Drug Repurposing in Oncology: How Existing Medicines Are Fighting Cancer

By Editorial Team
Cancer remains one of the most complex and challenging diseases globally, demanding innovative therapeutic strategies. One promising approach gaining momentum is drug repurposing — the use of existing, approved medications originally developed for non-cancer conditions to treat various cancers. This strategy leverages known safety profiles and pharmacology of these drugs, potentially accelerating cancer treatment development while reducing costs.

What Is Drug Repurposing?

Drug repurposing, also called drug repositioning, involves identifying new therapeutic uses for medications already approved for other diseases. Instead of developing a new drug from scratch, which can take over a decade and billions of dollars, repurposed drugs can move more quickly into clinical trials for cancer due to established safety and dosing data.

Examples of Repurposed Drugs in Cancer Therapy

Several well-known drugs have been successfully repurposed or are under active investigation for cancer treatment:

  • Thalidomide: Originally prescribed for morning sickness but withdrawn due to teratogenic effects, thalidomide was later found to have anti-angiogenic and immunomodulatory properties. It is now used to treat multiple myeloma and other blood cancers under strict regulation.

  • Metformin: A widely used anti-diabetic drug, metformin lowers insulin levels, which is significant because hyperinsulinemia can promote cancer growth. Metformin has shown potential in reducing cancer risk and improving outcomes in various cancers.

  • Itraconazole: An antifungal agent, itraconazole exhibits anti-angiogenic effects and has demonstrated activity against lung, breast, ovarian, prostate, and pancreatic cancers, either alone or combined with chemotherapy.

  • Penfluridol: An antipsychotic drug studied for its anti-cancer effects in melanoma, glioblastoma, breast, pancreatic, and lung cancers. It induces apoptosis and autophagy, reduces invasiveness by downregulating integrins, and enhances chemotherapy efficacy.

  • Clotrimazole: Another antifungal with potential to disrupt tumor cell cycles, currently being explored for anticancer properties.

These examples highlight the diverse range of drugs—from antipsychotics to antifungals and diabetes medications—that can be repurposed for oncology.

Advantages of Drug Repurposing in Oncology

  • Cost-Effectiveness: Repurposed drugs are often off-patent generics, significantly lowering treatment costs compared to novel cancer drugs.

  • Safety Profiles Established: Since these drugs are already approved for human use, their toxicity, dosing, and side effects are well documented, reducing early-stage trial risks.

  • Faster Development: The drug development timeline is shortened because preclinical safety assessments are largely complete.

  • Combination Therapy Potential: Repurposed drugs can be combined with existing chemotherapies or immunotherapies to enhance efficacy and overcome drug resistance.

Challenges and Barriers

Despite its promise, drug repurposing faces several hurdles:

  • Limited Commercial Incentives: Many repurposed drugs are generic, offering low financial returns for pharmaceutical companies, which limits investment in expensive clinical trials.

  • Scientific Validation Needed: While preclinical data are promising, robust clinical trials are essential to confirm efficacy and safety in cancer patients.

  • Bioavailability Issues: Some repurposed drugs have poor absorption or distribution in the body, requiring reformulation, such as nanoparticle delivery systems, to improve therapeutic levels.

  • Regulatory and Socio-Political Factors: Navigating regulatory approval for new indications and gaining acceptance in clinical practice can be complex.

Innovative Approaches to Accelerate Repurposing

Projects like the Repurposing Drugs in Oncology (ReDO) initiative work to identify promising candidates and facilitate their transition into clinical trials. Nanotechnology is also being explored to enhance drug delivery and overcome pharmacokinetic limitations.

Research teams worldwide are investigating repurposed drugs targeting critical cancer pathways, such as the TGF-beta signaling in lung cancer, aiming to prevent tumor growth and resistance.

The Future of Drug Repurposing in Cancer Treatment

Drug repurposing holds transformative potential to expand cancer treatment options with more affordable, safer, and effective therapies. As research advances, integrating repurposed drugs into combination regimens and personalized medicine approaches may improve patient outcomes.

However, realizing this potential requires coordinated efforts among researchers, clinicians, regulatory bodies, and funding agencies to overcome scientific, financial, and regulatory challenges.

In summary, drug repurposing is a promising frontier in oncology, turning familiar medicines into new weapons against cancer. With continued innovation and collaboration, this approach could significantly enhance cancer care accessibility and efficacy in the near future.

References:

  • AMDI Newsletter, 2022: Repurposing generic drugs for cancer treatment1

  • Nature, 2024: Drug repurposing for cancer therapy2

  • German Centre for Lung Research, 2025: Drug repurposing in lung cancer3

  • PMC, 2025: Drug repurposing for cancer treatment: current and future directions4

  • MDPI, 2023: Drug Repurposing for Cancer Treatment: A Comprehensive Review Cancer remains one of the most complex and challenging diseases globally, demanding innovative therapeutic strategies. One promising approach gaining momentum is drug repurposing — the use of existing, approved medications originally developed for non-cancer conditions to treat various cancers. This strategy leverages known safety profiles and pharmacology of these drugs, potentially accelerating cancer treatment development while reducing costs.

