Novel CAR-T Therapy Gets Breakthrough Status

A novel “off-the-shelf” CAR-T cell therapy developed by researchers at Washington University School of Medicine in St. Louis has received an FDA breakthrough therapy designation. The therapy, known as WU-CART-007 (soficabtagene geleucel), is designed to treat T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma, two difficult-to-treat malignancies that often relapse or fail to respond to existing therapies, the medical center says. It is being developed by Wugen, a spinout from WashU Medicine.

FDA’s action was based on preliminary clinical data suggesting the therapy can effectively target and eliminate cancerous T cells with manageable safety. In a Phase 1 study involving 28 heavily pretreated adolescent and adult patients, 11 were evaluable for response. Among those, the therapy achieved a 91% overall response rate, with 10 patients showing either complete remission or substantial reductions in disease burden, according to WashU. Eight patients (72.7%) achieved complete remission.

Notably, several patients who responded were able to proceed to stem cell transplantation — the only potentially curative option for these cancers — and remained disease-free for six to 12 months at the time of analysis.

“These are patients who typically have very limited options and poor outcomes,” said John F. DiPersio, a WashU physician-scientist who led development of the therapy. He emphasized the potential for the treatment to serve as a bridge to transplant, which many patients cannot otherwise access due to lack of remission.

T-cell leukemias and lymphomas are rare but highly aggressive. About 1,000 cases are diagnosed annually in the U.S., and patients with relapsed or refractory disease face a median survival of roughly six months, with long-term survival rates below 10%, the researchers say.

Unlike approved CAR-T therapies, which target B-cell cancers, WU-CART-007 is specifically engineered for T-cell malignancies, a setting that presents unique biological challenges, WashU says. Researchers had to overcome “fratricide,” where engineered T cells attack each other instead of the cancer, a hurdle that has historically limited development in this space.

A key differentiator is the therapy’s allogeneic, or “off-the-shelf,” design. Instead of using a patient’s own cells — a process that can take weeks — WU-CART-007 is manufactured from healthy donor cells and can be administered more rapidly. That speed could be clinically meaningful for patients with fast-progressing disease, who may not survive long enough to receive autologous CAR-T therapies, WashU says.

The therapy is currently being evaluated in an ongoing Phase 2 trial across multiple global sites.