First-in-the-world immunotherapy cancer treatment shows promise
Physicians and researchers from the Medical College of Wisconsin (MCW), Froedtert Hospital, Children's Hospital of Wisconsin, and BloodCenter of Wisconsin have successfully used a new immunology treatment, chimeric antigen receptor (CAR) T-cell therapy, to extend the life of a 52-year-old Wisconsin man.
Bret C., 52, of Appleton, Wisconsin, is the first patient to participate in the clinical trial. He was diagnosed with mantle cell lymphoma, a cancer of the immune system, in 2011. Despite chemotherapy, stem cell transplants, medications and other clinical trials, his cancer kept returning. When presented with the dual-targeted CAR-T cell immunotherapy clinical trial option in late 2017, just weeks after it had been approved by the FDA, Bret recognized he was out of options and agreed to participate. He received the CAR-T cell dose in late October, and just six weeks later, his cancer was in full remission.
The new treatment genetically alters a person's immune system to uniquely personalize to target cancer cells, a significant departure of more routine chemotherapy that attacks both healthy cells and cancer cells.
While CAR-T cell therapy has been under development since 2012, the patient treated at Froedtert is the first patient to participate in the first-ever, dual-targeted CAR-T clinical trial using the CliniMACS Prodigy device. In this trial, two targets or cells – CD19 and CD20 – were altered. The modified cells can identify cancerous cells, attach to the cancer cells, and effectively destroy the cancer cells. A pediatric clinical trial for CAR-T treatment at Children's Hospital of Wisconsin is expected to start later this year.
"Immunology and T-cell treatments show incredible promise for patients with cancer," said Parameswaran Hari, MD, MRCP, MS. Dr. Hari is professor and interim chief of the Division of Hematology and Oncology and director of the Froedtert & MCW adult Blood and Marrow Transplant (BMT) program and specializes in treating individuals with leukemia and lymphoma at the Froedtert & MCW Clinical Cancer Center. "This is a giant leap forward in personalized medicine. No other cancer center in the Midwest offers the combination of resources and this high level of personalized medicine expertise. The encouraging results of the CAR-T cell trial positions us and our partners as a leader in clinical cancer care, and paves the way for more effective and efficient treatment options."
"Bret's results from the CAR-T cell immunotherapy have been incredible," said Nirav Shah, MD, MSHP, principal investigator of the trial, assistant professor of medicine at MCW, division of Hematology and Oncology, specializing in lymphoma and stem cell transplant at Froedtert & MCW Clinical Cancer Center, and a member of the BMT and Cellular Therapy team. "We are harnessing this knowledge from years of research and creating improved outcomes for patients. There is amazing potential here for the future of cancer treatment, and a healthier world is closer than ever."
The immunotherapy clinical trial continues as the research team tracks the progress of the second participant, who received a dose of CAR-T cells in December 2017. The third patient will begin treatment in February, with one new participant being dosed every six weeks.
Children's Hospital of Wisconsin, Froedtert and MCW recognized the potential of this treatment in 2014 and began acquiring necessary equipment to prepare for a clinical trial. The CliniMACS Prodigy device enables the cancer team to conduct the CAR-T cell immunotherapy through a contained, cell-filtering desktop system that collects the patient's own T-cells and augments them with cancer-fighting genes, producing new cells ready to be infused back into the bloodstream within 14 days. With the CliniMACS Prodigy system, the entire process is performed in a laboratory on the Froedtert & MCW campus, saving precious time and money.
This critical equipment was made possible through philanthropic dollars raised by the Children's Hospital of Wisconsin Foundation together with the Midwest Athletes Against Childhood Cancer Fund.
"This clinical trial demonstrates the strength of our three organizations," said Dr. David Margolis, MD, Children's Hospital of Wisconsin, bone marrow and transplant program director. "The expertise of Medical College of Wisconsin researchers, state-of-the-art facilities of Children's and Froedtert and the generosity of this community, make this clinical trial possible. We look forward to starting a pediatric clinical trial for CAR-T treatment soon."
The successful outcome of this clinical trial is the result of years of collaborative cancer and cellular immunotherapy research at the BMT program. Pioneers in the field of immunotherapy, these researchers helped discover and develop how the body's own immune system has the power to fight cancer cells, leading to innovative ideas of alternatives to chemotherapy, radiation and transplants – cancer treatments that are often effective in killing cancer but also in destroying the body's healthy cells. This knowledge paved the way for the CAR T-cell treatment, which trains the patient's own immune cells to kill the cancer, rather than relying on foreign, toxic substances.
"BloodCenter of Wisconsin has long been a partner in the treatment of blood cancers in collaboration with Froedtert & the Medical College of Wisconsin and Children's Hospital,” said BloodCenter of Wisconsin Executive Vice President of Research, Gilbert C. White, MD. "The basic research being performed at BloodCenter provides an important infrastructure for the development of this clinical trial, and we are thrilled to have contributed to what is a groundbreaking outcome for the patient."
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