UMC Utrecht and VectorY Therapeutics collaborate in ALS Biomarker Study

Finding these biomarkers is expected to help diagnose ALS and monitor disease progression and improve the design of future clinical studie

18-Jun-2024
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UMC Utrecht has enrolled its first patient for a new study to identify biomarkers of amyotrophic lateral sclerosis (ALS). The observational investigator-initiated study is led by Professor Dr. Leonard van den Berg and supported by VectorY Therapeutics.

VectorY Therapeutics is developing innovative vectorized antibody therapies for neurodegenerative diseases. VectorY’s lead program, VTx-002, is targeting TDP-43 for the treatment of ALS. ALS is a devastating condition that in the Western world has an estimated lifetime risk of 1:400. It is characterized by the progressive degeneration of motor neurons, which leads to an average life expectancy after the first signs of symptoms of just three to five years. Currently, there is no cure available for ALS and available treatments only slow disease progression by months.

The new longitudinal study announced aims to identify blood or cerebrospinal fluid (CSF) biomarkers of ALS patients. Finding these biomarkers is expected to help diagnose ALS and monitor disease progression and improve the design of future clinical studies. It is also anticipated that biomarkers will provide insights into ALS pathogenesis, supporting the development of new therapeutics.

The study aims to enroll 70 ALS patients in the Netherlands. Serial blood and CSF samples will be collected, and multiple potential biomarkers identified and tested. These include TDP-43-related biomarkers, which are indicative of the pathogenic processes leading to motor neuron degeneration. The study results will be published in a peer-reviewed journal, contributing to the scientific community's understanding of ALS and its treatment. The study adheres to the principles of the Declaration of Helsinki and the Medical Research Involving Human Subjects Act (WMO) and has received approval from the Medical Research Ethics Committee, NedMec.

Sander van Deventer, CEO of VectorY, commented: "In recent years, key hallmarks of ALS pathogenesis have been elucidated, enabling the development of novel potentially disease-modifying therapies targeting TDP-43 pathology. Clinical development of these therapies is critically dependent on the availability of easily-measurable biomarkers that reflect the underlying pathogenesis. Our collaboration with UMC Utrecht is very important since it will enable access to longitudinal CSF and blood samples that are currently lacking and are essential for biomarker development.”

Leonard van den Berg, Professor of Neurology at UMC Utrecht, commented: “Identifying potential biomarkers for ALS is a crucial step for future research and I welcome this collaboration with VectorY Therapeutics. More research is needed into objective measurements that provide insights into the course of ALS. When more objective biomarkers are available it will be easier to identify effective treatment options. That is always the main goal: to find an effective treatment for everyone living with ALS as quickly as possible. Thanks to VectorY Therapeutics’ initiative and important contribution we will gain more insight into the development and course of ALS.”

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