From Cleveland.com
By Megan Sims
Doctors have announced the first successful treatment for Huntington’s disease, a devastating inherited condition that combines symptoms of dementia, Parkinson’s and motor neurone disease.
The groundbreaking gene therapy has slowed disease progression by 75% in patients, meaning the decline typically seen in one year would now take four years, potentially giving patients decades of improved quality of life, the BBC reports.
“We never in our wildest dreams would have expected a 75% slowing of clinical progression,” said Professor Sarah Tabrizi, director of the University College London Huntington’s Disease Centre, who led the UK part of the trial alongside Professor Ed Wild.
The treatment involves a complex 12- to 18-hour brain surgery, where a specially modified virus delivers therapeutic DNA deep into the brain. This DNA produces microRNA that intercepts and disables instructions for building the mutant huntingtin protein that kills brain cells in people with the disease.
Results from the 29-patient trial in the U.K. and U.S., showed significant improvements three years after surgery, with measurements based on cognition, motor function and daily life abilities.
The treatment also appears to be saving brain cells, BBC noted, as levels of neurofilaments in spinal fluid, indicators of dying neurons, were lower than at the start of the trial.
For Jack May-Davis, who carries the faulty gene that killed his father Fred at age 54, the breakthrough offers new hope.
“This absolutely incredible breakthrough has left me overwhelmed,” the 30-year-old barrister’s clerk told the publication. “It does allow me to think my life could be that much longer.”
The therapy is expected to be expensive and requires highly specialized neurosurgery. UniQure, the company behind the treatment, plans to apply for a license in the U.S. in early 2026, with discussions in the U.K. and Europe to follow, BBC said.
Professor Wild told the publication that the therapy “should last for life” since brain cells are not regularly replaced like other body tissues. He described the magnitude of the effect as “breathtaking.”
Approximately 75,000 people have Huntington’s disease across the U.K., U.S. and Europe, with hundreds of thousands more carrying the mutation that will eventually cause the disease, BBC noted.
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Brethren, this is truly miraculous.
HOW TO BE SAVED
Thank You JESUS!!
MARANATHA!
WOW AMEN!!
My family suffered from this disease. I had a great uncle who shot himself back when they had no clue as to what this was. My mom’s dad and his sister died from this. I had a cousin and my mother too. Then it took my brother. It’s a horrible disease. You can’t imagine what this disease does to you and how it makes the infected act. It’s like a horror movie. I seen my mom try to commit suicide twice. I could write a novel with all I and my family went through but the days only so long.
God Bless
I am SO sorry that your family members had to go through this nightmare. I posted this in hopes that some who are being affected by Huntington’s might see this breaking news and share it with friends and family members.
I have a friend and former co-worker who died of Huntington’s Disease. But I am a little leary of the Mrna treatment. I do not think she would have wanted anything that would have changed her God given DNA.
True, Julie – that is the other side of the coin!!!!
Maybe also a cure for Demoncratitis or Trump hate syndrome? Just sayin’…….
You’re just sayin’ right my brother in Christ!!!
Yes! I’m an RN. Huntington’s is right up there with Alzheimer’s! Very horrible disease! I truly pray this is an actual medical breakthrough!!🙏🙏🙏🙏 I’m truly sorry Wolf for what your family has gone through!!😪😪😪🙏🙏🙏💔