An off-the-shelf CAR-T cell therapy developed by researchers at WashU Medicine has been granted FDA Breakthrough Therapy designation for relapsed or refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma–two rare, fast-moving blood cancers with few effective treatment options. Licensed to Wugen, the therapy, WU-CART-007 (soficabtagene geleucel), is designed to buy patients enough disease control to reach stem cell transplantation, currently the only potentially curative option.
The designation follows early clinical data showing strong activity. In the phase 1 study, 10 of 11 evaluable patients responded, and eight achieved 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 John F. DiPersio of WashU Medicine, in a press release. A key advantage is speed: unlike personalized CAR-T products, WU-CART-007 is made in advance from healthy donor cells, avoiding the weeks-long manufacturing delay that can be fatal for patients with these diseases.
WashU Medicine says the therapy also addresses a problem unique to T-cell cancers: preventing the engineered T cells from attacking one another. “This FDA Breakthrough Therapy designation for soficabtagene geleucel highlights the role of Siteman Cancer Center, a leading NCI-designated Comprehensive Cancer Center, and WashU Medicine in advancing innovative CAR-T cell therapies for aggressive T-cell leukemias and lymphomas,” said Timothy J. Eberlein. A phase 2 trial is ongoing.
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