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Repurposing of Drugs in Cancer Treatment: Cancer Research

Repurposing of Drugs in Cancer Treatment: Cancer Research: In a groundbreaking development, researchers have discovered that repurposing old drugs could offer new avenues for cancer treatment. This innovative approach has the potential to overcome treatment resistance, particularly in challenging cases like glioblastoma, a lethal form of brain cancer.

Repurposing of Drugs in Cancer Treatment: Cancer Research
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Repurposing of Drugs in Cancer Treatment: Cancer Research

The Role of Cerebrospinal Fluid (CSF) in Treatment Resistance

Cerebrospinal fluid is a clear, colorless liquid that serves multiple functions, including cushioning the brain and spinal cord, providing nourishment, and aiding in waste removal. However, its role in cancer treatment has been largely overlooked.

The study led by Associate Professor Cedric Bardy at SAHMRI and Flinders University revealed that CSF could be a contributing factor in making brain cancers like glioblastoma more resistant to treatment. The fluid seems to induce a form of “cellular plasticity,” allowing cancer cells to adapt and resist therapies like radiation and chemotherapy. Understanding this mechanism could be crucial for developing new therapeutic strategies.

Trifluoperazine: The Old Drug with New Potential

Trifluoperazine, an anti-anxiety drug that has been in use since the 1950s, emerged as a promising candidate for resensitizing resistant cancer cells. The drug targets a nuclear protein called NUPR1, which plays a role in making cancer cells more resistant to a form of therapy-induced cell death known as ferroptosis.

By inhibiting NUPR1, trifluoperazine could potentially make these cells more susceptible to existing treatments. This is particularly significant because it offers a way to enhance the efficacy of current therapies without having to develop new drugs from scratch, thereby saving time and resources.

Clinical Implications and Future Directions

The findings of this study could have far-reaching implications. If trifluoperazine proves effective in clinical trials, it could be added to the standard care regimen for glioblastoma, improving survival rates for patients suffering from this aggressive form of cancer.

Moreover, the concept of drug repurposing could be extended to other forms of cancer and even rare diseases, offering a faster, more cost-effective route to finding effective treatments.

The Broader Landscape of Drug Repurposing

Drug repurposing is not a new concept; it has been explored in various medical fields. For example, sildenafil, initially developed for treating cardiovascular issues, was repurposed as Viagra for erectile dysfunction. In the realm of rare diseases, drug repurposing offers a way to bypass the lengthy and costly process of new drug development. By reusing existing drugs that have already been approved for other conditions, researchers can significantly shorten the time it takes to bring a new treatment to market.

Other Drugs: Repurposing of Drugs in Cancer Treatment: Cancer Research


  • Original Use: Initially developed as a sedative and antiemetic to combat morning sickness in pregnant women.
  • Repurposed For: Multiple myeloma and other hematologic malignancies.
  • Mechanism: Thalidomide modulates the immune system and inhibits angiogenesis, thereby preventing the growth of cancer cells.
  • Current Status: Approved for use in multiple myeloma and showing promise in clinical trials for other cancers.


  • Original Use: Widely used as an analgesic, antipyretic, and anti-inflammatory drug.
  • Repurposed For: Colorectal cancer prevention.
  • Mechanism: Aspirin inhibits the cyclooxygenase (COX) enzyme, reducing the production of prostaglandins that promote inflammation and cancer growth.
  • Current Status: Ongoing research to determine optimal dosing and target population for cancer prevention.


  • Original Use: Primarily used for the treatment of Type 2 diabetes.
  • Repurposed For: Various types of cancer, including breast and pancreatic cancers.
  • Mechanism: Metformin activates AMPK, leading to inhibition of the mTOR pathway, thereby halting cancer cell proliferation.
  • Current Status: Numerous clinical trials are underway to evaluate its efficacy as an adjunct to standard cancer therapies.


  • Original Use: Originally developed as a beta-blocker for treating hypertension and anxiety.
  • Repurposed For: Treatment of angiosarcoma, a rare form of cancer.
  • Mechanism: Propranolol inhibits beta-adrenergic receptors, which are involved in angiogenesis and metastasis.
  • Current Status: Early-phase clinical trials have shown promising results, and further studies are in progress.


  • Original Use: Used for treating chronic alcoholism.
  • Repurposed For: Treatment of glioblastoma and other solid tumors.
  • Mechanism: Disulfiram targets the proteasome and NF-kB pathway, leading to cancer cell death.
  • Current Status: Preclinical studies have shown promise, and it is currently being evaluated in clinical trials.