Dr. Cui studies T cells, a type of immune cells that have the ability to fight off cancer cells. Over time, however, T cells experience “T cell exhaustion,” which occurs when they become tired and lose their effectiveness. Dr. Cui studies the factors that lead to T cell exhaustion and, more importantly, is researching ways to reverse it.

One potential technology involves converting exhausted T cells into energized T cells to treat bladder cancer. Using this new technology, he hopes that some of the reenergized T cells will form T cells with longer memories that recognize cancer cells, helping to protect patients from cancer recurrence and metastasis.

“We are very grateful for the generous support from BCAN, which will propel our research forward in finding new treatment opportunities for cancer patients,” Dr. Cui said.

John Pulikkan, PhD: understanding how leukemia cells mutate

Hematopoiesis—the process by which blood cells are developed—is regulated by several genes. Leukemia develops when the genes necessary for blood cell development mutate and do not develop or function normally. Dr. Pulikkan studies how mutation in blood cells causes leukemia and is working toward developing new treatments to specifically kill leukemic cells.

Traditionally, chemotherapy is the primary treatment option for cancer, including leukemia. In addition to killing cancer cells, chemo can also damage normal, healthy cells, resulting in serious side effects and poor patient outcomes. However, targeted therapies specifically kill cancer cells without affecting normal cells. Recent studies have shown that targeted therapies can provide promising outcomes for patients with leukemia and other cancers.

Dr. Pulikkan’s research focuses on a gene called C/EBPalpha, which is essential for the development of granulocytes, which are white blood cells that help the immune system fight off infection. In many subtypes of leukemia, the C/EBPalpha gene is either mutated or non-functional. Dr. Pulikkan is investigating the mechanism by which C/EBPalpha mutations induce leukemia development.

“If you can develop a targeted therapy for leukemia with C/EBPalpha mutation, you can greatly improve patient survival,” he said. This innovative research will hopefully lead other investigators to develop ways to treat leukemia and other types of cancer.

Sid Rao, MD, PhD: pioneering pediatric leukemia research

The goal of the Rao lab is simple: to develop more effective, less toxic therapies to use in children with acute myeloid leukemia (AML). Children with AML, a cancer of the blood, have a long-term survival of approximately 50%, even though they receive high-dose conventional chemotherapy and often require a bone marrow transplant. Kids who are long-term survivors can often have chronic medical issues involving their kidneys, skin and heart, and experience difficulties with normal growth and in school. This highlights the desperate need for new approaches to both improve survival, but also decrease the long-term medical issues that are a consequence of their treatment.

Dr. Rao’s research group focuses on how different DNA changes, called mutations, combine for AML to develop. The idea is that by understanding how leukemia develops, we can identify key changes that can be targeted using precision oncology approaches.

Nan Zhu, PhD: identifying more effective, less toxic therapies

Dr. Zhu’s lab focuses on the study of acute myeloid leukemia (AML), a cancer of blood and bone marrow. AML occurs when the normal process of blood stem cells’ self-renewal and differentiation breaks down, leading to cancer cells that take over and crowd out normal blood cells. AML prognosis is still poor, with a five-year overall survival rate of about 50%.

The good news is that new, more effective and less toxic targeted therapies are being developed, and many of these have appeared in clinics over the last few years. “The overall goal of my lab is to identify therapeutic targets for AML treatment,” Dr. Zhu said. Her lab focuses on a protein called JMJD1C that has been shown to be required for leukemia cell survival. Current work involves understanding the molecular mechanism of its function in leukemia and using a pre-clinical model to show the feasibility of targeting JMJD1C in AML.

About the experts:

Weiguo Cui, MD, PhD, is an investigator at Versiti Blood Research Institute and associate professor in the Department of Microbiology and Immunology at the Medical College of Wisconsin.

John Pulikkan, PhD, is an associate investigator at Versiti Blood Research Institute and an assistant professor of cell biology, neurobiology and anatomy at the Medical College of Wisconsin.

Sid Rao, MD, PhD, is an investigator at Versiti Blood Research Institute.

Nan Zhu, PhD, is an associate investigator at Versiti Blood Research Institute.