The BATCure family survey is now live.
The project aims to help develop new therapies for Batten disease by identifying target genes and small-molecule treatments for three types of Batten: juvenile CLN3 (the most common), CLN6 and CLN7. It will also study if existing drugs can be repurposed for Batten.
To manage these objectives, the project will develop a whole set of tools, including new cell and animal models. Those will include zebrafish, with their well-characterized, transparent embryonic development; yeast, best used to study cell division; neuronal cells derived from induced pluripotent stem cells (iPSC); cells generated from common somatic cells and embryonic stem cells (ESC); multi-potent cells extracted from embryos; and self-organizing central nervous system (CNS) organoids, mini-organs derived from ESCs or iPSCs.
It will also provide access to a registry of Batten’s patients, with clinical data and patient samples. This could improve diagnosis, monitoring and characterization of the disease.
The three-year project has received $6 millions in funding from the European Union’s Horizon 2020 research and innovation program. It involves several European research labs, biotech companies and other institutions including Britain’s UCL, Manchester Metropolitan University, King’s College London and the Batten Disease Family Association; Italy’s Fondatione Telethon; Spain’s Universidad Salamanca, and LEITAT; Latvia’s OSI, Sweden’s AcureOmics; Germany’s UKE (Germany) and Denmark’s Orphazyme.
“We have to collaborate and combine different innovative approaches using both cellular systems and engineered animals, whose results could later be translated into appropriate and safe therapeutic methods,” Dr. Alessandro Simonati of Italy’s University of Verona — who was part of the previously EU-funded project DEM-CHILD, said in a press release.
Details can be found on the project website. BATCure members plan to present the results of their work through scientific publications and conferences.
The present survey will help understand patient needs and design future endeavors. The website contains no link for the survey, but the Batten Disease Family Association website says members of that group should have received a link to the survey by now.