Table 1.
Current medulloblastoma therapeutics and Hh-pathway targets that potentially may be applied to SHH-MB.
| Current and Potential Pipeline Medulloblastoma Drugs | |||
|---|---|---|---|
| Drug | Target | Pathway Affected | Limitations |
| Alkylating agents | DNA | DNA repair | Range of side effects |
| Cyclopamine | SMO | Hh | Low oral solubility |
| Vismodegib (GDC-0449) | SMO | Hh | Possible limited efficacy; mutations in SMO contributes to resistance |
| LDE-225 | SMO | Hh | In mouse model, tumors originally shrunk but then regrew [54] |
| GANT61 [52] | Gli1 and Gli2 | Hh | Tested in prostate cancer cells; Remains to be tested with medulloblastoma |
| HPI-1 [53] | Gli1 and Gli2 | Hh | Preclinical, remains to be tested with medulloblastoma |
| BMS-833923 [55] | SMO | Hh | Clinical trials with gastric and esophageal cancers; remains to be tested with medulloblastoma |
| Saridegib (IPI-926) [56] | SMO | Hh | Preclinical |