Using Real World Evidence to Show Initial Results of a Drug Combination in Pancreatic Cancer Patients

In collaboration with xCures and the Huntsman Cancer Institute at the University of Utah, Cancer Commons presented initial findings from our real-world data registry on a novel treatment combination for pancreatic cancer patients at a conference in San Diego, CA, today. The “Special Conference on Advancing Precision Advancing Precision Medicine Drug Development: Incorporation of Real-World Data and Other Novel Strategies” is hosted by the American Association for Cancer Research (AACR).

XCELSIOR is an IRB-approved, patient-centric, real-world data and outcomes registry established to develop operational and analytic methods in precision oncology. When patients participate in XCELSIOR, they share access to their medical records and receive personalized support—including help with access to therapy and information about the latest treatment options—through rigorous research and virtual tumor boards. Their information is captured and used to identify the best options for current and future patients.

The findings presented today focused on advanced pancreatic cancer patients who enrolled in XCELSIOR and were treated with a MEK inhibitor, trametinib, in combination with an autophagy inhibitor, hydroxychloroquine, a generic medication approved by the U.S. Food and Drug Administration (FDA) for treatment of malaria, as part of their clinical cancer care. An additional cohort of patients was treated with the same combination at the Huntsman Cancer Institute at the University of Utah, and we presented results from both cohorts.

In total, 12 heavily pre-treated metastatic pancreatic cancer patients who had exhausted standard-of-care options and received treatment with the combination of trametinib plus hydroxychloroquine were identified. Eight of these patients received the treatment for more than 30 days. The progression-free survival time for patients who received treatment for more than 30 days was 2.9 months, and the overall survival time was 7.4 months (since start of treatment).

Of seven evaluable patients, one patient had a partial response, three patients had stable disease (for at least 8 weeks), and three progressed while on the treatment.

“Though the study includes a small number of patients, these results are encouraging, and this regimen could potentially be an option for this group of patients who don’t have many more options,” said Conan Kinsey, MD, PhD, the senior author and presenter of the study.

With these findings, Cancer Commons and our collaborators demonstrate the feasibility of utilizing a real-world data registry to rapidly report on the safety profile and clinical benefits of novel cancer drug combinations in patients who are receiving therapies outside of a standard clinical trial. Using this approach created speed and cost efficiencies relative to standard multi-center oncology clinical trials.

XCELSIOR is still open for enrollment. If you and your physician are contemplating treatment with this combination therapy, or need help navigating your treatment options, please consider being part of our observational study. Click here to register.

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Image Credit: Lola Rahib, PhD (left), and Conan Kinsey, MD, PhD (right), present our findings in San Diego, CA