Scientists may have discovered the first treatment for Huntington’s disease. Brain disorders Until then, there was no effective treatment.
Researchers at the University of London (UCL) have published positive results for the global clinical trial AMT-130 for new gene therapy.
Developed by Dutch biotechnology company Uniqure, the therapy was first tested in the people of Huntington. Researcher.
Huntington’s disease is a rare, inherited neurodegenerative disorder that gradually damages neurons (neurons) in the brain, as defined by national neuropathy and stroke.
This disease usually occurs between the ages of 30 and 50. HTT genetriggers cells to create a protein called huntitin, which can cause damage to the brain.
Patients may experience a combination of movement, dementia, and psychiatric symptoms. Involuntary jerky movements and stiffness are common and can be difficult to walk, speak and swallow.
Cognitive symptoms include memory loss and issues with focus and decision-making. Emotional and behavioral According to multiple medical sources, changes may also be visible.
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According to UCL, the disease usually leads to disability and death within 20 years of onset of neurological symptoms.
AMT-130 is a one-time gene therapy injected into the brain, introduced through new functional DNA. Neurosurgery. This drug produces less harmful protein, the protein, hazardous.
In a three-year clinical trial at UCL, 29 Huntington patients were found to be Experimental drugs. Those who received a higher dose of AMT-130 experienced 75% less disease progression compared to those who received only standard of care, a study press release reports.
Disease progression was measured by a unified Huntington’s Disease Rating Scale, which assessed motor, cognitive and functional capabilities.
Another component of this study was the measurement of neurofilament photoprotein (NFL) found in the spinal fluid of Huntington patients as more neurons were damaged.
Participants who took the experimental medication were found to have less protein at the end of the trial, but levels were typically increased by 20% to 30% over that period.
This drug was generally found to be “manageable” and “well tolerated.” Safety ProfileThe release said.
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“These groundbreaking data are the most compelling evidence in the field to date, highlighting the disease-correcting effect of Huntington’s disease, where urgent needs persist, says Professor Sarah Tabritzi of UCL’s Huntington’s Disease Research Center.
“For patients, the AMT-130 may maintain daily functioning, so make them longer and meaningfully slower. Progression of the disease. ”
Based on the results, Professor Ed Wilde, the lead researcher at UCL and UCLH’s UCL Huntington’s Disease Centre Test Site, said AMT-130 was “probably” the first approved treatment to slow down Huntington’s disease, which he calls “a world-changing thing.”
“My patients on the trial have been stable over time in ways I’m not used to seeing with Huntington’s disease. One of them is my only medically retired Huntington’s disease patient who has been able to return to work.”
Dr. Ernest Lee Murray, a Board Certified Neurologist At Jackson Madison County General Hospital in Jackson, Tennessee, we focused on the reasons for years of difficulty in treating Huntington.
“It was a challenge to identify specific treatment goals,” Murray, who was not involved in the new study, told Fox News Digital. “There’s always the challenge of intersecting Blood-brain barrier.”
Most new therapies use animal models in early clinical trials where it is difficult to replicate Huntington’s complexity, he added.
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Current treatment for the disease focuses only on the main symptoms, Lee noted – “The uncontrollable movements that these patients experience, called chorea.”
“The fact that we have potential medicine This not only treats symptoms, but also changes the course of the actual illness.
The study is “moving in the right direction,” but there are some limitations, Lee said.
“It involves a very small cohort of patients and is in a very early stage of potential development,” he said. “We need to see whether larger blind studies confirm the potential effectiveness of treatment and monitor potential side effects.”
The results of the trial will be presented next month at the HD Clinical Research Council in Nashville, Tennessee.
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The company plans to submit an application to the FDA early next year to accelerate drug approval, Uniqure confirmed with Fox News Digital.
“If that happens, we need to work hard to make it available to everyone who needs it. Effective treatment “Wild has been added to the list.
