A potential treatment for DCAA

We are all desperately waiting for a treatment for Dutchtype CAA. Alnylam is developing ALN-APP - a RNA therapy that potentially slows down the disease.

The patient association, Dutch CAA Foundation, researchers from Perth, Boston and Leiden, the pharmacompanies Alnylam and Regeneron and of course research participants from Dutchtype CAA families have been working together to move towards a drug trial. For the past two years our focus has been on TRACK DCAA - a natural history study. With this study, we are growing our understanding of how Dutchtype CAA develops in the brain of gene carriers and how we can measure disease progress. 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.

ALN-APP is a RNA therapy that targets Amyloid Precursor Protein or APP, a protein that eventually leads to the toxic Amyloid Beta. The therapy shuts down part of our mRNA - a copy of our DNA that orders the production of proteins. Shutting down amyloid production might lead to less amyloid beta in the brain and this will help slow down DCAA.

The drug development is in its early stages. It has proven to be succesful in human cells in the lab and also in animals. Earlier this year Alnylam conducted a phase 1 study (focused on safety, tolerability and target engagement) in people with early Alzheimers. In this press release published in April of 2023 you can read more about this study and its results.

Talks with Alnylam are ongoing about doing a phase 2 study with families with Dutchtype CAA in the Netherlands. During a family meeting on July 22nd, Alnylam will present itself and the results of the drug trial so far to the Dutch families. We are working on making this meeting available through a livestream or recording for the Aussie families (and other families outside of the Netherlands) as well.

Discussions are also ongoing about bringing the trial to Perth. This means that we will be asking Australian family members to partcipate. So far, 24 members are part of TRACK DCAA. We really appreciate their participation and involvement and we are hoping more family members stand up to do so. This will increase our chances of a drug trial in Perth with ALN-APP. This means that if the drug turns out to be succesful, research participants have early access to it.

Are you from a DCAA family and thinking about participating in TRACK DCAA? Please do not hesitate to contact Samantha Gardener from the research team in Perth. She will be happy to answer all of your questions: s.gardener@ecu.edu.au.