FDA Breakthrough Therapy Designation Reflects Practice-Changing Trends Only at Siteman

Recognition follows strong early clinical results for a novel off-the-shelf CAR T therapy targeting rare and aggressive T-cell malignancies

A novel off-the-shelf CAR T-cell therapy pioneered at Siteman Cancer Center at Barnes-Jewish Hospital and WashU Medicine is gaining national recognition after delivering striking clinical results in patients with rare and aggressive blood cancers.

The FDA has granted Breakthrough Therapy Designation to WU-CART-007, an allogeneic anti-CD7 CAR T-cell therapy developed by WashU Medicine researchers. In early global trials, 73% of adults and adolescents with relapsed or refractory (R/R) T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma (T-ALL/LBL) achieved full remission following treatment — an outcome that positions the therapy as a potential gamechanger in T-cell malignancies.

“Relapsed T-cell leukemias and lymphomas represent one of the most challenging areas in hematologic oncology,” said oncologist John DiPersio, MD, PhD, director of the Center for Gene and Cellular Immunotherapy at WashU Medicine and an internationally recognized cell therapy leader at Siteman Cancer Center. “We are leading transformative advances for patients with these rare and aggressive cancers. Developing an off-the-shelf CAR T platform that can induce high remission rates in this population reflects the translational depth and cellular therapy infrastructure we’ve built at Siteman.”

DiPersio and Matthew Cooper, PhD, developed the therapy — manufactured using an off-the-shelf approach from healthy donors — to target CD7+ malignancies. The two founded the biotech company Wugen in 2018 to advance the research. Further clinical trials are underway in the U.S., Europe, Asia and Australia, including at Siteman Cancer Center and Siteman Kids at St. Louis Children’s Hospital.

In reviewing results from earlier clinical trials in children, researchers at Siteman Kids noted that WU-CART-007 (also known as soficabtagene geleucel, or sofi-cel) could be a gamechanger if the therapy continues to move almost all patients from disease-state to remission, thus enabling patients to undergo stem cell transplantation.

Learn more about:

• WU-CART-007 — S. FDA Grants to Wugen’s WU-CART-007 Breakthrough Therapy Designation
• Other recent advances at Siteman Kids

‘An Eco-System of Innovation and Excellence’

The rapid advance of WU-CART-007 is just the latest example of what Timothy J. Eberlein, MD, director of Siteman Cancer Center, says arises out of a robust eco-system of innovation, collaboration and excellence in cancer research at WashU Medicine.

Siteman is known internationally for its basic and translational research efforts and is one of only a few institutions to receive three prestigious Specialized Program of Research Excellence (SPORE) grants from the National Cancer Institute (NCI), for leukemia, endometrial and pancreatic cancer research. Blood Cancer United, formerly known as the Leukemia & Lymphoma Society, also has awarded scientists at Siteman a Specialized Center of Research (SCOR) grant for lymphoma research. Such grants are specifically designed to accelerate promising translational research into patient care.

“We are committed to continually advancing treatments for cancer and broadening options for patients,” Eberlein said. “Toward that goal, we have initiated several home-grown clinical trials that have changed the course of treatment for many cancers. Our depth and breadth of oncology research is wide, and our expertise is the result of innovation, dedication and multidisciplinary cross-collaboration that occurs throughout our center.”

Other examples of comprehensive, specialized programs at Siteman include the:

• Sarcoma program, one of the largest in the Midwest
• Blood Cancer Center, internationally recognized for research and clinical expertise
• Gynecologic Oncology program, the largest in the Midwest, with 10,000+ annual outpatient visits
• Brain Tumor Center, known nationally and internationally for innovative care and neuro-oncology research, including multiple investigator-initiated clinical trials

Translational Research Highlights

In addition to the latest breakthrough in leukemia and lymphoma research and care, examples of other research efforts that have changed practice guidelines include:

• New Standard of Care Established for Locally Advanced Head and Neck Cancers — In the first change in standard-of-care therapy in more than 20 years, the FDA approved the use of the immunotherapy drug pembrolizumab (Keytruda) for treatment of resectable locally advanced head and neck squamous cell carcinoma (HNSCC) in adults. The approval, announced in mid-2025, came after a clinical trial initiated at Siteman in 2013. That trial and later ones, including an international trial, demonstrated greater tumor shrinkage prior to surgery and longer survival rates when immunotherapy was added. “It’s exciting to see our ideas move toward clinical practice with such impressive and potentially life-changing results,” said Douglas Adkins, MD, co-director of the Head and Neck Tumor Center at Siteman, who co-led the clinical trials at Siteman and elsewhere.
• Addition of Brentuximab Vedotin for Relapsed Diffuse Large B-Cell Lymphoma Results in Statistically Significant Survival Benefit — With approximately 40% of patients diagnosed with diffuse large B-cell lymphoma (DLBCL) having relapsed or refractory disease, researchers at Siteman, led by Nancy Bartlett, MD, found in the ECHELON-Phase Three clinical trial that the use of an antibody-drug conjugate brentuximab vedotin, when combined with either lenalidomide or rituximab, was not only safe but also demonstrated improved survival benefit in patients with R/R DLBCL.
• Dostarlimab Plus Chemo for Primary Advanced or Recurrent Endometrial Cancer — Matthew Powell, MD, co-led national studies that found adding immune checkpoint inhibitors to standard therapy for endometrial cancer improves outcomes for many patients, with an average increase in overall survival of 31%.
• T-cell Immunotherapy Effective in Treating Rare Soft Tissue Cancers — Siteman Cancer Center’s Sarcoma program was a major clinical trial site for this study, which found that T-cell immunotherapy, specifically the drug afamitresgene autoleucel, or afami-cel, was effective and generated long-term responses in patients with rare soft tissue cancers.
• Sotorasib Approved as Targeted Therapy for Patients with Specific Type of Non-Small-Cell Lung Cancer — Following clinical trials at Siteman and globally, the FDA approved sotorasib for patients with non-small-cell lung cancer whose tumors express a G12C mutation in the KRAS gene and who have already undergone previous treatment. Ramaswamy Govindan, MD, who led the study, noted that the drug targeted the most common mutation, reduced tumor sizes, and improved overall survival rates.
• Medicare Approves Whole-Genome Test for Blood Cancers — A test for acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) developed at Siteman was the first whole-genome sequencing test for cancer to be approved for reimbursement by the Centers for Medicare & Medicaid Services. Called ChromoSeq, the test is now routinely used by oncologists to guide treatment decisions for patients with blood cancers.

“At Siteman, we have built one of the world’s leading cellular immunotherapy programs focused on developing next-generation treatments for rare and refractory cancers,” Eberlein said. “Many of our most impactful cell therapy trials are investigator-initiated and originated here, reflecting a translational infrastructure designed to bring innovative therapies to patients with the most aggressive and rarest cancers.”