Gene therapy may soon provide the ever-elusive Alzheimer's cure

In what promises to be a possible breakthrough in treating Alzheimer's, a team of scientists that includes many Greeks has developed a gene therapy technique that virtually arrests the progression of the disease in laboratory mice. The UK-based team is led by Dr. Magdalena Sastre and Prof. Nicholas D. Mazarakis of Imperial College London, who spoke to the Athens-Macedonian New Agency (ANA) about the research in an interview published on Wednesday.

Their findings were recently published in the Proceedings of the National Academy of Sciences of the United States of America (PNAS) journal, with Greek scientists Loukia Katsouri and Ioanna Eleftheriadou listed among its authors, in addition to Mazarakis and other colleagues.

Mazarakis explained that the treatment works by reducing generation of the amyloid plaque that destroys brain cells and causes the disease. The technique uses a harmless virus known as a 'vector' to insert a gene coding for the protein PGC-1α directly in to the brains of laboratory mice, targeting the memory centres and hippocampus where plaque builds up. This was shown to significantly reduce generation of amyloid-β, the plaque that destroys the brain, and associated neuronal loss.

The research, funded by the European Research Council and Alzheimer's Research UK, is still at an early stage but shows great promise as a potential treatment for this constantly spreading neurodegenerative disease.

"Alzheimer's is the most common neurodegenerative disorder, affecting more than 45 million people worldwide. There are currently no treatments that cure or stop the progression of the disease. Most of the existing treatments focus on drugs taken orally, which have not been proved effective. Our research shows that gene therapy could be a real alternative solution to oral drugs, provided it is administered in the early stages of the disease," Mazarakis said.

Unlike current treatments, which address some of the symptoms and seek to slow the disease's progression, the new gene therapy actually addresses the causative factors and neuronal damage that results in Alzheimer's.

Laboratory mice 

The scientists injected laboratory mice suffering from the initial stages of Alzheimer's with the new gene, using a virus vector. The mice subjected to the therapy developed much less amyloid plaque than control mice also suffering from the disease during that period, while they performed equally well as healthy, 'wild' mice in memory tests. They also did not present any loss of brain cells in their hippocampus.

The PGC-1α gene is a transcriptional regulator of genes involved in energy metabolism and acts by reducing the main enzyme involved in amyloid plaque production. Research has shown that both exercise and resveratrol (in pill form) increase levels of PGC-1α in the body.

The next step is to proceed to trials on humans, in the hope that the gene treatment will be useful in the early stages of Alzheimer's disease, when the symptoms first appear, Mazarakis said, noting that trials on humans could begin relatively soon, in three to five years, provided the necessary support for the programme is found from research councils and companies.

"There will first have to be more studies in order to assess how effective the treatment is on laboratory animals after the disease appears," he said, while noting that the results to date have been "very encouraging".

Mazarakis said that a similar gene therapy treatment using a virus vector developed for Parkinson's disease, Prosavin®, has already been tested on 15 patients with advanced Parkinson's with very encouraging results.

"A second definitive clinical trial is being planned for next year, with the use of an improved virus vector," he added, noting that the gene inserted in Prosavin® helps increase levels of dopamine in the brain.

"There are already studies indicating that PGC-1α could be used as a cure for Parkinson, if inserted into the brain early," the professor added.

The original paper can be found at the following link: http://www.pnas.org/content/early/2016/10/05/1606171113.abstract

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