The progressive neurological disorder currently affects more than 200,000 people worldwide
The University Medical Center (UMC) Utrecht, Netherlands, has announced that it has enrolled its first patient in a new study to identify biomarkers of amyotrophic lateral sclerosis (ALS), in collaboration with VectorY Therapeutics.
Findings from the longitudinal study could support the development of VectorY’s lead programme, VTx-002, a targeted TDP-43 treatment for ALS.
Affecting more than 200,000 people worldwide, ALS is a progressive neurological disorder that affects motor neurons, the nerve cells in the brain and spinal cord, impacting muscle movement and breathing.
Aiming to enrol 70 ALS patients in the Netherlands, the study will identify blood or cerebrospinal fluid (CSF) biomarkers of ALS patients to help improve diagnosis, monitor disease progression, improve the design of future clinical studies and provide insights into ALS pathogenesis to help support the development of new therapeutics.
All blood and CSF samples will be collected, while multiple potential biomarkers will be identified and tested, including TDP-43-related biomarkers, which are indicative of the pathogenic processes leading to motor neuron degeneration.
TDP-43 is an RNA/DNA-binding protein that plays a key role in the regulation of RNA processing in the nucleus and cytoplasm.
Currently in preclinical development, VectorY’s VTx-002 is a vectorised antibody that selectively clears misfolded and aggregated TDP-43 from the cytoplasm of neuronal cells, restoring the essential function of TDP-43 in the nucleus, to preserve neuronal cell function and health in ALS.
Leonard van den Berg, professor of neurology, UMC Utrecht, said: “Identifying potential biomarkers for ALS is a crucial step for future research.
“When more objective biomarkers are available, it will be easier to identify effective treatment options… for everyone living with ALS as quickly as possible.”
Sander van Deventer, chief executive officer, VectorY, commented: “Clinical development of [potentially disease-modifying therapies targeting TDP-43 pathology] therapies is critically dependent on the availability of easily-measurable biomarkers that reflect the underlying pathogenesis.
“Our collaboration… will enable access to longitudinal CSF and blood samples that are currently lacking and are essential for biomarker development.”
