Cross-Sectional Examination of Cerebrospinal Fluid Kinetic Biomarkers of Axonal Transport for Amyotrophic Lateral Sclerosis (Als) Diagnosis and Progression

Investigator: Richard Price, MD
Sponsor: KineMed, Inc.

Location(s): United States


The absence of meaningful biomarkers has remained a roadblock in the development and clinical application of treatments for neurological disorders. Biomarkers of this pathogenically causal process may be used for the development of drugs to treat Parkinson’s disease. Researchers, led by Dr. Patrizia Fanara at KineMed, Inc., and Dr. Marc Hellerstein at the University of California, Berkeley and KineMed, Inc., working with collaborators at the University of California San Francisco and the University of Osnabruck, Germany, used the 2H2O labeling approach to specifically track, for the first time, the movement of cargo proteins that rely on axonal transport prior to release into the cerebrospinal fluid (CSF).
The results demonstrate that the axonal transport of cargo proteins is impaired in preclinical models of ALS and Parkinson’s disease and correlates with disease severity. Moreover, treatments targeting this malfunction in preclinical models delayed or reversed disease symptoms. The study further revealed that similar abnormalities in axonal transport can be tracked through a single CSF sample in Parkinson’s patients, which makes this method easy to apply in clinical settings.