Radboudumc will initially focus on precisely mapping the disease SCA1, by following the natural course of the disease and by conducting research into changes in blood, cerebrospinal fluid and MRI scans in SCA1. At a later stage, Radboudumc will be involved in conducting the first treatment study within the project. Radboudumc team members involved:

Redenlab (www.redenlab.com) is an Australian company specialized in the automated analysis of language and speech. They have a lot of experience in this field, also with ataxia. The speech recordings that we will make during this study will be anonymized and shared via a secure connection with Redenlab, who will do the analysis of these recordings. The question here is whether such automated speech analyses can quickly and reliably pick up changes of the disease. Redenlab team members involved:

The LUMC (www.neurodlableiden.com/nl) specializes in the preclinical development of drugs for hereditary brain disorders. The 3-year pilot study that preceded the current studies was performed at the LUMC. This pilot study was funded by the 'Dutch SCA1 Families Fund' and facilitated by the Dutch Brain Foundation. In the current research projects, the LUMC validates the candidate drug (study 1) and develops a new drug (study 2) in SCA1 cell models.

Involved team members LUMC:

VICO Therapeutics (www.vicotx.com) is a Dutch biotech company based in Leiden, specializing in developing medications for rare hereditary diseases. VICO has developed a candidate drug for polyglutamine disorders, including SCA1, and is currently assessing its safety and initial indications of efficacy in a clinical trial in several European countries. In addition to SCA1 patients, patients with SCA3 and Huntington's disease are also participating in this clinical trial. For SCA1 in the Netherlands the Radboud University Medical Center (Radboudumc) is involved in this.

For more information about the clinical trial, click here.

Vico Therapeutics team members involved:

SCA1 Giving Circle (www.hersenziekte-sca1.nl). In July 2015, more than 10 SCA1 families formed the Giving Circle, after having seen family members suffer and die from SCA1 for years. The Giving Circle aimed to raise sufficient funds through campaigns and donations for research into a drug for SCA1. These campaigns started in mid-September, and in June 2016 there was sufficient to start the preliminary study in the LUMC. In the meantime, several SCA1 families have joined and are closely involved in the research.

SCA1 Giving Circle team members involved:

Dr. Inge Zuhorn is an expert in the field of nanomedicine and a researcher at the University Medical Center Groningen (UMCG). Her research focuses on the development of nanoparticles for targeted drug delivery, with a special focus on overcoming biological barriers such as the blood-brain barrier. In research 2, she focuses on the development of new ways of administering ASOs.

Involved team members University Medical Center Groningen