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UMC Utrecht and VectorY Therapeutics Collaborate on ALS Biomarker Research

NextEdge Admin

18 Jun 2024

3 min 8 sec

UMC Utrecht has initiated a pioneering study aimed at identifying biomarkers for Amyotrophic Lateral Sclerosis (ALS), a devastating neurodegenerative disease characterized by progressive motor neuron degeneration. Led by Professor Dr. Leonard van den Berg, the observational study is conducted in collaboration with VectorY Therapeutics, a company focused on developing vectorized antibody therapies for neurodegenerative conditions.

ALS poses a significant health challenge with a lifetime risk in the Western world estimated at 1 in 400 individuals. The disease typically leads to a rapid decline in motor function and has a short life expectancy of three to five years post-symptom onset. Presently, treatments are limited to slowing disease progression marginally.

The study, which plans to enroll 70 ALS patients in the Netherlands, aims to identify blood or cerebrospinal fluid (CSF) biomarkers. These biomarkers are crucial for diagnosing ALS, monitoring disease progression, and enhancing the design of future clinical trials. Specifically, the research focuses on biomarkers associated with TDP-43, a protein implicated in ALS pathogenesis.

The longitudinal study involves the collection of serial blood and CSF samples from participants to analyze and validate potential biomarkers. This data will not only aid in understanding ALS pathogenesis but also support the development of new therapies, including VectorY's VTx-002 program targeting TDP-43 pathology.

Ethically conducted under the principles of the Declaration of Helsinki and approved by the Medical Research Ethics Committee, NedMec, the study underscores a commitment to advancing ALS research responsibly. Results will be disseminated through peer-reviewed publications, contributing valuable insights to the scientific community.

Sander van Deventer, CEO of VectorY, emphasized the critical role of biomarkers in advancing therapeutic development, highlighting the collaborative effort with UMC Utrecht to access essential longitudinal samples.

Professor Leonard van den Berg expressed optimism about the study's potential to uncover objective measures for ALS, essential for identifying effective treatments promptly. He underscored the urgency in finding therapies to improve outcomes for ALS patients.