The Experimental Therapeutics Program collaborates with numerous Mayo Clinic researchers on defined cancer research focus areas.

The program's areas of cancer research include:

  • Clinical trials of new cancer agents
  • Pharmacogenetics
  • Signaling pathways
  • Small molecule inhibitors

Clinical trials of new cancer agents

Once novel agents or combinations of agents are developed and tested in the laboratory, the Experimental Therapeutics Program helps translate findings into clinical practice. The Mayo Clinic Cancer Center's Phase I Clinical Trials Group consists of physicians, nurses, laboratory scientists, research assistants and statisticians who collaborate to perform early clinical testing of novel chemotherapeutic strategies developed at Mayo Clinic and elsewhere.

These clinical trials are designed to assess the safety and efficacy of new treatments. In conjunction with these clinical trials, blood samples or tumor biopsies are often examined to determine the impact of the treatment on the targeted signaling pathway.

After initial clinical trials, some treatments go on for more-extensive clinical testing in phase II and phase III trials conducted in other programs within the Mayo Clinic Cancer Center, through the Mayo Clinic Phase II Consortium, or through the Alliance for Clinical Trials in Oncology.

Faculty members collaborating on clinical trials of new cancer agents include:

Learn more about early cancer clinical trials and the Early Cancer Therapeutics Clinic at Mayo Clinic.


Pharmacogenetics researchers in the Experimental Therapeutics Program focus on the role of genetic inheritance in drug response. They study two aspects of a person's response to drugs: drug response and adverse drug reaction.

Some people with cancer don't get the desired response from a therapeutic drug, for example, while others have a strong negative reaction to it. Several factors may contribute to these reactions, such as age, sex and underlying disease, and cancer faculty members hope their research advances understanding about pharmacogentics in cancer.

Faculty members collaborating on research in pharmacogenetics include:

Signaling pathways

The study of signaling pathways involved in proliferation, repair and programmed cell death (apoptosis) is a major research focus of the Experimental Therapeutics Program. This research offers new insight into the nature of malignant cells and cellular responses to anti-cancer agents.

Research on signaling pathways in cancer can help identify potential new targets for chemotherapy, including treatments that are more selective and less toxic.

Cells duplicate their DNA and divide over and over again with remarkable precision. A complicated series of signals tells cells when to divide and when to stop growing. Sometimes — perhaps once in a billion times — there is a malfunction that damages a cell. When the signaling pathways are working correctly, the damaged cell recognizes that it's defective and either undergoes repair (usually of damaged DNA) or triggers its own death.

Sometimes, though, a damaged cell loses the ability to self-repair or self-destruct. In some cases, this happens because a cell loses the ability to sense damage. Other times, even if the damage is detected, the repair pathways might be altered. Finally, cells might lose the ability to trigger cell death. Any of these changes can contribute to the development of cancer.

Many chemotherapeutic drugs kill cancer cells by damaging DNA or other cellular components, resulting in signals that trigger apoptosis. Therefore, alterations in signaling pathways that contribute to cancer development can also affect cancer therapy.

Faculty members collaborating on research in signaling pathways of cells include:

Small molecule inhibitors

Alterations in signaling pathways involved in DNA repair and apoptosis might contribute to the development of cancer and also affect the ability of tumor cells to be killed by chemotherapeutic approaches. Investigators in the Experimental Therapeutics Program are researching how alterations in these pathways might contribute to chemotherapy resistance.

Researchers are also developing and evaluating inhibitors of these signaling pathways. In some cases, these inhibitors seem to trigger apoptosis and reverse the cancer phenotype of cells with altered pathways. In other cases, the inhibitors themselves do not directly kill cancer cells but sensitize those cells to killing by existing chemotherapeutic agents.

The Experimental Therapeutics Program is also investigating the action of a number of agents that exhibit anti-cancer activity in model systems through mechanisms that are still not completely understood. These studies often provide insight into new, previously unsuspected alterations in cancer cells and improved understanding of the action of the agents under investigation.

Faculty members collaborating on research in small molecule inhibitors include: