Table 1.

Targeted therapies against MYCN and ALK currently in development

Drugs targeting MYCN	Drug	Development phase		Pediatric trial identifier
Class I – Drugs targeting DNA binding functions of MYCN
MYCN/MAX heterodimerization	10058-F4	Pre-clinical
	Mycro3	Pre-clinical
Class II – Drugs targeting transcription of MYCN
BET bromodomain	OTX015	Phase I	– Adult
	JQ1	Pre-clinical
	I-BET762	Pre-clinical
	Pfi1	Pre-clinical
	CPI203	Pre-clinical
Class III – Drugs targeting synthetic lethal interactions of MYCN
CHK1, CDK2, and CHK2	SCH900776	Phase I	– Adult
CHK1	LY2606368	Phase I	– Adult
	GDC-0425	Phase I	– Adult
	GDC-0575	Phase I	– Adult
CDK2	SCH727965	Phase I/II	– Adult
Class IV – Drugs targeting oncogenic stabilization of MYCN
mTOR – rapalogs	Rapamycin	Phase I/II		NCT01331135, NCT01625351, NCT01804634
	Temsirolimus	Phase I/II		NCT01204450, NCT00808899
	Everolimus	Phase I		NCT00106353, NCT01049841, NCT01637194
	Ridaforolimus	Phase I		NCT00704054, NCT01431547, NCT01431534
mTOR – ATP competitive	AZD2014	Phase I	– Adult
	OSI027	Phase I	– Adult
	MLN0128	Phase I	– Adult
PI3K/mTOR dual specificity	BKM120	Phase I/II/III	– Adult
	GDC-0980	Phase I/II	– Adult
	NVP-BEZ235	Phase I/II	– Adult
Akt	Perifosine	Phase I		NCT00776867, NCT01049841
	MK2206	Phase I		NCT01231919
Aurora A	AT9283	Phase I		NCT01767194
	MLN8237	Phase I/II		NCT01601535
Class V – Drugs targeting the expression or function of MYCN
MYCN expression	Isotretinoin	Current therapy
Drugs targeting ALK	Drug	Development phase		Pediatric trial identifier
Inhibition of ALK kinase activity
ALK/c-Met dual specificity	Crizotinib	Phase I/II		NCT01644773, NCT00939770
ALK/EGFR dual specificity	AP26113	Phase I/II	– Adult
ALK	LDK378	Phase I		NCT01742286
ALK	CH5424802	Phase I/II	– Adult
Immunotherapy of ALK
ALK	PF-03446962	Phase I/II	– Adult