According to a recent press release from uniQure, the first round of observations from a trial investigating AMT-130 for Huntington’s disease (HD) have been released. As of now, four patients are enrolled in the Phase I/II trial, two of which have been treated with the investigational gene therapy. uniQure is happy with the initial results, and they’re hoping that AMT-130 will be able to help Huntington’s patients in the future.
About Huntington’s Disease
Firstly, it’s important to understand what Huntington’s disease is. It’s a rare, neurological condition characterized by a progressive loss of physical and mental functioning. Symptoms typically manifest in the 30s and 40s, and affected individuals live for about 15 to 20 years afterwards. These symptoms fall into three categories: uncontrolled motor symptoms, emotional problems, and cognitive decline. The first category includes twitching, shrugging, issues with swallowing, and problems with coordination and walking. The next category sees symptoms like anxiety, depression, mood swings, impulsive behavior, and changes in behavior. Lastly, cognitive decline includes forgetfulness, poor decision-making, and issues with retaining or learning information.
These are all the result of mutated HTT gene, which is responsible for the instructions for the huntingtin protein. This protein builds up before binding to neurons, causing them to progressively die off. This mutation is passed down in an autosomal dominant pattern. Unfortunately, there is no cure or treatment to reverse the effects of Huntington’s disease; treatment is symptomatic.
This Phase I/II trial is being conducted in the United States, with four HD patients included so far. Two have received AMT-130 while the others underwent a sham surgery. The update released by uniQure gave us data from one year of follow-up:
- Neurofilament light chain (NfL) returned to baseline levels in patients treated with AMT-130
- NfL remained at a steady level in patients in the control group
- The safety profile remained consistent
- AMT-130 appears to be well-tolerated
- No serious adverse events
Another trial update is expected in the second half of 2022, and it will focus on a low-dose cohort that includes ten participants. The complete efficacy and safety data will arrive sometime during the first half of 2023. Hopefully, it continues to be positive, as Huntington’s disease patients are in a need of a viable treatment option.
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