The first patients have been dosed in a trial of CAR T therapy in relapsed/refractory leukemia or lymphoma: © nobeatsoffierce – stock.adobe.com
The first patients have been dosed in a phase 2 clinical trial evaluating a new off-the-shelf CAR-T cell therapy for the treatment of pediatric and adult patients with relapsed or refractory (R/R) T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma (T-ALL/LBL).
WU-CART-007, according to a news release from biotechnology company Wugen, Inc., is a potential first-in-class, investigational, anti-CD7 CAR-T cell therapy.
Glossary
Composite complete remission rate: patients who achieved either standard complete remission or a lesser form of complete remission.
Minimal residual disease: small amounts of cancer cells left in the body after treatment.
Regenerative Medicine Advanced Therapy (RMAT) designation: an FDA designation granted to a drug that is a regenerative medicine therapy intended to treat, modify, reverse or sure a serious or life-threatening disease or condition where preliminary clinical evidence suggests it has the potential to address unmet medical needs.
Fast Track designation: an FDA process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
Orphan Drug designation: an FDA designation granted to a drug to prevent, diagnose or treat a rare disease or condition, qualifying sponsors for incentives such as tax credits for clinical trials, exemption from fees and potential seven years’ market exclusivity.
Rare Pediatric Disease designation: part of an FDA program intended to incentivize drug development for rare pediatric diseases.
Overall response rate: patients who responded partially or completely to treatment.
The trial is currently recruiting participants and will have an estimated enrollment of approximately 125 patients, according to its listing on clinicaltrials.gov. The trial has eight locations: City of Hope in Duarte, California, Children’s Hospital Los Angeles, Moffit Cancer Center in Tampa, Florida, Washington University Saint Louis in Missouri, Children’s Hospital of Philadelphia and The University of Texas MD Anderson Cancer Center in Houston, as well as two locations in Victoria, Australia.
The trial, according to the listing, aims to evaluate the composite complete remission rate of WU-CART-007 in patients with R/R T-ALL/LBL and the treatment’s effectiveness in inducing complete minimal residual disease negative response.
The trial has an estimated primary completion date of Dec. 30, 2026, and a study completion date of Dec. 30, 2028, according to the listing.
“It’s been 20 years since a new medicine was approved for patients with relapsed or refractory T-ALL/LBL, which remain challenging hematologic malignancies with limited treatment options in the relapsed or refractory setting,” said Dr. Cherry Thomas, chief medical officer of Wugen, in the news release. “WU-CART-007 has shown clinical response and manageable safety, making it a promising off-the-shelf cell therapy candidate to fill a longstanding treatment gap. The enthusiasm around the program and the need for new treatments have been reflected in the study recruitment thus far, as it is enrolling faster than anticipated.”
More on WU-CART-007
CAR-T cell therapy is typically a type of treatment that, as defined by the National Cancer Institute, involves a patient’s T cells — part of their immune system — being extracted and changed in a laboratory so that they will now attack cancer cells, and then are given back to the patient.
However, WU-CART-007 is made using healthy donor-derived T cells, according to the news release from Wugen, in order to eliminate the risk of malignant cell contamination.
WU-CART-007, according to the news release, has already received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA).
The company previously announced results from a phase 1/2 cohort expansion study that, according to the news release, showed clinically manageable safety and evidence of anti-leukemic activity, with an overall response rate of 91% and a composite complete remission rate of 73% in heavily pretreated patients with R/R T-ALL/LBL.
“The data we have previously reported from our Phase 1/2 study on the WU-CART-007 program has paved the way for the initiation of this pivotal study, and suggests it has the potential to set a new standard of care for relapsed or refractory T-ALL/LBL,” said Kumar Srinivasan, president and CEO of Wugen, in the news release. “The program has earned multiple U.S. Food and Drug Administration accelerated approval pathway designations, including RMAT, Fast Track, Orphan Drug and Rare Pediatric Disease, as well as Priority Medicines designation in the EU. With this pivotal study now underway, we are advancing toward a much-needed off-the-shelf CAR-T option for patients who face historically poor outcomes and limited treatment alternatives.”
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