Stephen Gottschalk, MD

Many children, who are diagnosed with bone or muscle tumors (sarcomas), which have spread to more than one site in their body, cannot be cured. Thus, there is an urgent need to develop new therapies.  

We propose to develop a therapy, called immunotherapy, which uses the patient’s own immune system to destroy their sarcoma. Immunotherapies comes in many different forms, and this grant is focused on cell therapy, which consists of taking immune cells from patients, genetically engineering them to recognize and kill tumor cells, and then infusing cells back into the patient.  

Cell therapy has already been very effective for certain types of blood cancers. However, the activity of immune cell therapy against sarcoma and other solid tumors has been limited. In this Alliance for Cancer Gene Therapy funded research we will develop a novel approach to attack sarcomas with genetically engineered immune cells, which relies on attacking not only the cancer cell but also the supporting blood vessels, which are critical for tumor growth.  

In addition, we will explore if broadening the attack to target not only one, but two proteins present on sarcomas improves the anti-sarcoma activity of immune cells. State of the art technologies will be used, and we will test the anti-sarcoma activity of our immune cells in models that closely mimic human disease. At the conclusion of the research, we expect to have optimized our immune cell therapy approach for sarcoma and based on our results are planning to develop a clinical study.  

This Alliance for Cancer Gene Therapy Research Fellow is funded in part by Swim Across America and Wendy Walk.

“Our ability to re-engineer immune cells by inserting genes or manipulating the human genome has the potential to revolutionize how we take care of cancer patients…I believe we will see smart combination therapies become fundamental in the fight against cancer. These combinations may include more than one type of immunotherapy, conventional therapies such as surgery, chemotherapy and/or radiation, and/or novel small molecule inhibitors.”