Table 1.
Summary of clinical off-target based repurposing studies
| Drug | Original Target | Original Disease | Off-Target(s) | Repurposed Disease | Clinical Status | Revenue |
|---|---|---|---|---|---|---|
| Crizotinib | MET | Solid Tumors | EML4-ALK | NSCLC | Approved | >$3 bio (2012–18) |
| ROS1 Fusions | NSCLC | Approved | ||||
| Imatinib | BCR-ABL | CML | KIT | GIST | Approved | N/A for GIST alone |
| Midostaurin | PKC | Solid Tumors | FLT3 | FLT3+ AML | Approved | N/A |
| KIT | SM & MCL | Approved | ||||
| Thalidomide | Unknown | Morning Sickness | Immune Modulation (CRBN) | ENL, MM | Approved | ~$4 bio (2005–18) |
| Lenalidomide | MM, MDS (5q), MCL | Approved | >$53 bio (2006–18) | |||
| Pomalidomide | MM | Approved | ~$7 bio (2013–18) | |||
| Erlotinib | EGFR | NSCLC | JAK2 | MDS, AML | Discontinued (Phase II) | - |
| Tozasertib | AURKA-C | Multiple Cancers | BCR-ABLT315I | CML & Ph+ ALL | Discontinued (Phase II) | - |
| Desipramine | SERT, NET | Depression | Various Receptors | SCLC | Discontinued (Phase II) | - |
| Dasatinib | SRC, ABL | CML & Ph+ ALL | DDR2 | NSCLC | Ongoing | - |
| BTK | CLL | Ongoing | - | |||
| Ibrutinib | BTK | MCL, CLL, WMG | EGFR | NSCLC | Ongoing | - |
| ERBB2 | ERBB2+BC & GEC | Ongoing | - | |||
| Axitinib | VEGFRs | RCC | BCR-ABLT315I | CML | Ongoing | - |
| Ponatinib | BCR-ABLT315I | CML | RET | RET+ MTC & NSCLC | Ongoing | - |
| Cabozantinib | MET, VEGFR2 | MTC, RCC, HCC | ROS1 Fusions, NTRK Fusions | NSCLC | Ongoing | - |
| Ceritinib | EML4-ALK | NSCLC | ROS1, | NSCLC | Ongoing | - |
| IGF1R, FAK, RSK1/2 | NSCLC | Ongoing | - | |||
| IGF1R, ACK1 | Melanoma | Ongoing | - | |||
| Itraconazole | Lanosterol-14a-de-methylase | Fungal Infections | SMO | Multiple Cancers, BCC | Ongoing | - |
| Disulfiram | ALDH | Alcoholism | NPL4 | Multiple Cancers | Ongoing | - |
| Chloroquine | Heme | Malaria | PPT1 | Multiple Cancers | Ongoing | - |
| Artemisinin | Reactive Oxygen Species | Malaria | BAD | Multiple Cancers | Ongoing | - |
| Sulfasalazine | Unknown | IBD, RA | SPR | Cancer Pain | Ongoing | - |
BC: breast cancer; BCC: basal cell carcinoma; GEC: gastroesophageal cancer; HCC: hepatocellular carcinoma; IBD: inflammatory bowel disease; MTC: medullary thyroid cancer; RA: rheumatoid arthritis; RCC: renal cell carcinoma; WMG: Waldenstrom’s macroglobulinemia; N/A: not available.