Table 5.

Biological/targeted therapies in DIPG.

Target	Agent	Newly diagnosed or progressive/relapsed, number of patients (n)	Median PFS (months)	PFS at 6 months (%)	Reference
EGFR	Erlotinib	Newly diagnosed with RT	8	90	Geoerger et al. (115)
	Gefitinib	Newly diagnosed with RT (n = 43)	7.4	88	Pollack et al. (116)
		Newly diagnosed with RT (n = 20)	NR	48	Geyer et al. (117)
	Nimotuzumab	Newly diagnosed with RT (n = 41)	5.5	NR	Massimino et al. (118)
		Progressive/relapsed (n = 44)	1.7	NR	Bartels et al. (119)
EGFR/VEGFR	Vandetanib	Newly diagnosed with RT (n = 21)	NR	88	Broniscer et al. (120)
PDGFRA	Imatinib	Newly diagnosed with RT	NR	70	Pollack et al. (121)
VEGF	Bevacizumab	Progressive/relapsed (n = 31) Progressive/relapsed (n = 2) Newly diagnosed with RT (n = 14)	2.3 2.25 8.8	9.7 NR NR	Gururangan et al. (122) Narayana et al. (123) Salloum et al. (124)
mTOR	Temsirolimus	Progressive/relapsed (n = 5)	2.5	NR	Geoerger et al. (125)
Farnesyl transferase	Tipifarnib	Newly diagnosed with RT (n = 40)	NR	44	Haas-Kogan et al. (126)
Integrins (αvβ3 and αvβ5)	Cilengitide	Progressive/relapsed (n = 31)	NR	NR	MacDonald et al. (127)
Histone de-acetylase (HDAC)	Valproic acid Panabinostat	Newly diagnosed with RT/chemotherapy Murine DIPG models	9.5 –	NR –	Felix et al. (128) Grasso et al. (129)

PFS, progression free survival; RT, radiation therapy; NR, not recorded.