Developing New Pancreatic Cancer Drugs: From Laboratory to Patient Use

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Every day, researchers are working to develop new and more effective treatments for pancreatic cancer. But how does a drug studied in the laboratory become a drug that patients can use? The short answer is that after extensive laboratory testing, the drug must go through rigorous clinical trial testing to ensure safety and effectiveness.

An Overview of the Research Process
There are three stages of pancreatic cancer biomedical research: basic science, translational research, and clinical research. Basic scientific research provides a better understanding of a disease’s biology, such as genetic changes, protein alterations, and the interactions between the cells that cause tumors to grow and develop. Translational research is a bridge between basic science in the laboratory and studies in a clinical setting with patients. It determines whether ideas about disease biology can benefit patients. Translational research often involves working with tumor tissue samples or animals before the idea is tested in patients. Finally, clinical research tests novel therapies or diagnostic tools in studies that involve pancreatic cancer patients.

A Hypothetical Example: Drug Targeting Mutant K-ras
Let’s look at a hypothetical drug and the journey it must take from a scientist’s idea to becoming an approved treatment option for pancreatic cancer. A scientist strategizes that blocking the activity of a mutated protein called K-ras could be a  promising way to stop or slow the growth of pancreatic tumors. K-ras has been shown to be mutated and activated in nearly all pancreatic cancer cases, and the tumor is thought to depend on mutant K-ras activity to grow and survive. So, if a drug could stop the activity of mutant K-ras, perhaps it could stop the tumor from growing.

  1. Basic Science: Researchers first need to understand the biology of K-ras mutation and the effect it has on cancer cells. Examples of discoveries might include understanding the difference between mutant and normal K-ras, determining the frequency of K-ras mutation in pancreatic cancer cases, and finding out how K-ras mutation influences pancreatic cancer cells’ growth and survival. All of these basic science experiments are critical to validate the scientist’s idea that targeting K-ras could make a difference.
  2. Translational Research: Translational research often requires collaboration with other scientists. For example, in this case, a researcher with a strong chemistry background would be needed to decide how to stop the activity of mutant K-ras and to help design the actual drug. Usually multiple candidate drugs are made and compared to one another to find which are the most effective. Experiments would be done on pancreatic cancer cells in a dish to help determine if the new drug kills these cancer cells. Tests would also be done on cells from a healthy pancreas to make sure that the drug does not negatively impact healthy cells. Next, mice or other animal models of pancreatic cancer would be tested to make sure the drug is effective at stopping or slowing the growth of the tumor and does not cause any unwanted side effects.
  3. Clinical Trials: The final step in getting a drug from laboratory to patient use involves careful testing with pancreatic cancer patients. This testing is done by enrolling patients into trials examining the drug that has already gone through extensive laboratory and animal studies. The clinical trials determine the safest and most effective dose of the drug and also confirm that the new drug is an improvement over the standard options for treating pancreatic cancer.

The Pancreatic Cancer Action Network encourages all patients to consider clinical trials when exploring options at any stage of treatment. For information about clinical trials or for a personalized clinical trials search, please call our PanCAN Patient Services toll-free at 877-272-6226 or complete our contact form.