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The CureQ project focuses on the hereditary brain disorders Spinocerebellar ataxia (SCA) type 1 and type 3 and Huntington’s disease.
A Dutch consortium that focuses on predicting, slowing down and curing certain rare movement disorders has received almost five million in grant from NWO / National Science Agenda (NWA) for the CureQ project. Neurologist Bart van de Warrenburg is one of the main applicants.
The CureQ project focuses on the hereditary brain disorders Spinocerebellar ataxia (SCA) type 1 and type 3 and Huntington’s disease. These diseases are caused by too many repeats of the same piece of DNA (repeats). This leads to an accumulation of harmful proteins that cause the diseases. The diseases can start at a relatively young age and often quickly lead to significant disabilities.
Experimental treatments
There are as yet no treatments that can stop the disease or slow its decline. Recently, however, promising studies have started with experimental genetic treatments, via spinal taps or a single administration in the brain, to inhibit the production of the proteins that cause the disease. Before these treatments can be applied safely and in a targeted manner, a number of important questions must be answered. Van de Warrenburg: “At the moment we do not know what the optimal moment is to start a treatment and who can benefit from these therapies. To determine that moment, we need to find ways to better predict the onset and course of these diseases. But how do carriers of these hereditary errors deal with these predictions, and do they want to know them? Furthermore, the treatments under investigation are quite invasive and we need to look for alternative strategies.”
Divers
Patients with these movement disorders are divided into groups based on the time of onset of the disease: very early age, adulthood, and old age. Van de Warrenburg: “Both the age of onset of the disease and the manifestations of the disease are very different and these groups probably also require different treatments. Answering the above questions therefore requires a careful and differentiated approach.”
Consortium
Researchers and doctors from various Dutch universities, various HBO courses, ethicists, biotechnology companies, patient associations and the Proefdiervrij foundation will collaborate in the CureQ project, with the aim of enabling a person-oriented approach and treatment for SCAs and Huntington’s disease.
The Academic Alliance between Radboudumc and MUMC+ for Rare Neurological Movement Disorders is an important pivot in patient-related research within the project. The intended breakthroughs in new therapeutic strategies, combined with the use of cultured cell models of gene carriers, should lead to individualized treatments and perspective for gene carriers and their families. The total budget for the research is €5.5 million, of which €4.7 million is subsidized by the Netherlands Organization for Scientific Research.
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- Name author and/or edited by: Radboudumc
- Photographer or photo agency: : INGImages
- Source for this article: : Radboudumc
- What is the URL for this resource?: https://www.radboudumc.nl/nieuws/2022/vijf-miljoen-voor-zeldzame-bewegingsstoornissen
- Original title: Five million for rare movement disorders
- Target audience: Healthcare Professionals, Students
- Date: 2022-03-23