Year Funded: 2019
“Because sarcomas can occur in different parts of the body and account for only about 15 percent of all childhood cancers, treatments and protocols have been challenging to pinpoint,” says Stephen Gottschalk, MD, chair of Bone Marrow Transplantation and Cellular Therapy at St. Jude Children’s Research Hospital (Memphis, Tenn.).
“Every day when I look around me [at St. Jude Children’s Research Hospital], I see children with life threatening diseases and I never need to ask myself why I’m here.
“But I’m a glass half full kind of guy. Today, 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. With gene transfer and editing technologies, we can make a patient’s immune system much more powerful in recognizing and destroying cancer cells.”
Dr. Gottschalk has invested more than 10 years of his life into exploring and developing new ways to reshape the immune system. His clinical focus on pediatric oncology dovetails with these research pursuits and his passion for achieving greater understanding of how the human immune system works.
With a grant from the Alliance for Cancer Gene Therapy (ACGT), Dr. Gottschalk is conducting preclinical studies with the ultimate goal of developing a clinical trial. His novel strategy leverages an approach to CAR T-cell therapy that yielded promising results in other preclinical models. Using not one, but two different protein targets, Dr. Gottschalk expects to fortify a patient’s immune system to successfully attack and destroy sarcomas along with the blood vessels that support their growth.
“I’ve always been fascinated by how patients who have no other health issues develop cancer. It’s a puzzle that has always motivated me,” says Dr. Gottschalk. “Cell and gene therapies offer great promise. We have seen some outstanding results in recent years and there is much more opportunity ahead.
“Further exploration of immune cell types and how to best combine cell therapies with other therapies will help us improve their effectiveness. 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.”