Health
New Treatment Shows Promise for Angelman Syndrome Patients
Ionis Pharmaceuticals has some exciting news! Their new treatment, ION582, has shown great results in a recent clinical trial aimed at helping those with Angelman syndrome.
In the HALOS trial, which involved 51 patients aged 2 to 50, the researchers injected three different doses of ION582 directly into the spinal canal. After six months, most patients reported significant improvements in their symptoms, particularly in areas like communication, cognition, and motor skills.
A whopping 97% of patients who received the medium and high doses saw meaningful changes according to a specific scale that measures how clinicians view these symptoms. That’s a major positive sign!
The results were so promising that Ionis is now planning to move forward with a larger Phase 3 study set to kick off in early 2025. CEO Brett Monia mentioned how enthusiastic they are about collaborating with the wider Angelman syndrome community to further develop this treatment.
Angelman syndrome, caused by mutations in a gene called UBE3A, can lead to lifelong difficulties and reliance on caregivers. Because only the mother’s UBE3A genes are active in specific parts of the brain, ION582 aims to activate the paternal gene to help improve overall functioning.
The first part of the trial has wrapped up, and patients will now continue into the second part to see long-term effects of the doses they received. Researchers hope to gain insights into how this treatment might help with communication, cognition, sleep issues, and even daily living skills over the next four years.
According to Dr. Lynne Bird from the University of California San Diego, these results are particularly encouraging. She pointed out how the improvements seen with ION582 are better than what typically happens with the natural progression of Angelman syndrome.
The HALOS trial findings were shared at a recent Angelman Syndrome Foundation conference, where families were eager to hear about this potential breakthrough in treatment.
