FDA Grants Breakthrough Status to Immunotherapy Combination for Pancreatic Cancer

The gist: A new pancreatic cancer treatment combines two drugs, known as CRS-207 and GVAX Pancreas. This combo treatment boosts a patient’s own immune system to fight cancer. It has been tested in volunteer patients in clinical trials, and has shown promising results for people with metastatic pancreatic cancer. The U.S. Food and Drug Administration (FDA) has now granted breakthrough therapy designation for CRS-207 plus GVAX Pancreas, meaning that review and approval will be accelerated so that the drug can more quickly reach patients in the U.S., outside of clinical trials.

“The FDA today granted breakthrough therapy designation to the combination of two immunotherapies — CRS-207 and GVAX Pancreas — for the treatment of metastatic pancreatic cancer, according to Aduro BioTech, the combination treatment’s manufacturer.

“The combination of GVAX, an irradiated, granulocyte-macrophage colony–stimulating factor vaccine, and CRS-207, an immunotherapy vaccine containing live-attenuated Listeria monocytogenes bacteria, induces a potent innate and T-cell–mediated immune response.

“ ‘We are extremely pleased to receive Breakthrough Therapy Designation and the high degree of FDA collaboration toward advancement of our program that it confers,’ Stephen T. Isaacs, chairman, president and CEO of Aduro, said in a press release. ‘This designation underscores the potential of our combination immunotherapy approach to make a difference in the lives of patients with pancreatic cancer, which remains a very difficult cancer to treat. We are encouraged by our phase 2 results and look forward to completing enrollment in our phase 2b ECLIPSE trial by end of 2015.’ “

Immunotherapy Duo Improves Survival in Metastatic Pancreatic Cancer

Overall survival was improved in metastatic pancreatic cancer patients through an innovative immunotherapy strategy in a multicenter study reported at the 2014 Gastrointestinal Cancers Symposium.

“ ‘This is the first time a randomized study has shown that immunotherapy is effective in pancreatic cancer,’ said Dung T. Le, MD, Assistant Professor of Medicine at the Johns Hopkins University Sidney Kimmel Comprehensive Cancer Center, Baltimore. ‘This is just a first step, and we believe we’ll be able to take this approach further.’ ”

“The novel treatment, which may be better tolerated than standard chemotherapy, involves two different anticancer vaccines: GVAX Pancreas followed by CRS-207. GVAX is composed of pancreatic cancer cells that have been genetically modified to secrete granulocyte-macrophage colony-stimulating factor (GM-CSF), which stimulates the immune system. GVAX is given with low-dose cyclophosphamide to inhibit regulatory T cells and boost the vaccine’s efficacy. The second vaccine, CRS-207, is live-attenuated Listeria monocytogenes (Lm), which has been genetically modified to be safe for human use while retaining its ability to stimulate an immune response against the protein mesothelin on pancreatic tumor cells.

“The combination essentially trains the body to recognize and attack pancreatic tumors. In mouse tumor models, Lm/GVAX vaccines are synergistic, and in a phase I study of CRS-207, patients with pancreatic ductal adenocarcinoma who had received prior GVAX lived more than 15 months, Dr. Le explained.”

Editor’s note: Immunotherapy treatments work by boosting a patient’s own immune system to fight cancer. This story describes such a treatment, which combines two “cancer vaccines” called GVAX Pancreas and CRS-207. Scientists found promising results for the treatment in pancreatic cancer patients.