A new clinical research study at Roswell Park Cancer Institute (RPCI) will assess the safety and efficacy of a novel immunotherapy approach to treating advanced melanoma. Developed at RPCI by Roswell Park researchers, the investigational vaccine shows promise for treating tumors in patients with stage III/stage IV melanoma, and perhaps as a future therapy for other solid-tumor cancers as well.
John M. Kane III, MD, FACS, a surgical oncologist who is Chief of the Melanoma/Sarcoma Service at RPCI, will lead the phase I clinical trial, which will enroll between 12 and 20 patients over the course of approximately three years. Participating patients with advanced melanoma will receive a series of three vaccinations over six weeks.
The vaccine combines a "heat-shock" protein (HSP) with a whole-protein antigen, gp100. Dr. Kane's collaborator in developing the vaccine, John Subjeck, PhD, a researcher in RPCI's Department of Cell Stress Biology, laid the groundwork for the study with his pioneering work in identifying the various functions of HSPs, which normally protect cells by binding to other proteins just long enough to see them through periods of stress caused by heat and other factors. "Although heat-shock proteins act as protective chaperones," Dr. Kane says, "it appears that they are also very powerful stimulators of the immune system, amplifying the effectiveness of our vaccine."
The HSP vaccine, Dr. Kane notes, is recombinant, or created entirely in the laboratory, meaning that no cells from patients are required for its manufacture. It can be made in large quantities and is stable for several years. Importantly, he says, "because we're able to use a whole-protein target, all components of the immune system are stimulated, and the number of melanoma patients who could potentially respond to this vaccine is much greater compared to many other immune-based therapies that target only part of a protein." No significant side effects were reported from earlier preclinical studies of the vaccine.
Dr. Kane received Rapid Access to Intervention Development (RAID) support for the development of this melanoma vaccine from the National Cancer Institute (NCI), a division of the National Institutes of Health. These highly selective awards provide funding for particular projects, but are not restricted to a set dollar value. They are intended to help new and promising developmental therapies advance quickly to the clinical-trial phase.
This research will be funded in part by a generous donation from the Jennifer Linscott Tietgen Family Foundation, which is dedicated to supporting melanoma cancer research in memory of their daughter, Jennifer.
"When we lost Jennifer at a young age to melanoma, it was earth-shattering for our family," the Tietgens say. "We knew we needed to do something to make a difference, and the best way we could think of was funding melanoma research projects. The Roswell Park melanoma team has an advanced understanding of this devastating cancer and is working tirelessly to improve diagnostic testing and treatment."
The study is the sixth launched through RPCI's Center for Immunotherapy since the Center opened in 2010.
Melanoma, the deadliest form of skin cancer, is the fifth-most-common type of new cancer diagnosis in American men and the sixth-most-common in American women. The NCI estimates that 76,690 new cases will be diagnosed and 9,480 people will die from the disease in the U.S. this year.
The study, A Phase I Trial of a Recombinant Human hsp110-gp100 Chaperone Complex Vaccine for Advanced Stage IIIB/C or IV Melanoma, is ClinicalTrials.gov study NCT01744171. For more information, including details on eligibility criteria, go to http://clinicaltrials.nlm.nih.gov/ct2/show/NCT01744171.
Source: Roswell Park Cancer Institute