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Tumor Treating Field Therapy in Combination with Bevacizumab for the Treatment of Recurrent Glioblastoma
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ONC201: a new treatment option being tested clinically for recurrent glioblastoma

Marie D Ralff1,2, Amriti R Lulla2,3, Jessica Wagner2,4

  • 1MD/PhD Program, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, USA.

Translational Cancer Research
|September 4, 2018
PubMed

Insights

The experimental drug ONC201 shows early promise for treating recurrent glioblastoma by targeting cancer cell death pathways. Further clinical trials are underway to confirm its efficacy in patients with this aggressive brain tumor.

Area of Science:

  • Neuro-oncology
  • Molecular pharmacology
  • Clinical trial research

Background:

  • Glioblastoma is a highly aggressive brain tumor with poor prognosis.
  • Current treatments (surgery, radiation, temozolomide) offer limited long-term survival, with frequent relapse.
  • There is a critical need for novel therapeutic strategies against glioblastoma.

Purpose of the Study:

  • To evaluate the early efficacy of imipridone ONC201 in patients with recurrent or refractory glioblastoma.
  • To assess the safety and preliminary signs of activity of ONC201 in a phase II clinical trial.

Main Methods:

  • A phase II clinical trial was conducted involving 17 patients with recurrent or refractory glioblastoma.
  • Patients received treatment with the small molecule ONC201.
  • Early results were assessed via futility analysis.

Main Results:

  • ONC201 demonstrated preliminary signs of efficacy in patients with recurrent or refractory glioblastoma.
  • The drug targets the integrated stress response (ISR) pathway, inducing TRAIL and DR5 expression.
  • Early futility analysis suggests potential for further investigation.

Conclusions:

  • ONC201 shows promising preliminary efficacy in a small cohort of glioblastoma patients.
  • The drug's mechanism involves activating pro-death signaling via the ISR pathway.
  • Further clinical evaluation in an expansion cohort is warranted and ongoing.
Keywords:
ONC201clinical trialglioblastoma

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