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Neuroendocrine and Rare Tumor Advances: A New and Promising TRAIL Emerges

Dwight H Owen1, Nikolaos A Trikalinos2

  • 1Division of Medical Oncology, The James Comprehensive Cancer Center, Columbus, Ohio.

Clinical Cancer Research : an Official Journal of the American Association for Cancer Research
|May 2, 2022
PubMed

Insights

ONC201, an oral drug, shows promise for treating rare pheochromocytomas-paragangliomas. This phase II study found it well-tolerated with notable efficacy, though further research is needed for activity biomarkers.

Area of Science:

  • Pharmacology
  • Oncology
  • Neuroendocrinology

Background:

  • ONC201 is an oral selective dopamine D2 receptor antagonist and caseinolytic protease P activator.
  • A phase II study investigated ONC201 in patients with pheochromocytomas-paragangliomas, a type of rare neuroendocrine neoplasm.

Discussion:

  • The study demonstrated that ONC201 was well-tolerated in patients.
  • Notable efficacy was observed in the treatment of pheochromocytomas-paragangliomas.
  • Biomarkers for ONC201 activity in this patient population require further elucidation.

Key Insights:

  • ONC201 exhibits therapeutic potential for rare neuroendocrine neoplasms.
  • The drug is orally administered and shows a favorable safety profile.
  • Further investigation is necessary to identify predictive biomarkers for treatment response.

Outlook:

  • Future research should focus on identifying biomarkers to personalize ONC201 treatment.
  • Expanding clinical trials may confirm the efficacy and safety of ONC201 in a larger patient cohort.
  • ONC201 could represent a novel therapeutic option for patients with limited treatment alternatives.

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