Venetoclax – new treatment for colorectal cancer

Colorectal cancer (CRC) is the world’s fourth most deadly cancer with almost 900.000 deaths annually and is the third most common type of cancer, accounting for 10,6% of the cases in the world. One of the most common complication events in digestive tumours is venous thromboembolic events, attributed to a combination of clinical and biological factors and an increased morbidity and mortality. Biomarkers, such as Bcl-2 proteins or hepsin are crucial in the detection and early diagnosis, and sometimes they are intrinsically related to specific complications in the development of cancer. 

The inventor team at the Catholic University of Murcia (UCAM) has identified a new indication of Venetoclax -a drug currently used in leukemia treatment- for colorectal cancer treatment, which has been shown to inhibit the proteolytic activity of hepsin and reduce cell migration and invasion of colorectal cancer cells. Venetoclax (VTC) is a small molecule selective inhibitor of the antiapoptotic protein Bcl-2 that has been proved to promote programmed cell death of chronic lymphocytic leukemia cells regulating the release of proapoptotic factors.

The new therapeutic effects of VTC rely on its reduction of cell migration and invasion, which are linked with the proliferation of metastatic nodes and the reduction of thrombin genesis. It has been proven that these effects are not linked to the reduction of proliferation and the enhancing of cancer cell death, showing its robust therapeutic potential in the treatment of colorectal cancer.

This novel drug indication brings many benefits:

  • Irreversible inhibitor of hepsin. The use of VTC preserves the extracellular matrix integrity by inhibiting hepsin proteolytic action and reducing its tumour activation signaling.
  • Reduces cell migration and cell invasion, in vitro results have shown the efficacy of VTC use in tumoural tissues.
  • Reduces thrombin generation. VTC reduces the velocity and the amount of thrombin generated, which is directly related to platelet aggregation and blood clotting.
  • Does not affect cell cycle, anti-migration and anti-invasion effects of VTC unrelated to cell death or less cell proliferation.
  • FDA approved in 2020 for leukemia treatment, proving its security for humans.
  • Indicated for gastrointestinal cancers, preferably indicated for colorectal cancer.
  • Reduces in vivo invasion in a zebrafish model.

The represented institutions are looking for a collaboration that leads to commercial exploitation of the presented invention.

Institution: Universidad Católica de Murcia and Instituto Murciano de Investigación Biomédica Virgen de la Arrixaca

TRL: 3-4

Protection status: Patent Application

Contact: Noelia Mas /