Discover how high-throughput screening is accelerating drug discovery and repurposing at Memorial Sloan Kettering Cancer Center
Finding new medicines is like searching for a needle in a haystack of 400,000 compounds. Traditional drug discovery has been slow, expensive, and unpredictable—but MSKCC is changing that paradigm.
10-15 years from discovery to patient
Significantly reduced development time
384-well plates enable massive parallel testing
Robotic systems operate 24/7
Advanced computing processes massive datasets
compounds including FDA-approved drugs
human genes targeted for silencing
Researchers designed tests using retinoblastoma cancer cells to identify therapeutic effectiveness 1 .
The entire chemical library was tested against cancer cells to identify active compounds 1 .
Initial "hit" compounds were retested to confirm activity and eliminate false positives 1 .
Confirmed hits underwent further testing to understand mechanism of action 1 .
Digoxin was identified as promising and moved toward clinical application 1 .
| Finding | Significance |
|---|---|
| Activity against retinoblastoma | New use for existing medication discovered |
| Already FDA-approved | Development timeline significantly shortened |
| Patients received treatment | Real-world impact demonstrated |
| Research Tool | Function | Applications |
|---|---|---|
| Chemical Compounds | Small molecules that perturb biological systems | Drug discovery, probe identification |
| siRNA Duplexes | Synthetic RNA for temporary gene silencing | Target identification, functional genomics |
| shRNA Hairpins | DNA sequences for permanent gene silencing | Long-term gene knockdown studies |
| CRISPR-Cas9 Systems | Gene-editing technology | Functional genomics, gene function studies 7 |
| Cell Line Models | Cancer cells representing disease models | Disease modeling, compound screening |
| High-Content Imaging | Automated microscopes for cellular analysis | Morphological analysis, multi-parameter detection |
Advanced organoids and co-cultures that better mimic human tumors for more accurate screening.
Machine learning algorithms to identify patterns and predict promising compounds more efficiently.
Using patient-specific cells to identify optimal treatments for individual disease characteristics.
Expanding screening approaches to neurodegenerative diseases, infectious diseases, and rare genetic disorders.
The PU-H71 inhibitor discovered at MSKCC has advanced to Phase 1b/2 clinical studies, demonstrating the real-world impact of these screening approaches 8 .
By combining scale, technology, and collaboration, MSKCC's screening platforms are building a more efficient path from laboratory discoveries to patient treatments.
"In the ongoing battle against disease, these high-tech approaches provide powerful new weapons—finding needles in haystacks that can save lives."