UTRECHT, Netherlands & AMSTERDAM--(BUSINESS WIRE)--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 today 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.
Individuals interested in enrolling for the study can find out more by contacting UMC Utrecht ALS Research: als-onderzoek@umcutrecht.nl.
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.”
Notes to Editors
About UMC Utrecht
The University Medical Center Utrecht (UMCU; Dutch: Universitair Medisch Centrum Utrecht) is the University hospital of the city of Utrecht in Netherlands and is one of the seven UMC’s in the country. It is affiliated with the Utrecht University. UMC Utrecht comprises the academic hospital, the faculty of Medicine as well as the Wilhelmina Children's hospital. In total approximately 12,000 people work at the UMCU including medical staff, nursing staff, residents, support personnel and researchers, making it one of the largest hospitals in the Netherlands with a large research department.
About VectorY
VectorY is on a mission to provide patients with neurodegenerative diseases a longer, better life by creating transformative vectorized antibody treatments. Our platform combines the promise of precise therapeutic antibodies with one-time AAV-based delivery to the CNS. Unique in-house expertise in antibodies, AAV vectors, protein degradation, manufacturing and neuroscience drives the rapid development of much needed disease-modifying therapies for neurodegenerative diseases such as ALS. For more information, see www.vectorytx.com.
About VTx-002
VTx-002 is being developed to delay disease progression and preserve the quality of life of ALS patients. VTx-002, currently in preclinical development, is a vectorized antibody that selectively clears misfolded and aggregated TDP-43 from the cytoplasm of neuronal cells. Thereby, it restores the essential function of TDP-43 in the nucleus leading to preservation of neuronal cell function and health.
