RARE Daily

LifeArc and ALS Therapy Development Institute Collaborate to Find and Validate Biomarkers

May 28, 2024

Rare Daily Staff

The UK charity LifeArc and the US non-profit ALS Therapy Development Institute said they are collaborating to identify, validate, and develop new biomarkers that are needed to improve the evaluation of patients and drug development for the rare neurodegenerative disease amyotrophic lateral sclerosis.

Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (NMD) is a progressive and fatal disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death. More than 90 percent of people with ALS have sporadic disease, showing no clear family history.

The collaboration will focus on validating MMP-9 and TIMP-1 as minimally invasive biomarkers for ALS. The research will use serum and plasma samples from people living with ALS, through the ALS Research Collaborative study. By correlating the levels of these biomarkers with longitudinal and cross-sectional clinical data, the project aims to develop a kit for use in both clinical and research settings for prognosis and potential monitoring of the disease.

Analyzing the protein levels in these samples, along with other clinical data from the study, will enable the team to determine the utility of MMP-9 and TIMP-1, and other protein analytes,” the organizations said.

The project, which will run for two years, is part of LifeArc’s MND Translational Challenge. Under the terms of the collaboration, LifeArc will have the exclusive option to further develop the project’s outputs, leveraging its translational and diagnostic development capabilities.

“Diagnostic and prognostic biomarkers for MND/ALS are a needed tool for patient care and would offer crucial insights for clinical research and drug development. Currently, we rely on non-specific biomarkers common to different neurodegenerative diseases due to a lack of validated biomarkers specific for MND/ALS and a lack of consensus among researchers,” said Manuela Cerina, scientific director of neurodegeneration at LifeArc. “Recent evidence shows that high or low plasma levels of Matrix Metalloproteinase 9 (MMP-9) and Tissue Inhibitor of MetalloProteinases 1 (TIMP-1), proteins significantly correlate with ALS progression and severity. These biomarkers can be measured from blood plasma and serum, keeping the testing minimally invasive for the patients.”

Stay Connected

Sign up for updates straight to your inbox.