Grant for research into rare movement disorders

Project CureQ focuses on the genetic brain disorders Spinocerebellar ataxia (SCA) type 1 and type 3 and Huntington’s disease.

A Dutch consortium focused on predicting, slowing and treating some rare movement disorders has received nearly five million grants from the NWO/National Science Agenda (NWA) for the CureQ project. Neurologist Bart van de Warenburg is one of the main applicants.

Project CureQ focuses on the genetic brain disorders Spinocerebellar ataxia (SCA) type 1 and type 3 and Huntington’s disease. These diseases are caused by the replication (replication) of the same piece of DNA. This leads to the accumulation of harmful, disease-causing proteins. Diseases can begin at a relatively young age and often quickly lead to significant disabilities.

Experimental treatments
There are still no treatments that can stop the disease or slow its progression. However, studies have recently begun promising with experimental genetic therapies, via spinal taps or a single administration into the brain, to inhibit the production of disease-causing proteins. 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 is the optimal moment to start treatment and who can benefit from these treatments. 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 genetic errors cope with these predictions, And do they want to know it? Moreover, the treatments currently under investigation are very invasive and we need to look for alternative strategies.”

diverse
Patients with these movement disorders are divided into groups based on the time of onset of the disease: very young age, puberty, and old age. Van de Warrenburg: “Both the age of onset and disease manifestations are very different, and these groups may also require different treatments. Thus, answering the above questions requires a careful and distinct approach.”

Alliance
Researchers, clinicians from various Dutch universities, different HBO courses, ethicists, biotechnology companies, patient associations and the Proefdiervrij Foundation will collaborate on the CureQ project, with the goal of enabling a people-oriented approach to the treatment of SCAs and Huntington’s disease.

The academic alliance between Radboudumc and MUMC+ for rare neurological movement disorders is an important focus of patient-related research within the project. Intended breakthroughs in novel therapeutic strategies, combined with the use of cultured cell models of gene carriers, should lead to individual therapies and a perspective for gene carriers and their families. The total research budget is 5.5 million euros, of which 4.7 million euros is supported by the Netherlands Organization for Scientific Research.