An innovative cell-based immunotherapy developed by WashU Medicine researchers is changing the paradigm of blood cancer care. The pioneering therapy — known as WU-CART-007 — has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration, expediting its path to saving lives at the bedside.

Licensed to Allotera Therapeutics (formerly Wugen), a WashU Medicine biotech startup based in St. Louis’s Cortex Innovation District, the therapy targets T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LL). These highly aggressive blood cancers traditionally leave patients with few treatment options and a devastating five-year survival rate of under 7%.

While conventional CAR-T therapies require a grueling three-to-four-week manufacturing process using a patient’s own immune cells, WU-CART-007 is pre-manufactured from healthy donor cells. This means it’s ready for immediate use right off the shelf, dramatically reducing critical logistical and financial barriers to deliver life-saving treatment for patients who can’t wait another minute.

Early-phase clinical studies showed that the therapy can selectively target and eliminate cancerous T cells with manageable side effects. In fact, the therapy achieved a 91% overall response rate, with a staggering 72.7% of patients reaching complete remission.

“This therapy has the potential to enable long-term survival for this patient population by controlling the disease and allowing patients — who would otherwise not be eligible — to proceed to stem cell transplantation, the only potentially curative treatment for these blood cancers,” said WashU Medicine oncologist John F. DiPersio, MD, PhD, the Virginia E. & Sam J. Golman Professor of Medicine and director of WashU Medicine’s Center for Gene and Cellular Immunotherapy, who first developed the therapy in his lab.

As phase 2 clinical trials progress, WashU Medicine and Allotera Therapeutics are redefining what’s possible in cancer care, translating the most cutting-edge research into promising new treatments to offer patients more options — and more hope.