Over the last decade, cell therapy has emerged as a cutting-edge treatment approach that is transforming cancer care. Built for each individual, it harnesses the power of a person’s living cells, which are collected, reengineered, and returned to the body to fight disease with a level of precision unmatched by traditional drugs or radiation. Researchers at MCW are already pushing cell therapy into new frontiers, exploring its potential in neurological disorders, genetic diseases, and other serious conditions.
Driving this innovation is the MCW Cancer Center’s Cell Therapy Shared Resource (CTSR), a lab that manufactures cell therapies on-site and equips investigators with tools and expertise to turn discoveries into patient-ready treatments. In the lab, teams engineer each patient’s cells, test them for safety and effectiveness, and deliver them in as little as eight days, compared with the two to three weeks typical at other centers. They also support clinical trial design and track how therapies perform in real time. By bringing together advanced technology and scientific collaboration, the CTSR is helping to develop treatments as unique as the patients who receive them.
“Our cell therapy lab is like a pharmacy, but it’s a personalized pharmacy. Every therapy is made for just one patient, each designed to be as safe, precise, and effective as possible,” said Peiman Hematti, MD, CTSR Director.
The World-Class Lab Building Better Therapies
The most visible impact of the CTSR has been the acceleration of chimeric antigen receptor (CAR) T-cell therapy, which reengineers a patient’s immune cells to recognize and destroy cancer. This approach has given patients with aggressive blood cancers—leukemias, lymphomas, and myelomas—the chance at remission, and in some cases, cures.
By producing the specialized cell products that make these therapies possible, the CTSR has powered numerous CAR-T therapy trials over the past decade and helped position MCW at the forefront of one of the most dynamic areas in cancer treatment.
One of the latest milestones comes from an investigator-initiated trial led by Nirav Shah, MD, CTSR Medical Director, with results recently published in the Journal of Clinical Oncology. The study tested a new dual-targeted CAR-T therapy for mantle cell lymphoma, designed to program T cells to recognize both CD19 and CD20—two markers found on cancer cells. Using this approach, the team achieved a 100% overall response rate and an 88% complete remission rate, with manageable side effects. The therapy was manufactured on-site in the CTSR, reflecting years of collaborative work at MCW and laying the groundwork for potential future FDA approval.
“Dual targeting with CAR-T cells in relapsed, refractory mantle cell lymphoma led to unprecedented clinical response, demonstrating how next generation therapies may move the needle in difficult to treat malignancies,” said Dr. Shah.
The CTSR has supported several other landmark advances that deliver innovative care locally and influence the global standard of care, including:
- The international CARTITUDE-4 study, led locally by Binod Dhakal, MD, showing that cilta-cel, a novel CAR T therapy, reduced the risk of multiple myeloma progression or death by nearly 75%.
- A phase 2 melanoma trial, led locally by Amy Harker-Murray, MD, resulting in the FDA approval of lifileucel, the first cell therapy for a solid tumor. Unlike CAR T therapy, this approach used tumor-infiltrating lymphocytes, immune cells taken from a patient’s tumor, multiplied in the lab, and reinfused to fight cancer.
- An early-phase trial testing CAR T therapy in breast and lung cancers, led locally by Lubna Chaudhary, MD, which is opening the door to new options in solid tumors that have long resisted treatment.
From the Brain to the Bloodstream: Cell Therapy Beyond Cancer
Cell therapy is no longer confined to cancer. Now, the CTSR is driving new trials that use living cells to calm seizures, restore movement, and improve genetic disorders.
In a national epilepsy study, led locally by Sean Lew, MD, Professor of Neurosurgery, investigators are testing NRTX-1001, which places lab-grown brain cells into the part of the brain where seizures start. These cells release GABA, a natural chemical that quiets overactive circuits, offering a non-destructive alternative to brain surgery. Froedtert & MCW is the only site in Wisconsin, one of just four in the Midwest. “Currently the most effective options involve removing or ablating brain tissue. This approach could stop seizures while preserving memory and function,” said Dr. Lew.
For Parkinson’s disease, Kunal Gupta, MD, PhD, Assistant Professor of Neurosurgery, is helping launch a trial of ANPD001. The therapy uses a patient’s own skin cells, reprogrammed into dopamine-producing neurons, and transplants them into the brain to restore the signals lost in Parkinson’s. Froedtert & MCW is expected to be the second of just five sites nationwide to offer this trial. “If this trial is successful, it would represent a new class of treatments,” said Dr. Gupta.
And in hemophilia A, decades of research by David Wilcox, PhD, Professor of Pediatric Hematology and Oncology, led to a new gene-modified cell therapy now being tested clinically by Mary Eapen, MRCPI, MS, MBBS, Professor of Hematology and Oncology. Investigators take a patient’s blood stem cells, engineer them to make the missing clotting protein, and return them to the body. Early results, published in the New England Journal of Medicine, showed a patient who once had about ten bleeding episodes per year experienced none after treatment.
From delivering cancer cures to correcting clotting, the CTSR has transformed from a modest operation into a lab recognized internationally for innovation. Its next chapter will unfold in the new Center for Cancer Discovery, where expanded space, tools, and collaborations will accelerate the next wave of breakthroughs.
“Our new lab in the CCD will feature specialized rooms and state-of-the-art equipment, enabling us to develop new types of cell therapies beyond CAR T. Just as importantly, it will bring us closer to our basic science colleagues, helping translate their discoveries into clinical care,” said Dr. Hematti.
Learn about the Cell Therapy Shared Resource.