Table 3.
Short-listed repurposed treatments for SSMD and GPX4-related diseases
| Name | Rationale | Availability | SSMD Status |
|---|---|---|---|
| Vitamin E | Potent antioxidant known to prevent ferroptosis | Over-the-counter |
Administered to 2 of 4 patients Dosage: 15 mg 2X/day |
| N-Acetyl-Cysteine (NAC) | Increases glutathione biosynthesis to boost residual GPX4 activity | Over-the-counter |
Administered to 2 of 4 patients Dosage: 300 mg 3X/day |
| CoQ10 | Essential for repair of peroxidized lipids. Acts as an antioxidant | Over-the-counter |
Administered to 2 of 4 patients Dosage: 50 mg 2X/day |
| Selenium | Limiting step in GPX4 production, may increase selenoprotein expression | Over-the-counter |
Administered to 1 of 4 patients Dosage: 75 µg 1X/day |
| L-methionine | Increases glutathione biosynthesis through transsulferation pathway to boost residual GPX4 activity | Over-the-counter | Pending administration |
| RT001 | Protects lipid membranes against peroxidation | In clinical trials for treating multiple indications | Administered to 2 of 4 patients under Expanded Access |
| Dimethyl fumarate | Activates Nrf2 gene, master regulator oxidative stress response | Approved in USA for treating multiple sclerosis | Evaluating for of off-label use |
Collection of potential and in-use repurposed treatments for current SSMD patients