Potential treatment

We are all desperately waiting for a treatment for Dutchtype CAA. Alnylam is developing an RNA therapy, Mivelsiran, that potentially slows down the disease.

The patient association, Dutch CAA Foundation, researchers from Perth, Boston and Leiden and the pharma company Alnylam, and of course research participants from Dutchtype CAA families, have been working together to move forward towards a drug trial. Since 2021, our focus has been on TRACK DCAA - a natural history study. With this study, we grew our understanding of how Dutchtype CAA develops in the brain of gene carriers and how we can measure disease progression. TRACK DCAA is a vital study toward drug development, because you need to know how to measure if the therapy is actually slowing down disease progression.

TRACK DCAA, together with other learnings from studies like DIAN, has now led to the first drug trial ever in DCAA. Mivelsiran is a RNA therapy that targets Amyloid Precursor Protein (APP), a protein that eventually leads to the toxic amyloid beta (Aβ). The therapy blinds part of our mRNA - a copy of our DNA that orders the production of proteins. This technique potentially slows down amyloid production – leading to less Aβ in the brain. The drug does not influence existing deposits of Aβ in the brain, but we do hope it slows down the build-up of new ones.

The development of Mivelsiran is ongoing. It has proven to be successful in human cells in the lab and in animals. Since 2023, Alnylam is conducting a phase 1 study (focused on safety, tolerability and target engagement) in people with early Alzheimer’s. In a press release published in April of 2023 you can read more about this study and its results: here.

Because of positive results in phase I, Alnylam has chosen to move forward in a phase 2, in (D)CAA. This means the safety, tolerability and efficacy of the drug will be further studied. Learn more about Alnylam and Mivelsiran through this webinar.

DCAA family members in Perth who know they carry the gene, are able to enroll in cAPPricorn-1, the phase 2 study with Mivelsiran, from March 2025. Inclusion closes probably late 2025/early 2026. Learn more about cAPPricorn-1 here.  You can sign up directly by contacting Samantha Gardener from the research team in Perth. She will be happy to answer all of your questions: s.gardener@ecu.edu.au.