Commercialization of a Diagnostic test for amyotrophic lateral sclerosis (ALS)

DESCRIPTION provided by applicant Amyotrophic lateral sclerosis ALS or Lou Gehrigandapos s disease is a fatal neurodegenerative disease with a typical lifespan of years after diagnosis The initial symptoms can be similar to many other neurologic disorders and often begin with limb weakness muscle fasciculations or increasing difficulties in speech or breathing No definitive diagnostic tests currently exist for ALS and clinical diagnosis typically takes months from symptom onset and relies on ruling out other potential causes of the clinical symptoms Earlier diagnosis will permit more rapid initiation of treatment with newer drug therapies in development or equally important provide a means to rapidly rule out ALS as a cause of the symptoms Iron Horse Diagnostics Inc was created to finalize the development of diagnostic and prognostic indicators of ALS and market these assays as laboratory developed tests LDTs in a Clinical Laboratory Improvement Amendments CLIA certified lab We have produced a series of peer reviewed publications convincingly demonstrating that significant levels of pNfH and complement c can be detected in the blood and CSF of ALS patients and that the levels detected by our assays provide diagnostic utility for ALS We propose a series of tasks to optimize and validate our assays in a CLIA certified laboratory the last remaining steps before commercialization This is a FastTrack application because the remaining steps before Phase III are well defined with highly definable go no go gates During Phase I of the proposed work we will finalize and optimize assays as diagnostic indicators of ALS by testing samples for indicative proteins and translating the assay technology onto a new platform Phase II will expand on this by conducting a multi center prospective clinical research study and qualifying the assays using patient samples in a CLIA certified lab to determine the overall accuracy of the ALS diagnostic The goal of this proposal is to generate a sensitive and validated LDT Lab Developed Test for these biomarkers useful for clinically diagnosing ALS in a CLIA certified lab A certified LDT will be marketed to clinicians as a service PUBLIC HEALTH RELEVANCE Amyotrophic lateral sclerosis ALS is a fatal neurodegenerative disease that currently lacks a diagnostic test Iron Horse Diagnostics has cerebrospinal fluid and blood based diagnostic assays that in our application will be qualified validated and commercialized as a laboratory developed test LDT for ALS