Drug repurposing for unmet medical needs
While nearly 8000 rare diseases have been identified, only 5 per cent have licensed treatments. As most of these diseases are life-threatening, it underscores the urgent need for new drugs. Drug repurposing (also called drug repositioning) consists in identifying new uses for approved or investigational drugs that are outside the scope of the original medical indication. It represents an opportunity for rare diseases and patients with unmet needs. It is an alternative option in drug development and is often presented as being a viable, risk-managed strategy for pharmaceutical companies developing orphan drugs.
| Brief summary for repurposing approaches | |||
| Repurposing approach | Challenges | Examples | |
| Phenotypic in vitro assays | Hit validation and target deconvolution; | Pimozide and tamoxifen are prescribed to treat psychiatric disorders and breast cancer, respectively. Found to have the ability to kill Toxoplasma through novel pathways | |
| In-vivo-based phenotypic screens | possible lead compounds with poor pharmacokinetics might not be active in the primary screens | ||
| Target-based method | Poor productivity | l-type calcium channel blockers to treat cryptococcosis | |
| Pathway- or network-based methods | Incorporate the cellular context and genetic background into the disease networks to enable more stratified and selective target predictions, as well as how to make the prediction models more realistic for the practical drug discovery and therapeutic applications | Potential repositioning of GV1001 as a therapeutic agent for testosterone-induced benign prostatic hyperplasia | |
| Signature-based approaches | Provide data for primary analysis only, further analysis is required | New drug candidates for the treatment of atypical meningioma | |