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2027 Scholar-Innovator and ADDF-Harrington
Oncology
Novel Pharmacologic Approaches to Target Wild-Type IDH1 in Pancreatic Cancer
2024 Harrington Scholar-Innovator
Pancreatic cancer is a notoriously challenging malignancy with an often-grim prognosis.
A mutant form of the enzyme isocitrate dehydrogenase 1 (IDH1) is a well-established drug target for pancreatic cancer. According to Dr. Winter, however, the “wild-type” enzyme produced by the IDH1 gene—not the mutant type—predominates in 99% of cancers. A wild-type enzyme is the standard form of an enzyme found in nature, possessing its original structure and typical function.
Dr. Winter says the wild-type of IDH1 is especially important for cancer cell survival in the nutrient-limited tumor microenvironment, which is a hallmark of pancreatic cancer. He and his colleagues, therefore, set out to target the wild-type enzyme and have developed a series and a lead compound of wild-type IDH1 inhibitors.
“My clinical and research interests have always been around pancreatic cancer,” Dr. Winter says. “It’s the hardest cancer to treat, it’s the most lethal cancer, and it’s a cancer where there are no effective novel therapies. So there’s a void in the clinical space for novel drugs, and there’s an opportunity to make a big impact.”
The next steps in Dr. Winters’ research include taking the promising data from murine studies of the lead compound and continuing to carry out pre-IND protocols to prepare for the clinic.
“The ultimate goal is to cure pancreatic cancer, but I think it would be a major victory for Harrington Discovery Institute, for my lab, and for this project if our lead compound could progress through clinical trials and demonstrate efficacy in improving survival in patients,” Dr. Winter says.
"We want to help people with this very aggressive disease and provide them with better treatment options. That's what drives us from day to day."