Proteomics Blood-Based Biomarker Discovery for ALS
Abstract
Background: There is broad consensus in the ALS research, clinical, and patient communities that diagnostic and prognostic biomarkers are of paramount importance to the ultimate goal of developing effective treatments for all people with ALS. We need biomarkers to accelerate drug development. This would, first, be helpful to ALS patients and their clinical care teams because they could make more informed decisions about how aggressive to be with regard to therapeutic approaches and interventions like feeding tubes, respiratory assistance, and changes to the home. This would also be immensely helpful for clinical trialists aiming to enroll people with ALS in clinical trials, minimize placebo cohort sizes, and enroll broader profiles of people with ALS into experimental trials. Prognostic biomarkers would allow clinical trial designers and data analysts to predict how a participant would have likely progressed in their disease in the absence of any treatment by looking at the biomarker levels prior to treatment intervention. Furthermore, a prognostic biomarker might change with treatment and provide early indications regarding whether a person is responding to a treatment or not. This study has the potential to benefit all people with ALS. Study Design: We will collect blood from between 50 and 60 people with ALS who are currently enrolled in or will join the Precision Medicine Program at ALS Therapy Development Institute. The program currently has more than 300 participants enrolled. Participants will not need to leave their homes for blood collection. We will analyze approximately 5,000 proteins in the blood of these participants. We will use both standard and artificial intelligence approaches to identify patterns of blood proteins that predict faster or slower ALS. Blood proteins that predict faster or slower disease might be considered biomarkers. Timeframe: This study will take two years to complete. We can complete this project quickly because the protocol has already been approved by our Institutional Review Board and because we already have volunteers enrolled. Contribution to eradicating ALS deaths and suffering: Successful completion of this study could yield blood-based biomarkers that can be used to reduce the restrictiveness of clinical trial selection criteria by establishing predicted disease courses for each trial participant. In addition, clinical trials might be shorter because changes in biomarkers might be strong indicators of drug efficacy. Shorter trials and more ease of enrollment decreases the time to effective treatment development dramatically. This means effective treatments are delivered to people with ALS sooner.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Dec 05, 2021
- Source ID
- W81XWH2110240
Entities
People
- Fernando Vieira
Organizations
- ALS Therapy Development Institute
- United States Army