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Isotretinoin, the acne drug, is given to children with high-risk neuroblastoma for six months after transplant to nudge any surviving cancer cells into growing up into harmless nerve cells. A 1999 trial showed it improved survival and it has been part of standard care since.
Isotretinoin (13-cis-retinoic acid) was approved by the FDA in 1982 for severe acne. Its use in neuroblastoma follows laboratory work showing retinoids induce differentiation of neuroblastoma cells. In the Children's Cancer Group trial CCG-3891 (New England Journal of Medicine, 1999), six months of high-dose pulsed isotretinoin after myeloablative therapy with autologous marrow rescue improved event-free survival, and the drug became the post-consolidation backbone onto which anti-GD2 immunotherapy was later added in ANBL0032.
It is taken by mouth in two-week courses; dry skin and lips, raised triglycerides and hypercalcaemia are the usual effects, and it is highly teratogenic. In medulloblastoma, the SJMB03 trial tested isotretinoin maintenance and found no benefit. The neuroblastoma and medulloblastoma pages name it.
A vitamin A derivative that, through retinoic acid and retinoid X receptors, pushes residual neuroblastoma cells to stop dividing and differentiate towards mature neural cells, given after high-dose therapy when tumour burden is minimal. Connects to Retinoic acid receptor alpha (RARA) and Retinoid X receptor (RXR).
1.Isotretinoin acts on Retinoic acid receptor alpha (RARA), the hormone receptor or the enzyme that makes the hormone.
| Region | Year | Indication |
|---|---|---|
| US | 1982 | Severe recalcitrant nodular acne (use in high-risk neuroblastoma is off-label but standard) |
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Query for this drug: (TITLE:"Isotretinoin" OR ABSTRACT:"Isotretinoin" OR TITLE:"Accutane" OR ABSTRACT:"Accutane" OR TITLE:"13-cis-retinoic acid" OR ABSTRACT:"13-cis-retinoic acid" OR TITLE:"13-cis-RA" OR ABSTRACT:"13-cis-RA" OR TITLE:"cis-retinoic acid" OR ABSTRACT:"cis-retinoic acid") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Isotretinoin, not a curated reading list.
Shares Retinoid X receptor (RXR), Retinoic acid receptor alpha (RARA) and the tag subtype-drugs-wave.
Shares COG ANBL0032, Dinutuximab (ch14.18) / dinutuximab beta, Neuroblastoma (paediatric).
Shares COG ANBL0032, Dinutuximab (ch14.18) / dinutuximab beta, Neuroblastoma (paediatric).
Shares Dinutuximab (ch14.18) / dinutuximab beta, High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares Dinutuximab (ch14.18) / dinutuximab beta, High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares Dinutuximab (ch14.18) / dinutuximab beta, High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares Dinutuximab (ch14.18) / dinutuximab beta, High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares COG ACNS0332, Group 3 and group 4 medulloblastoma (non-WNT/non-SHH).