Medical College of Wisconsin (MCW) cancer investigators recently treated the 100th patient in an ongoing, multi-arm, clinical trial using an in-house manufactured, dual-targeted CAR-T therapy for patients with B-cell malignancies. Built on decades of research in academic medicine and carried out with patients at the Froedtert & MCW Clinical Cancer Center, the innovative and proprietary treatment process enables the team to develop personalized and potentially lifesaving therapies for patients with blood cancers in a matter of days.
“Treating patients on clinical trials allows us to both learn more about how to treat rare and aggressive cancers while also offering patients opportunities to get cutting-edge treatments that may provide hope when standard approaches have not been effective. In this way, we’re making meaningful strides in cancer discoveries at an unprecedented pace,” said Nirav Shah, MD, associate professor of hematology and oncology.
Dr. Shah added that having an onsite manufacturing lab saves critical time for patients who need CAR-T therapy. Patients participating in the in-house trial led by research teams at MCW receive their modified T-cells within eight to 12 days after extraction, rather than the three to four weeks required to transport a patient’s cells to off-site commercial labs for processing. The institution’s CAR T-Cell Lab is the only such onsite facility in Wisconsin.
Dr. Shah and the team strive to eventually gain approval from the U.S. Food and Drug Administration (FDA) for the treatment developed in-house, which would enable MCW to offer the therapy as a standard approach for patients with relapsed blood cancers. “The progress we’ve seen over the last decade is remarkable. Five years ago, patients with relapsed blood cancers had few options; now we’re seeing durable remissions in patients previously felt to be incurable. Some of our early CAR-T patients have been cancer-free for more than five years. We’re just getting the opportunity to share their stories now.”
Two of Shah’s patients recently spoke on The Word on Medicine podcast, which aired Saturday, June 22, on radio stations and streaming platforms. One patient, Nyamwange Keen, was treated for chronic lymphocytic leukemia (CLL) at the end of 2023. He was the 100th patient to be treated via the in-house CAR-T therapy trial, after receiving many previous lines of therapies. Keen shared a very sentimental thank you on the podcast for what he felt was a “transformational” opportunity.
“I was at a dead end when I came here. CAR-T has provided an opportunity of hope and promise for a great future for me. For the first time in all these years, I have no detectable CLL. I asked Dr. Shah, ‘What do I do now?’ And he told me, ‘Go and live your life.’ Even with the growing pains of CAR-T, I feel more optimistic now than ever. I am peaceful, I am encouraged, and I am stronger because I have a new lease on life,” said Keen. 
MCW Contributes to New FDA Approvals for Patients with Blood Cancer
MCW Cancer Center investigators have conducted more than 15 CAR-T clinical trials to identify new and promising treatments for patients with blood cancers such as lymphomas, leukemias, and multiple myeloma. Notably, Dr. Shah was involved in the development of lisocabtagene maraleucel for patients with CLL as a key investigator on the TRANSCEND CLL 004 trial. Results of this study were published in The Lancet, where Dr. Shah served as a co-author, and led to FDA approval of this agent in patients with relapsed, refractory CLL that has failed other lines of therapy.
Additionally, this spring, the FDA approved another B-cell maturation antigen- (BCMA) targeted treatment as a second-line therapy for multiple myeloma, after promising results from the Phase 3 CARTITUDE-4 study were published in The New England Journal of Medicine and subsequently presented by its lead author, MCW’s Binod Dhakal, MD, associate professor of hematology and oncology, at the 2023 American Society of Clinical Oncology Annual Meeting. The landmark study showed that cilta-cel CAR-T therapy reduced the risk of disease progression and death by approximately 74 percent, and effectively made the case that CAR-T therapy could be used in earlier lines of treatments for some patients.
While advancements in CAR T-cell therapy have proven successful for many blood cancers, MCW Cancer Center investigators are hopeful that further research will lead to innovative CAR-T therapies for the treatment of solid tumors. Lubna Chaudhary, MD, associate professor of hematology and oncology, is putting this to the test in the phase I LYL797 clinical trial for patients with triple negative breast cancer and non-small cell lung cancer. “CAR T-cell therapy holds the potential to help millions more patients if it can be successfully expanded into solid cancers,” said Dr. Chaudhary.
Learn how CAR T-cell therapy works from MCW’s Project Wonder.