Related Experiment Video
Updated: Jan 20, 2026

Enrichment and Characterization of the Tumor Immune and Non-immune Microenvironments in Established Subcutaneous Murine Tumors
Published on: June 7, 2018
Small molecule immunomodulation: the tumor microenvironment and overcoming immune escape
Arsen Osipov1, May Tun Saung1, Lei Zheng1
1Department of Oncology, The Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Abstract:
Immunotherapy has led to a paradigm shift in the treatment of many advanced malignancies. Despite the success in treatment of tumors like non-small cell lung cancer (NSCLC) and melanoma, checkpoint inhibition-based immunotherapy has limitations. Many tumors, such as pancreatic cancer, are less responsive to checkpoint inhibitors, where patients tend to have a limited duration of benefit and where clinical responses are more robust in patients who are positive for predictive biomarkers. One of the critical factors that influence the efficacy of immunotherapy is the tumor microenvironment (TME), which contains a heterogeneous composition of immunosuppressive cells. Myeloid-derived suppressor cells (MDSCs) and tumor-associated macrophages (TAMs) alter the immune landscape of the TME and serve as facilitators of tumor proliferation, metastatic growth and immunotherapy resistance. Small molecule inhibitors that target these components of the TME have been developed. This special issue review focuses on two promising classes of immunomodulatory small molecule inhibitors: colony stimulating factor-1 receptor (CSF-1R) and focal adhesion kinase (FAK). Small molecule inhibitors of CSF-1R reprogram the TME and TAMs, and lead to enhanced T-cell-mediated tumor eradication. FAK small molecule inhibitors decrease the infiltration MDSCs, TAMs and regulatory T-cells. Additionally, FAK inhibitors are implicated as modulators of stromal density and cancer stem cells, leading to a TME more conducive to an anti-tumor immune response. Immunomodulatory small molecule inhibitors present a unique opportunity to attenuate immune escape of tumors and potentiate the effectiveness of immunotherapy and traditional cytotoxic therapy.
Insights
Small molecule inhibitors targeting colony stimulating factor-1 receptor (CSF-1R) and focal adhesion kinase (FAK) show promise in overcoming immunotherapy resistance by reprogramming the tumor microenvironment (TME) and enhancing anti-tumor immunity.
Area of Science:
- Oncology
- Immunology
- Pharmacology
Background:
- Immunotherapy has revolutionized advanced cancer treatment, but efficacy varies, with tumors like pancreatic cancer showing limited response to checkpoint inhibitors.
- The tumor microenvironment (TME) contains immunosuppressive cells, including myeloid-derived suppressor cells (MDSCs) and tumor-associated macrophages (TAMs), which hinder anti-tumor immune responses and contribute to immunotherapy resistance.
- Developing strategies to modulate the TME is crucial for improving patient outcomes in various advanced malignancies.
Purpose of the Study:
- This review focuses on immunomodulatory small molecule inhibitors targeting CSF-1R and FAK as potential strategies to enhance immunotherapy efficacy.
- To explore how CSF-1R and FAK inhibitors reprogram the TME and influence immune cell populations.
- To assess the potential of these small molecule inhibitors in overcoming immune escape and potentiating both immunotherapy and traditional cytotoxic therapies.
Main Methods:
- Review of current literature on small molecule inhibitors targeting CSF-1R and FAK in cancer.
- Analysis of the mechanisms by which CSF-1R and FAK inhibitors modulate the TME, including immune cell infiltration and stromal components.
- Examination of preclinical and clinical data regarding the efficacy of these inhibitors in combination with other cancer therapies.
Main Results:
- Small molecule inhibitors of CSF-1R can reprogram TAMs within the TME, promoting T-cell-mediated tumor eradication.
- FAK small molecule inhibitors have been shown to reduce the infiltration of immunosuppressive cells like MDSCs, TAMs, and regulatory T-cells.
- FAK inhibitors also modulate stromal density and cancer stem cells, creating a more permissive TME for anti-tumor immune responses.
Conclusions:
- CSF-1R and FAK inhibitors represent promising therapeutic avenues for overcoming immunotherapy resistance by targeting key components of the immunosuppressive TME.
- These small molecule inhibitors offer a strategy to enhance the effectiveness of existing immunotherapies and traditional cytotoxic treatments.
- Targeting the TME with immunomodulatory small molecules holds significant potential for improving treatment outcomes in patients with advanced malignancies unresponsive to current therapies.
Related Concept Videos
The Tumor Microenvironment
Escape Velocity
To calculate the escape velocity, it is assumed that no energy is lost to any frictional forces. In practice, a satellite...
Escape Velocities of Gases
What is the Immune System?
Bioavailability Enhancement: Determination and Conceptual Approaches in Overcoming Bioavailability Problems
Molecules and Compounds

