Early results show promise of CAR-T cell therapy for treating rare blood cancer

Armin Ghobadi, MD, prepares the new “universal” CAR-T cell product for a patient’s infusion as part of a clinical trial of the newly engineered CAR-T cell therapy called WU-CART-007.

CANCER DIGEST – May 31, 2025 – Eight of 11 patients (72.7%) treated in a early stage international clinical trial of a new drug for a rare form of blood cancer achieved complete remission, meaning there were no signs of cancer after treatment, a new study shows.

The trial led by John DiPersio, MD, PhD and other researchers at Washington University in St. Louis involved 28 adults and adolescents with T cell acute lymphoblastic leukemia that had not responded to several other therapies. The results of the trial were published in the May 30, 2025 journal Blood.

The rare cancer affecting T cells is particularly difficult to treat as T cells are the immune system’s major players in eliminating other types of cancer, only about 1,000 people are diagnosed with it annually in the U.S. If patients do not respond to initial conventional therapy, or the cancer returns, patients survive only six months. The average 5-year survival rate is 7 percent.

The new therapy is a version of CAR-T cell treatment, called WU-CART-007, developed by Wugen, a Washington University technology transfer startup founded by DiPersio and other Washington U investigators. It is hoped the treatment can provide a "bridge" to stem cell transplantation for these patients, which is the only potential cure for these blood cancers.

The newly engineered CAR-T cell therapy is considered a “universal” CAR-T cell therapy because unlike current CAR-T cell therapies that depend on genetic engineering of the patient's own T cells, WU-CART-007 can be produced from cells donated by any healthy individual and used to treat any patient with a T cell cancer.

In addition to Washington University, the clinical trial was conducted at cancer centers in Australia, and several sites in Europe and the U.S. In all 28 patients were enrolled in what is called a dose escalation trial, meaning patients are given increasing amounts of the new treatment to find the optimal dose.

Once the optimal dose was determined 13 patients received the full dose of 900 million CAR-T cells following the depletion of their immune cells to make room for the newly engineered therapeutic T cells. Two of the 13 died from the cancer or treatment complications such as infections during the study period.

Of the 11 who completed the treatment 10 had their cancer disappear or significantly reduced for an overall response rate was 91 percent. Of these patients 8 showed no evidence of cancer six to 12 months after treatment for a complete response rate of nearly 73 percent.

“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,” the study’s first author Armin Ghobadi, MD, said in a press release. "These responses are remarkable because the patients in this trial had run out of options. They had very aggressive cancers return after several lines of therapy, including several who relapsed after an earlier stem cell transplant.”

Most patients (88.5%) experienced a side effect called cytokine release syndrome, which is essentially full-body inflammation. Other side effects included nerve toxicity causing headaches, dizziness or seizures, and mild graft-vs-host disease.

The researchers concluded the study results stating the results need to be confirmed in larger clinical trials. A larger international clinical trial is already underway.

Source: Washington University Medicine press release