Arctic Therapeutics research suggests preventative strategy against HCCAA

The research provides insights into the potential of N-acetylcysteine (NAC) as a treatment for HCCAA, a disease with no currently approved therapeutic options. The study highlights how NAC may reduce disease-associated biomarkers and slow disease progression. The research provides insights into the potential of N-acetylcysteine (NAC) as a treatment for HCCAA, a disease with no currently approved therapeutic options. The study highlights how NAC may reduce disease-associated biomarkers and slow...

REYKJAVIK, Iceland, 02/04/2025 (informazione.news - comunicati stampa - salute e benessere)

The research provides insights into the potential of N-acetylcysteine (NAC) as a treatment for HCCAA, a disease with no currently approved therapeutic options. The study highlights how NAC may reduce disease-associated biomarkers and slow disease progression.

The study is the first phase 2a clinical trial to assess the safety and therapeutic potential of high-dose NAC in patients carrying the L68Q-CST3 sequence variation of HCCAA. Results indicate that NAC is well tolerated, reduces disease specific biomarkers and stabilises disease progression, suggesting NAC may offer a preventative strategy against HCCAA.

These findings could accelerate the development of preventative therapies for individuals at risk of HCCAA and other forms of CAAs.

The open-label phase 2a clinical trial was conducted at Iceland's Landspitali University Hospital from March 2019 to December 2021 . A total of 17 participants were enrolled, all confirmed carriers of the L68Q-CST3 mutation. The study assessed:

Building on these findings, as well as internal in-vitro data that demonstrates that NAC-amide (NACA) has more than 10-fold higher potency in preventing or dispersing amyloid complex aggregates than NAC and increased ability to penetrate the blood brain barrier and get into cells involved in the disease pathogenesis, ATx has launched a European Medicines Agency-approved registration study to monitor safety, tolerability, and efficacy using NACA in patients with HCCAA.

For more details, read the full study in : Original Investigation.

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