A British-led gene therapy that rewires the circuits of the brain has been hailed as a “game changer” for the millions suffering from Parkinson’s disease.
Scientists say a tiny, one-time injection deep in the brain could be the start of a “new era” in the treatment of the disease, which affects over 160,000 patients in the UK.
Trials have shown the novel treatment appears to “reset” faulty wiring, helping patients move properly again - and early evidence suggests it may also slow the disease itself.
“It’s one of the most exciting things we’ve done,” said Dr Alexandria “Zandy” Forbes, boss of London biotech firm MeiraGTx.
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“We saw using AI that our therapy had changed the brain in a way that allows people to move but may also slow degeneration. It really is a breakthrough.”
The therapy, known as AAV-GAD, has been tested in three groundbreaking early-stage studies in Parkinson’s patients.
Fourteen patients were involved in the most recent, which delivered a missing gene directly into the brain to reprogramme nerve cells - calming the tremors and stiffness that make everyday life so difficult.
The patients were divided into three groups - five got a high dose, five a low dose, and four received a sham (dummy) procedure.
Parkinson's disease affects over 160,000 patients in the UK
|GETTY
After six months, those on the high dose improved by an average of 18 points on the gold-standard Parkinson’s movement scale. A change of just five to ten points is considered medically meaningful.
By contrast, the low-dose and placebo groups saw no significant improvement.
Lead investigator Dr Ali Rezai, a neuroscientist and Director of the US Rockefeller Neuroscience Institute, West Virginia, said: “These safety and outcome results are excellent.
“The extent of motor score improvements in patients who received the high-dose treatment combined with significant quality of life improvement measures are very encouraging for both patients and physicians.”
He said it was the first clear sign that a single gene therapy could make a measurable difference to real-world function.
After the initial mobility gains were recorded, scientists sent brain scans for independent analysis by artificial intelligence experts at University College London.
This confirmed that the circuits of the brain had been physically altered - indicating that the therapy had rebalanced brain networks that go haywire in Parkinson’s.
LATEST DEVELOPMENTS:“We provided the data from the brain scans of the patients before, during and after treatment,” said Dr Forbes.
“The group from UCL and (AI company) Hologen were able to use their AI to look into the brain and showed that we had physically changed the circuitry of the Parkinson’s patients that we treated.”
The AI analysis showed calming signals spreading to other brain regions, including the substantia nigra - the area where Parkinson’s begins and where dopamine-producing cells die off.
Parkinson’s robs people of movement by killing off the dopamine-producing cells that control the body’s coordination. Without dopamine, the brain’s subthalamic nucleus - a region that regulates movement - goes into overdrive, sending out bursts of the chemical glutamate that cause tremors and stiffness.
Instead of topping up dopamine, MeiraGTx’s therapy flips the chemistry by delivering a gene called GAD (glutamic acid decarboxylase) into that overactive brain region.
The gene makes an enzyme that converts glutamate into GABA, the brain’s natural calming signal. This restores balance in the motor circuits and smooths out movement.
“We quietened down the circuitry, modifying the physiology of the brain, and we saw improvement in movement,” said Dr Forbes.
Fourteen patients were given a missing gene directly into the brain to reprogramme nerve cells - calming the tremors and stiffness that make everyday life so difficult
|GETTY
The AI data also suggested that the therapy does more than help movement - it appears to positively affect connections in areas linked to mood and memory, raising hopes that it could help the depression and cognitive problems that often accompany Parkinson’s.
This isn’t the first success for MeiraGTx. The company previously worked with Moorfields Eye Hospital in London to restore sight to blind children using a similar gene therapy approach.
“We treated children who were all born blind due to the lack of a single functional gene. When we delivered a correct copy of the gene to the retina of these young children by a one-time injection, each child treated gained vision, transforming their lives,” said Dr Forbes.
Now the team at MeiraGTx, based in Shoreditch, east London, is preparing for a large-scale global trial to confirm the Parkinson’s results.
The study will enrol people who no longer respond to medication and for whom there is no effective long-term medical therapy.
If successful, this gene therapy treatment could become the first to both restore movement and even slow the progression of the world’s fastest-growing neurological disease.
“These are really important stories,” said Dr Forbes. “This is science that has the potential to literally change these patients’ lives, and provides hope to millions of patients for whom other effective treatments are not available.”
Parkinson’s affects around 168,000 people in the UK, and numbers are rising.
The results mean MeiraGTx’s therapy has now been given fast-track status by the US regulator, the FDA, clearing the way for a late-stage trial with the potential to support approval.
“This could mark the start of a new era,” said Dr Forbes.
“For the first time, we’re seeing marked improvement for this condition along with a physiological change in the brain from a one-time gene therapy treatment.”
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