CAR-T cell Immunotherapy shows Promise for⁢ Aggressive‍ Blood Cancers

Updated June 01, 2025
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An international⁣ clinical⁣ trial, spearheaded by Washington University School of Medicine researchers in St. Louis,indicates that⁢ a novel immunotherapy is effective against aggressive blood cancers⁢ while exhibiting manageable side effects. ⁣the study focused on a ⁤CAR-T ‍cell ‍immunotherapy designed to target ⁢cancerous T cells.

The trial included patients diagnosed with rare cancers, specifically T cell acute lymphoblastic leukemia or⁤ T cell lymphoblastic lymphoma, who had exhausted ⁣standard treatment options. Impressively, the majority of patients receiving ⁢the full dose⁣ of this ⁣new immunotherapy achieved complete remission.

John F. DiPersio, MD, PhD, the Virginia E. &⁤ Sam J. Golman Professor‍ of Medicine at WashU Medicine, who developed the therapy, noted its potential as a “transformative advance” for patients with these aggressive cancers who lack alternative treatments. He added that the CAR-T cell treatment could serve as a “bridge-to-transplant” therapy,enabling stem cell transplantation for patients otherwise ineligible.

The trial involved 28 adult and adolescent patients whose cancers either returned‍ after multiple therapies or never responded to initial ⁢treatment. These cancers affect approximately⁤ 1,000 people annually in the⁤ U.S.Patients whose cancers are unresponsive⁢ or relapse typically survive only six months, with less than 7% living beyond five years.

The therapy, WU-CART-007, was developed by Wugen, a biotech startup founded by DiPersio and Matthew Cooper, PhD. The clinical trial took place in australia, Europe, and ⁢across multiple U.S. sites, including Siteman Cancer Center ‍at Barnes-Jewish Hospital and WashU Medicine in⁢ St. Louis.

The trial’s dose-escalation phase ⁣helped determine‍ the optimal dose of 900 million CAR-T cells. Prior to receiving the cells, patients underwent lymphodepletion to reduce existing immune‍ cells. Of the 11⁢ evaluable patients,91% responded to the treatment,with 72.7% achieving complete remission. Six patients who underwent subsequent transplants remained disease-free six to 12 months later.

“These response and remission rates — ranging from 70%-90% of‍ patients –⁢ are much ⁤higher than we would expect ⁣from standard-of-care for this cancer type, which typically leads to remission in only 20%-40% of patients,” said Armin Ghobadi, MD, a professor of medicine at WashU Medicine.

Ghobadi emphasized the remarkable responses, given the⁣ patients’ lack of other options and the aggressive nature of their relapsed cancers.

Most patients experienced cytokine release syndrome, predominantly mild or moderate. This common side effect of CAR-T cell therapy results from the release of inflammatory chemicals by immune cells. A smaller percentage experienced more severe cytokine release‍ syndrome, and rare side effects like neurotoxicity and graft-versus-host disease were also observed and managed.

This “universal” CAR-T⁢ cell‍ therapy utilizes CRISPR gene editing to create cells from⁢ any healthy donor, making ‍it readily available “off-the-shelf.” This contrasts with existing CAR-T cell therapies that require weeks to adapt a patient’s own immune cells.

The CRISPR editing process removes the T cell receptor to minimize graft-versus-host disease and prevents the⁢ CAR-T cells ⁣from attacking⁢ each other. The cells ⁣are ⁢engineered to target ‍the CD7 protein on cancerous T cells, destroying⁢ the cancer.

What’s next

A larger ⁤international clinical trial is underway to ⁤further evaluate this universal CAR-T cell therapy⁢ as a⁣ potential approved treatment for deadly T cell cancers, according to DiPersio.