A new treatment that proves to slow the progression of Huntington’s disease marks a breakthrough for a condition that has long been considered untreatable. Huntington’s is a rare, inherited neurodegenerative disorder that gradually deteriorates a person’s physical and mental abilities, often leading to death within 10 to 30 years of onset. Until now, treatments have only managed symptoms, rather than altering the disease’s course.
The research team, including Sung, tested a new treatment called AMT-130 on people who were in the early stages of HD. The treatment works by halting the part of the body’s gene production responsible for producing the toxic huntingtin protein. By targeting the messenger RNA that carries instructions from DNA to produce this protein, AMT-130 prevents its formation without altering the DNA itself — a key safety advantage over other genetic therapies.
After three years, those who received the higher dose showed a 75 percent slower disease progression compared to those who did not receive the treatment. The treatment helped trial participants maintain more of their movement, thinking and daily functioning abilities without causing significant side effects.
This was not a clinical trial, it was a safety study of only 11 treated. No controls.
CEO of UniQure already dumped all his stock options.
It was a phase I\II trial. Dafuq u on about?
Phase I\II determine efficacy and safety profiles.
In this case they are proceeding with a new cohort where the administration will be concurrent with immune suppression to counteract inflammation in the pos-op phase.
They will likely apply for a phase III after since this is an orphan drug.