The Food and Drug Administration (FDA) approved the use of pembrolizumab (anti-PDI) immunotherapy for patients with advanced pancreatic cancers who have a defect in their capacity to repair DNA.
Support from the Lustgarten Foundation was part of the success of the clinical trial that demonstrated the efficacy of pembrolizumab to the FDA. In addition to supporting the research, the Foundation has been encouraging and covering the cost for pancreatic cancer patients to receive mismatch-repair deficiency (MMR) testing in an effort to increase enrollment in the clinical trial.
"It is the first example of 'personalized immunotherapy.' A specific immune treatment can now be recommended for patients based exclusively on the genetic characteristics of their tumor,” Bert Vogelstein, M.D, co-director of the Ludwig Center at the Johns Hopkins Kimmel Cancer Center and a Lustgarten Foundation Distinguished Scholar who helped direct this study, said. “If the tumor shows a repair defect, then it is very likely that it will respond to this drug, regardless of how advanced the cancer is at the time of treatment."
It is estimated that approximately 1 in 50 advanced pancreatic cancer patients have MMR in their tumors that make them candidates for this type of therapy. Pembrolizumab is the first cancer drug based on a predictive cancer marker, rather than tumor type, to be approved by the FDA.
A study conducted at the Johns Hopkins Bloomberg-Kimmel Institute, funded in part by the Lustgarten Foundation, demonstrated that a small group of patients with advanced cancers had a significant response to pembrolizumab immunotherapy. The responsive tumors had a mismatch-repair deficiency, resulting in a large number of genetic mutations that stimulate an immune response. However, this native immune response is too weak to reject the tumor. Treatment with pembrolizumab energized this response, resulting in dramatic tumor shrinkages with minimal side effects relative to traditional chemotherapy.
"This is an incredibly important step forward and we are delighted to have had a key role in its success," said David Tuveson, M.D, Ph.D., Director of Research for the Lustgarten Foundation. "Patients have responded very well to this drug. This is the beginning of personalized medicine for pancreatic cancer patients."
The Lustgarten Foundation encourages all patients to get screened for microsatellite instability.
The Lustgarten Foundation is America's largest private foundation dedicated to funding pancreatic cancer research. Based in Bethpage, N.Y., the Foundation supports research to find a cure for pancreatic cancer, facilitates dialogue within the medical and scientific community, and educates the public about the disease through awareness campaigns and fundraising events.