Potential treatment

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

The patient association, Dutch CAA Foundation, researchers from Perth, Boston and Leiden, the pharma companies 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 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.

ALN-APP is an 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 ALN-APP is in an early stage. It has proven to be successful in human cells in the lab and in animals. Earlier this year Alnylam conducted a phase 1 study (focused on safety, tolerability and target engagement) in people with early Alzheimer’s. In this press release published in April of 2023 you can read more about this study and its results here.

Talks with Alnylam are ongoing regarding conducting a phase 2 study with families with Dutchtype CAA in the Netherlands and Australia. During a family meeting on July 22nd 2023, Alnylam presented themselves and the results of the drug trial so far to the Dutch families in Katwijk.  During the webinar on August 10th, they will do the same for you. The link has been made available to you through email, if you have not received it, please contact Sanne van Rijn at svanrijn@hchwa-d.nl.

Discussions are ongoing about bringing the trial to Perth. This means that we need Australian family members to participate. So far, 24 DCAA family 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. Research participants have early access to a drug that might turn out to be successful in slowing down DCAA. You can read more about what participation in TRACK DCAA looks like here.

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