Development of Lovastatin or Related Compounds as a Therapy for ALS
Abstract
Amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease is a condition in which motor neurons of the brain and spinal cord degenerate. The disease worsens over time, resulting in loss of muscle control and eventual death. One way to better understand ALS is to use information from anonymous health records to see what medical factors are related to whether someone develops ALS and how long they survive with ALS if they get the disease. Using a large Medicare dataset with several years of billing records from anonymous patients with or without ALS, we identified medications that appear to help prevent ALS. We then tested some of these medications in mice. We discovered that one of these medications -- a statin used to treat high cholesterol -- appeared to benefit mice who are genetically predisposed to develop a condition similar to ALS. The mice given the statin lived longer and lost motor neurons slower than untreated mice. In order to better understand whether this medication, or a new similar medication, might help prevent or treat ALS, more research is needed to understand how the drug works to protect the neurons that are damaged in ALS. In this proposal, we will expand on our prior work to include more anonymous health records with information about patients’ cholesterol levels and evaluate cholesterol lowering mechanisms in mice, including mice with different genetics than our original tests. This work will help us understand which target in the body affected by the drug is responsible for motor neuron protection. Our goal for this project is to better understand whether and why the tested medications might be suitable to use in humans to prevent or slow ALS. With the successful completion of these studies, we will have defined a new target for designing medications to treat all or some types of ALS.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Jan 04, 2024
- Source ID
- HT94252310154
Entities
People
- Timothy M Miller
Organizations
- United States Army
- Washington University in St. Louis