Gene therapy - Times article

This is from today's Times. Even though the headline is about Alzheimer's the company are running clinical trials for pwp.

"People could be treated for Alzheimer’s disease long before its symptoms become apparent using a technique that has delivered promising results in other brain disorders.

Experts told a neuroscience conference in London this week that there was a good prospect for a genetic therapy for Alzheimer’s after success against Parkinson’s and Huntington’s, two less common brain diseases.

Tiny inflections in several dozen genes twist the biology of the brain in ways that leave some people with a 90 per cent chance of developing Alzheimer’s by the time they reach old age, while others have a 90 per cent chance of avoiding it.

Bradley Hyman, professor of neurology at Harvard Medical School, said that reaching into the skull and tweaking these strands of DNA could substantially improve the odds and might even become a way of treating people who already had the disease.

This would involve either injecting patients with harmless viruses packed with new genetic instructions, or using compounds known as antisense, which silence the chemical messages emitted from unwanted genes.

“It’s an incredibly exciting technology,” Professor Hyman told the World Neuroscience Innovation Forum at the Francis Crick Institute in London. “It really feels like it’s maturing and it’s about to burst on to the scene.”

Steven Paul, chief executive of Voyager Therapeutics, said that his team had had good results in preliminary human trials using the method to treat Parkinson’s. “It’s still early days, but we can now produce this in practice,” he said. “We’re also encouraged by the fact that the little viruses that we use to get these genes in the brain or silence these genes are getting better and better. We now have capsids [protein shells of viruses] that can get across the blood-brain barrier 100 or 200 times better than the current crop of capsids. It’s an exciting time.”

Six months ago a group of scientists at Imperial College London reported that they had prevented mice from developing an equivalent of Alzheimer’s by ferrying the PGC-1alpha gene into their brains.

Magdalena Sastre, a senior author on the study, said that the team planned to try other kinds of gene therapy that could moderate malfunctions in the brain’s immune system that have been implicated in dementia. She said that getting the same method to work in humans could be tricky and there was a danger of side-effects.

Professor Hyman suggested that the technique could “dial up” the ApoE-e2 gene, which seems to have a protective effect. His experiments in mice have suggested that this could reverse the disease even after its signs had begun to appear in the brain.

Another important obstacle will be cost. Gillian Leng, deputy chief executive of the National Institute for Health and Care Excellence, which approves medicines for the NHS, said that the expense of gene therapies was “interesting”.

She hinted, however, that Nice might be prepared to pay more than usual for the treatments to help to bring them into use."

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