ONC201 May Help Treat Refractory Cancer

Researchers at Oncoceutics, Inc., have identified a molecule known as ONC201 that could help treat refractory cancer. ONC201 alters the gene expression of cancer stem cells and prevents them from self-renewing. Cancer stem cells play a key role in cancer recurrence and lack of response to chemoradiation treatments.

Cancer Stem Cells and ONC201

Cancer stem cells are associated with poor survival rates due to recurrence and metastasis. Researchers have been studying the effects of inhibiting the renewal of these cells to treat cancer. Studies have shown that a molecule called ONC201 specifically targets stem cells that give rise to colorectal cancer and prevents their self-renewal. However, it is still unclear how ONC201 prevents gene expression in cancer stem cells.

Gene Expression Analysis for Colorectal Cancer

Varun Vijay Prabhu and colleagues at Oncoceutics, Inc., USA conducted an analysis of the gene expression of stem cells lines that give rise to glioblastoma, prostate cancer, and colorectal cancer. Tissues derived from patients suffering from these cancers were treated with the molecule ONC201. The molecule effectively killed the tumor cells by disrupting the signaling pathways of these cancer stem cells. This also provided the researchers with a measurable dynamic biomarker to track response.

Cancer Treatment with Stem Cells

Not all tumors can be treated with ONC201. Certain cancer cells acquire resistance to this molecule. The scientists suggest that before treatment with ONC201, patients may undergo gene expression analysis and a signature could be used to predict whether the cancer will respond to ONC201.

Glioblastoma Trial with ONC201

The NCI has awarded a grant to Oncoceutics to study the effect of ONC201 on recurrent glioblastoma. This ongoing study at the Dana Farber Cancer Institute and Massachusetts General Hospital will now be expanded to enroll more patients. Initially, 17 patients were enrolled at both institutions combined. All the patients enrolled in the study had a diagnosis of glioblastoma with at least one recurrence after treatment with standard chemoradiation therapy.

The trial is being expanded to enroll an additional 36 patients based on initial positive results. Early results indicate decreased disease burden, improved clinical activity, and a good safety profile for treatment with ONC201. Moreover, the dosing regimen for the drug is patient-friendly. Scientists are eagerly awaiting the results of this study.

ONC201 offers a ray of hope to patients with glioblastoma recurrence following temozolomide and radiation therapy. Brain cancers are typically complex and difficult to treat due to the limitations imposed on drug delivery by the blood-brain barrier. If successful, ONC201 will signal the end of decades of stagnation in new therapies for brain cancers.


  1. https://eurekalert.org/pub_releases/2017-08/p-omi072617.php
  2. https://cancerletter.com/articles/20170125_7/

Mira Swave, MD

Contributor at Regenerative Medicine Now

Mira Swave, M.D. is a specialist in the field of Regenerative Medicine.
Share the knowledge

Leave a Reply

Your email address will not be published. Required fields are marked *

Get our newsletter for the latest news & updates.

Share the knowledge: