But further studies are needed to establish whether gene therapy would be safe, effective and practical to use in people with the disease,” he added.
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