TDP43 in Circulating Neuron-Derived Extracellular Vesicles as Prognostic and Diagnostic ALS Biomarker
Abstract
Ultimate applicability of the research: This project is designed to advance the development of a new blood-based biomarker that can predict whether a patient with ALS will progress rapidly or slowly. Such a marker can help patients plan for care and is needed to help drug developers narrow the pool of patients who participate in clinical trials to (a) increase the likelihood of successfully identifying drugs that work and (b) reduce the number of patients required for each trial. It is expected that such biomarker would accelerate drug development and increase the success rate of ALS clinical trials. Type(s) of ALS patients the project is designed to help and how it will help them: This tool is currently intended for patients with sporadic ALS, of which, more than 90% have the marker of interest. Faster, less expensive drug development would benefit patients in this population by getting drugs to market faster and allowing drug developers to spread their investments across more drug candidates. The tool could also potentially help doctors decide which patients are likely to respond to specific drugs, and it could help patients and their families make better plans based on the patient’s likelihood of progressing slowly or quickly. Potential clinical applications, benefits, and risks: The initial application of this biomarker is for improving clinical trial design and recruitment, but it has the potential to also be used to help doctors determine which patients will progress slowly to inform care planning. The primary clinical risk of the tool would be incorrectly classifying a patient by their likely progression speed, which may result in care planning being misaligned with actual outcomes. However, there currently is no test available to predict progression, so the tool would be a net improvement. In addition, up to 15% of patients are misdiagnosed, and the tool could eventually be used to correct this and redirect doctors to test for other conditions that likely have a different prognosis and treatment plan. Projected time it may take to achieve a patient-related outcome? With adequate funding, this tool could be applied to clinical trial recruitment within 1 year for evaluating treatment response and within 2 years to predict rate of progression. Use in clinical care as a CLIA test could also be achieved within 2 years, though U.S. Food and Drug Administration (FDA) approval and insurance coverage would be achieved in 3 to 6 years depending on the type of additional testing the FDA requests. Likely contributions of this study to accelerating progress toward eradicating deaths and suffering from ALS: This project has the potential to aid in conducting more efficient clinical trials, which allow testing more treatments and eventually increase the chance of finding an effective treatment for at least a subgroup of ALS.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Jan 04, 2024
- Source ID
- HT94252310077
Entities
People
- Erez Eitan
Organizations
- United States Army