    What Is Drug Repurposing?

    Drug repurposing, also called drug repositioning, involves identifying new therapeutic uses for medications already approved for other diseases. Instead of developing a new drug from scratch, which can take over a decade and billions of dollars, repurposed drugs can move more quickly into clinical trials for cancer due to established safety and dosing data.

    Examples of Repurposed Drugs in Cancer Therapy

    Several well-known drugs have been successfully repurposed or are under active investigation for cancer treatment:

    • Thalidomide: Originally prescribed for morning sickness but withdrawn due to teratogenic effects, thalidomide was later found to have anti-angiogenic and immunomodulatory properties. It is now used to treat multiple myeloma and other blood cancers under strict regulation.

    • Metformin: A widely used anti-diabetic drug, metformin lowers insulin levels, which is significant because hyperinsulinemia can promote cancer growth. Metformin has shown potential in reducing cancer risk and improving outcomes in various cancers.

    • Itraconazole: An antifungal agent, itraconazole exhibits anti-angiogenic effects and has demonstrated activity against lung, breast, ovarian, prostate, and pancreatic cancers, either alone or combined with chemotherapy.

    • Penfluridol: An antipsychotic drug studied for its anti-cancer effects in melanoma, glioblastoma, breast, pancreatic, and lung cancers. It induces apoptosis and autophagy, reduces invasiveness by downregulating integrins, and enhances chemotherapy efficacy.

    • Clotrimazole: Another antifungal with potential to disrupt tumor cell cycles, currently being explored for anticancer properties.

    These examples highlight the diverse range of drugs—from antipsychotics to antifungals and diabetes medications—that can be repurposed for oncology.

    Advantages of Drug Repurposing in Oncology

    • Cost-Effectiveness: Repurposed drugs are often off-patent generics, significantly lowering treatment costs compared to novel cancer drugs.

    • Safety Profiles Established: Since these drugs are already approved for human use, their toxicity, dosing, and side effects are well documented, reducing early-stage trial risks.

    • Faster Development: The drug development timeline is shortened because preclinical safety assessments are largely complete.

    • Combination Therapy Potential: Repurposed drugs can be combined with existing chemotherapies or immunotherapies to enhance efficacy and overcome drug resistance.

    Challenges and Barriers

    Despite its promise, drug repurposing faces several hurdles:

    • Limited Commercial Incentives: Many repurposed drugs are generic, offering low financial returns for pharmaceutical companies, which limits investment in expensive clinical trials.

    • Scientific Validation Needed: While preclinical data are promising, robust clinical trials are essential to confirm efficacy and safety in cancer patients.

    • Bioavailability Issues: Some repurposed drugs have poor absorption or distribution in the body, requiring reformulation, such as nanoparticle delivery systems, to improve therapeutic levels.

    • Regulatory and Socio-Political Factors: Navigating regulatory approval for new indications and gaining acceptance in clinical practice can be complex.

    Innovative Approaches to Accelerate Repurposing

    Projects like the Repurposing Drugs in Oncology (ReDO) initiative work to identify promising candidates and facilitate their transition into clinical trials. Nanotechnology is also being explored to enhance drug delivery and overcome pharmacokinetic limitations.

    Research teams worldwide are investigating repurposed drugs targeting critical cancer pathways, such as the TGF-beta signaling in lung cancer, aiming to prevent tumor growth and resistance.

    The Future of Drug Repurposing in Cancer Treatment

    Drug repurposing holds transformative potential to expand cancer treatment options with more affordable, safer, and effective therapies. As research advances, integrating repurposed drugs into combination regimens and personalized medicine approaches may improve patient outcomes.

    However, realizing this potential requires coordinated efforts among researchers, clinicians, regulatory bodies, and funding agencies to overcome scientific, financial, and regulatory challenges.

    In summary, drug repurposing is a promising frontier in oncology, turning familiar medicines into new weapons against cancer. With continued innovation and collaboration, this approach could significantly enhance cancer care accessibility and efficacy in the near future.

    References:

    • AMDI Newsletter, 2022: Repurposing generic drugs for cancer treatment1

    • Nature, 2024: Drug repurposing for cancer therapy2

    • German Centre for Lung Research, 2025: Drug repurposing in lung cancer3

    • PMC, 2025: Drug repurposing for cancer treatment: current and future directions4

    • MDPI, 2023: Drug Repurposing for Cancer Treatment: A Comprehensive Review (5)

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