RARE Hope Leads Global Scientific Working Session in Iceland to Advance Therapeutic Strategies for AHC
Reykjavík, Iceland — May 13, 2025 — RARE Hope, a nonprofit accelerating translational research for rare neurological disorders, led a high-level scientific working session in Reykjavík from May 9–11. The meeting brought together an international team of experts—including four members of RARE Hope’s Scientific Advisory Board—to consider therapeutic strategies to improve quality of life for patients with Alternating Hemiplegia of Childhood (AHC).
Designed and moderated by RARE Hope President Nina Frost, the agenda centered on proposing and evaluating emerging hypotheses related to AHC pathophysiology and reviewing repurposed drug candidates identified through computational prediction tools. The collaborative format leveraged significant multidisciplinary expertise spanning clinical neurology, electrophysiology, structural biology, and systems modeling, enabling rigorous debate and integration of perspectives.
Several candidate compounds were prioritized for potential advancement into exploratory studies using established AHC animal models. These efforts will help determine whether specific drug classes can modify disease phenotype and support broader investigation of therapeutics aimed at improving patient quality of life.
An ongoing area of focus for the group involves developing a framework to group ATP1A3 mutations into mechanistic or phenotypic clusters. Improved stratification based on underlying biology may help align treatment strategies with patient subtypes and inform future clinical trial design.
“Our goal is to leverage the exceptional expertise of our scientific partners. We’re combining mechanistic insight and AI predictions to establish rational drug exploration pathways,” said Nina Frost, President of RARE Hope. “This session delivered clear, actionable priorities to guide the next phase of therapeutic investigation.”
The working session was hosted by Siggi Jóhannesson, President of the Icelandic AHC organization and the AHC Federation of Europe. Siggi founded the initiative, known as ATT-Now, in 2022 to focus research efforts on alleviating the most severe symptoms of AHC. A dedicated parent and tireless advocate, Siggi is a unifying force for AHC research and patient communities across Europe and the world.
RARE Hope remains committed to catalyzing high-impact, data-driven research efforts to accelerate therapeutic development for rare neurological diseases and address the critical needs of patients.