Reverse repurposing: Potential utility of cancer drugs in nonmalignant illnesses

Area of Science:

• Oncology
• Genomics
• Pharmacology

Background:

• Dysregulation in growth and immune processes contributes to both cancer and nonmalignant diseases.
• Genomic alterations in nonmalignant conditions often resemble those targeted in cancer therapy.
• Targeted cancer therapies may offer new treatment avenues for benign diseases with similar molecular drivers.

Purpose of the Study:

• To explore the therapeutic potential of repositioning oncology agents for nonmalignant diseases.
• To identify shared molecular targets between cancer and benign conditions.
• To highlight the possibility of bridging cancer and benign disease treatments.

Main Methods:

• Review of genetic pathways implicated in both cancer and noncancerous conditions (e.g., MEK, PI3K/AKT/mTOR, FGFR, NRG1/ERBB).
• Identification of targeted compounds and inhibitors used in cancer therapy.
• Assessment of established or theoretical therapeutic potential for benign diseases.

Main Results:

• Several pathways (MEK, PI3K/AKT/mTOR, FGFR, NRG1/ERBB) are involved in both malignant and benign conditions.
• Targeted antagonists and inhibitors (e.g., BTK inhibitors, JAK inhibitors, CD20 antibodies) show potential for cross-disease application.
• Pharmacologically tractable cancer drivers are implicated in nonmalignant disorders like Alzheimer's, neurodegenerative diseases, rheumatoid arthritis, dwarfism, and endometriosis.

Conclusions:

• Oncology agents targeting specific genetic drivers may be repurposed for treating serious nonmalignant diseases.
• Expanded repositioning of cancer drugs offers a promising strategy for addressing unmet medical needs in benign conditions.
• This approach holds potential to benefit a wide spectrum of nonmalignant disorders.