Table 2 Epigenetic targeted therapy in GBM clinical trials

From: From signalling pathways to targeted therapies: unravelling glioblastoma’s secrets and harnessing two decades of progress

Therapy

Target

Phase

Indication

No. Patients

Treatment

mOS (weeks)

mPFS (weeks)

PFS6 (%)

PFS12 (%)

Best response to treatment

Ref

PD

SD

PR

CR

Azacytidine

DNMT1

I/II

IDH-mutant glioma

5

Azacytidine + Olutasidenib

 

8.29

    

0

NCT03684811

Trotabresib

BET

I

Newly diagnosed

18

Trotabresib + RT/TMZ

 

33.1

57.8

 

2

14

 

1

344

Belinostat

Pan-HDAC

II

Newly diagnosed

13

Belinostat + RT/TMZ

80.5

40.5

84

     

348

Panobinostat

Pan-HDAC

II

Recurrent

24

Panobinostat + Bevacizumab

39.2

21.8

30.4

 

3

14

7

0

349

Vorinostat

Pan-HDAC

I/II

Newly diagnosed

107

Vorinostat + RT/TMZ

70

34.8

      

350

II

Recurrent

66

Vorinostat

24.8

48.7

15.2

   

2

351

II

Recurrent

40

Vorinostat + Bevacizumab

45.2

16.1

30

   

9

 

352

II

Recurrent

44

Vorinostat + Bevacizumab

33.9

16.1

26

10

    

354

I/II

Recurrent

39

Vorinostat + Bevacizumab + TMZ

54.4

29.1

53.8

20.5

 

19

15

2

353

II

Recurrent

37

Vorinostat + Bortezomib

13.9

6.5

0

 

32

 

1

 

355

Valporic acid

Class I/IIa HDAC

II

Newly diagnosed

37

Valporic acid + RT/TMZ

129

45.7

70

38

    

362

  1. CR complete response, BET bromodomain and extraterminal-containing protein family, DNMT DNA methyl transferase, HDAC histone deacetylase, mOS median overall survival, mPFS median progression-free survival, PD progressive disease, PFS6 progression-free survival at 6 months, PFS12 progression-free survival at 12 months, PR partial response, RT radiotherapy, SD stable disease, TMZ temozolomide
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