Riluzole
| Evidence Level: L5 | Predicted Indications: 8 |
Table of Contents
- Riluzole
- Pharmacist Assessment Report
- Riluzole: From Amyotrophic Lateral Sclerosis to Bilateral Parasagittal Parieto-Occipital Polymicrogyria
- One-Sentence Summary
- Quick Overview
- Why is This Prediction Reasonable?
- Clinical Trial Evidence
- Literature Evidence
- Australia Market Information
- Safety Considerations
- Conclusion and Next Steps
- Disclaimer
Riluzole: From Amyotrophic Lateral Sclerosis to Bilateral Parasagittal Parieto-Occipital Polymicrogyria
One-Sentence Summary
Riluzole is a glutamate-modulating medicine originally used to treat amyotrophic lateral sclerosis (ALS). The TxGNN model predicts it may be effective for bilateral parasagittal parieto-occipital polymicrogyria (a cortical malformation), but there are currently no clinical trials and no publications supporting this specific prediction. The prediction is a model output only.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Amyotrophic lateral sclerosis |
| Predicted New Indication | Bilateral parasagittal parieto-occipital polymicrogyria |
| TxGNN Prediction Score | 0.9999 |
| Evidence Level | L5 |
| Australia Market Status | Listed on the PBS Schedule (1 of 1 items) – TGA (ARTG) registration status is not included in this data |
| Number of PBS Items | 1 (valid: 1) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available. Based on known information, riluzole is generally described as a glutamate-modulating agent. Its efficacy in ALS is established, though the survival benefit is modest (roughly 2-3 months in the literature compiled for this report).
The data compiled for this report show no mechanistic link between glutamate modulation and a cortical developmental malformation such as polymicrogyria. The high TxGNN score is a model prediction only, and no trials or publications back it. This prediction should be treated as a hypothesis, not a supported repurposing opportunity.
Other predictions ranked highly by the model are more biologically plausible because they sit close to ALS. These include lower motor neuron syndrome with late-adult onset, monomelic amyotrophy, Mills syndrome and ALS type 22. None of them has trial or literature support in the data either.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
Currently no related literature available for the predicted indication of bilateral parasagittal parieto-occipital polymicrogyria.
Australia Market Information
| ARTG Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| ARTG 11662T | Teglutik | Oral suspension 50 mg per 10 mL, 300 mL | Not available in the data compiled |
Safety Considerations
- Conditions flagged as contraindications or cautions in the knowledge graph (these come from a drug-disease database, not from approved label text): neutropenia, agranulocytosis, substance abuse/dependence, hyperbilirubinaemia (including hereditary forms and Gilbert syndrome), interstitial lung disease, and drug-induced liver injury.
- Drug Interactions: 44 interactions were recorded. Those listed with a Moderate severity include:
- Obeticholic acid
- Naltrexone
- Deferasirox
- Osilodrostat
- Anagrelide
- Asparaginase Escherichia coli
- Brentuximab vedotin
- Clofarabine
- Methotrexate
- Epirubicin
- Minor interactions include omeprazole, caffeine, theophylline and aminophylline.
- Liver safety: Published material notes that about 50% of ALS patients on oral riluzole tablets have ALT above the upper limit of normal, 8% above 3× ULN and 2% above 5× ULN.
Please refer to the TGA-approved Product Information (PI) for full safety information, as label warnings and contraindications were not available in the data compiled.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction has only a model score behind it (Evidence Level L5). There are no trials or publications, and no plausible mechanistic link to a cortical malformation. Safety signals, particularly liver injury and neutropenia, add to the case for caution.
To proceed, the following is needed:
- Detailed mechanism of action data for riluzole
- A plausible biological rationale linking glutamate modulation to polymicrogyria, with preclinical evidence
- Approved indication text and PI warnings and contraindications for the Australian product
- A review of the closer-to-ALS predictions (for example ALS type 22, monomelic amyotrophy, lower motor neuron syndrome), which may be better candidates for further evaluation
- Confirmation that the oral suspension is suitable for the intended population
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.