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John Pulikkan, PhD

What's in a gene?

Milwaukee – March 19, 2019

Associate Investigator John Pulikkan, PhD, hopes to develop targeted therapies to treat acute myeloid leukemia.


Typically, cancer patients—whether they have lung cancer or leukemia—are treated with chemotherapy. Chemo’s aim is to attack harmful cancer cells; however, it also kills normal, healthy cells, leaving patients susceptible to infection and other harmful side effects. What if there were a way to specifically target cancer cells, without putting healthy cells at risk?

Finding a new way to treat diseases like cancer is not a simple process. First, investigators must identify which genes in the human body are mutating and causing the disease. Second, they must discover an inhibitor (in most cases, a drug) that will prevent or cure the disease. Finally, they must ensure this inhibitor has no toxic side effects and that it is safe for patients to use.

Associate Investigator John Pulikkan, PhD, may be new to the Blood Research Institute (BRI), but his expertise in identifying inhibitors precedes him. During his postdoctoral work at the University of Massachusetts Medical School, Dr. Pulikkan studied how mutations in the DNA of blood cells direct leukemia formation. In collaboration with researchers at the University of Virginia. Dr. Pulikkan co-discovered AI-10-49, a small molecule inhibitor of the aberrant gene CBFβ-SMMHC, which causes acute myeloid leukemia (AML). He tested this inhibitor against leukemic samples to show that the inhibitor could be a potential treatment option for patients with leukemia.

Now, Dr. Pulikkan brings that expertise to the Versiti Blood Research Institute, where he will study CEBPA, a gene in the human body that is mutated in acute myeloid leukemia. Little is known about CEBPA mutations in leukemia, and very few labs are working on it. Dr. Pulikkan is working hard to understand how it mutates and causes leukemia.

For investigators, identifying the genes that mutate and cause cancer is relatively easy. But in order to develop new treatment methods for patients, investigators need to better understand what causes them to mutate in the first place, which is more challenging. Without knowing how and why these mutations cause leukemia, it’s difficult to develop a treatment that will solely attack leukemia cells, leaving healthy cells be. “If we know this, it will help us design drugs that will fix the problem,” Dr. Pulikkan says. Once investigators have identified how the “problem” genes cause AML, they can begin work on targeted therapies that would eventually be taken into clinical trials.

Eventually, Dr. Pulikkan would like to develop drugs that are specially targeted to the mutated leukemia cells, so that the drugs would have no side effects—sparing patients the potentially harmful side effects of chemotherapy. This innovative research will hopefully help other investigators develop ways to treat other forms of cancer, and not just blood cancers like acute myeloid leukemia.

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