A new type of gene therapy, which would wipe out the symptoms of Parkinson’s in mice, has been approved for a phase 2 trial in people
It is hoped that one-and-done gene therapy will prevent the death of dopamine neurons in the brain, which cause Parkinson’s disease.
Currently, patients are treated with drugs to stop their symptoms, but this can cause a lot of side effects, including problems with their mental health.
The one-and-done gene therapy is being developed by drug company Gilead Sciences.
It is currently in a phase 2 trial for patients with a specific genetic mutation, which makes the brain cells die off prematurely.
There are around 20,000 people in the UK with Parkinson’s disease, and about 20,000 in the US.
In a separate study, a different gene therapy was approved by the US Food and Drug Administration (FDA) in August.
The drug works by removing a protein called Nox2 from a protein that is normally made by the immune system.
This prevents the body from attacking viruses and bacteria, and has also been used to treat some types of cancer.
The same company, Genentech, which made the first gene therapy to treat HIV, also holds the patent for the drug that caused the latest trial to be approved.
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The Parkinson’s disease charity Parkinson’s UK has written to the NHS in England and Wales urging them to support the drug trials.
The charity said that the UK should take a leading role in this area.
“We urge the government to ensure that this is not a one-off trial,” the letter said.
“The trial will take place at a time when the UK has the capacity to support this kind of research. We are very concerned that the government is not providing the resources to support this trial in England and Wales.”
Dr Susan Crowe, a neuroscientist at King’s College London, said that if a trial could prove that the drug worked in people, it would be “huge”.
“If it’s a successful trial it will be an important development,” she told the BBC.
“It will be the first drug to target the virus, but it won’t be the first one to treat Parkinson’s disease.
“We need to make sure we get the drugs into the clinic. This is something that has to be given to people, because it will help many, many people.”
But she said that if it failed, the research would be “very disappointing”.
Meanwhile, a group of charities that supports people with Parkinson’s have called for a ban on the import of the drug, saying that it should be made available only for use in the UK.
Professor Peter White, from Parkinson’s UK, said: “We need a controlled trial to make sure that the drug works. If it doesn’t, then we will have to think very carefully about what other approaches are available.”
A spokesman for the UK Medical Research Council said: “The decision to approve the trials in the US is final and we have no plans to reconsider the decision.
“The work of the US scientists is in line with the best available evidence