A new publication, authored by a trio of PanCAN leaders, celebrates the current moment in pancreatic cancer research as one “galvanized” for rapid progress in the next decade, thanks in large part to innovative clinical trials poised to bring new treatments to patients more efficiently.

Clinical trials are research studies that investigate new treatments or new combinations of treatments. Every treatment available today was approved through a clinical trial. Typically, clinical trials compare one investigational treatment to the standard of care for one patient population. It’s a model that over time has yielded important discoveries for a range of cancer types. Although there have been a few recent advances for pancreatic cancer, including several targeting specific genetic alterations, some of the biological characteristics of this disease have made it more difficult to treat.

The new paper, published in the Journal of Cancer Metastasis and Treatment, outlines clinical trial innovations that take on the challenge that pancreatic cancer poses to drug development.

The authors, including PanCAN Director of Scientific Communications Allison Rosenzweig, PhD, PanCAN Senior Director of Clinical Trial Portfolio and Program Management Cassadie Moravek and PanCAN Chief Science Officer Lynn Matrisian, PhD, MBA, offer optimism that the industry’s embrace of new approaches and the FDA’s support of this work means that patients stand to see progress in the coming years.

“We were honored to be invited to submit this paper, and we’re encouraged by the trend toward more innovative clinical trial designs to improve the drug development process and ultimately lead to better patient outcomes,” said Dr. Matrisian.

PanCAN strongly recommends clinical trials at diagnosis and at every treatment decision. In bringing new approaches to clinical research, the goal is to better serve patients by allowing for a “faster, more efficient and cost-effective approach to testing investigational therapies.” The authors cite the innovations below as accelerating progress.

Precision Medicine

Precision medicine is treatment based on a patient’s biology. Biomarker testing of tumor tissue reveals genes and proteins within the tumor that may help to identify effective therapies, and genetic testing for inherited mutations can also help inform a patient’s treatment options. The paper references several efforts around the world – including PanCAN’s Know Your Tumor® service – that are focused on collecting large numbers of pancreatic cancer tissue samples with a goal to identify biomarkers to predict which treatments may work best for a particular cancer’s genetic profile. Some clinical trials taking a precision medicine approach focus on one disease type, like pancreatic cancer, and compare the effectiveness of several investigational treatments based on different biologically defined subtypes. Others test an investigational therapy on patients with tumors in different parts of their bodies that have the same biological features. These efforts look at how a range of treatments, including chemotherapy, targeted therapies and other investigational drugs, work on cancers with different biomarkers.


The paper highlights two platforms that investigate immunotherapies, or treatments that harness the power of a person’s immune system, to fight pancreatic cancer. The “platform” feature of these trials allows multiple immunotherapeutic approaches to be tested and compared to one or several standard-of-care options. Investigational treatments can be added or dropped from the trial over time, depending on preclinical and clinical evidence. Whereas only a small subset of patients with pancreatic cancer currently benefits from immunotherapy, these trials aim to broaden the effectiveness of this approach in more patients.

Adaptive Platform Trials

Several clinical trials using an adaptive design promise to bring new treatments to market more quickly than the traditional clinical trial. These trials, including PanCAN’s Precision PromiseSM, study multiple investigational treatments in parallel. Through careful statistical monitoring, the trial “adapts” over time to allow more patients to be assigned to investigational treatments that are showing more promise during the course of the study. They can also drop treatments shown to be ineffective, resulting in both time and cost savings. Several platform trials focused on pancreatic cancer build on successes seen for other hard-to-treat cancers, including glioblastoma.

Taken together, these clinical trial innovations bring “considerable optimism” that advances are on the horizon, said Dr. Rosenzweig.

“We owe it to patients to make the process as efficient and innovative as possible, to bring new and better treatment options to patients and their loved ones faster,” she said.

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