TGF-β-targeting antisense oligonucleotides showed improved survival compared to historical controls of recurrent and refractory high-grade glioma in phase I/II, but the phase III trials were terminated due to the inclusion of temozolomide (TMZ) in the standard-of-care in GBM therapy and resulting lack of patient accrual. The gene discussed is TGFB1; the disease is central nervous system cancer.