Table 2.

Summary of the available data for the top five agents identified by the task force.

Agent Rank	Agent	Preclinical Evidence	Adult Clinical Data	Pediatric Clinical Data	Drug Availability	Consensus Decision
In trial	temsirolimus	×	×	×	×	In trial
1	PARP 1 inhibitor/cytotoxic agent	×	×	Ongoing phase 1 of olaparib in combination with temozolomide for EWS 2 and RMS 3 (age ≥ 16 years)	×	Need more preclinical combination studies, need phase 1 combination data
2	MEK 4 inhibitor/IGF1R 5 inhibitor	×	×	×		Need more preclinical testing to determine if doses in in vitro studies are achievable in humans
3	PLX038	×	×			Need preclinical combination studies, need phase 1 pediatric dose
4	HDAC 6 inhibitor	×	×	×	×	Need more preclinical testing in vivo to mimic human PK 7 data
5	eribulin	×	×	Ongoing phase 2 in RMS 3, ongoing phase 1/2 in combination with irinotecan in R/R 8 solid tumors	×	Need phase 2 pediatric data

The top five agents are listed in order of their rank based on votes by the task force members. Areas where the task force felt there was sufficient evidence or data are noted with “×” and areas where there were no available data or insufficient evidence are blank. ¹ Poly ADP-ribose polymerase. ² Ewing sarcoma. ³ Rhabdomyosarcoma. ⁴ Mitogen-activated protein kinase. ⁵ Insulin-like growth factor 1 receptor. ⁶ Histone deacetylase. ⁷ pharmacokinetic. ⁸ Relapsed/refractory.