Off-the-shelf cellular therapy targets relapsed or refractory T-cell leukemia/lymphoma
A potentially groundbreaking clinical trial is now open at Siteman Kids at St. Louis Children’s Hospital to evaluate an allogeneic, off-the-shelf cellular therapy designed to treat patients with relapsed or refractory T-cell acute lymphoblastic leukemia or T-cell lymphoblastic lymphoma (T-ALL/LBL).
Siteman Kids is one of only five pediatric centers in the U.S. — and the only one in the Midwest — to participate in the clinical trial (NCT06514794), which is a phase 2 study evaluating the effectiveness of an investigational anti-CD7 CAR T-cell therapy called WU-CART-007. Early clinical trials showed that WU-CART-007 had a response rate of 80% in adults and adolescents with relapsed or refractory (R/R) disease. WU-CART-007 was developed at Washington University School of Medicine by Matthew Cooper, PhD, to target CD7+ malignancies.
“To have a new treatment option that could potentially have the same dramatic response in kids as in the adult population would be amazing,” said Jeffrey Bednarksi, MD, PhD, director of the Stem Cell Transplant & Cellular Therapy Program at St. Louis Children’s Hospital. “To have it developed at Washington University highlights the tremendous expertise campus-wide in translating exciting basic research efforts into clinical trials.”
Washington University pediatric hematologist and oncologist Thomas Pfeiffer, MD, leads the pediatric clinical trial at Siteman Kids at St. Louis Children’s Hospital along with Bednarski. A similar trial is underway for adults with R/R T-ALL/LBL at Siteman Cancer Center at Barnes-Jewish-Hospital.
While T-ALL/LBL is rare, it is considered an aggressive cancer. Most patients respond well to standard-of-care chemotherapy prior to a hematopoietic stem cell transplant; however, in those with relapsed or refractory cancer, treatment options are limited.
“The last time we had a new therapy for these cancers was 10 years ago when a novel chemotherapy agent was developed that was successful in 25-30% of relapsed patients,” Bednarski said. “WU-CART-007 could be a game changer if it works to move almost all patients from disease-state to remission so that we can then schedule a transplant.”
“WU-CART-007 has received Fast Track and Orphan Drug approval from the Food and Drug Administration, and a similar designation in Europe,” said Washington University pediatric oncologist Shalini Shenoy, MD, one of a team of pediatric oncologists involved in this latest study. “It is not an upfront therapy but is only for those who have relapsed or who have refractory disease. We know that it already has shown a definite and significant benefit in adults and some adolescents, and the FDA designations will speed up implementation and reviews of pediatric clinical trials for the therapy.”
In addition to the U.S., these latest clinical trials of WU-CART-007 will enroll pediatric and adult patients in Europe, Asia and Australia.
“Time is of the essence for children with T-ALL/LBL who have relapsed,” Shenoy said. “What sets Siteman Kids apart is that we have the infrastructure in place to expedite care and insurance approvals, and we have a dedicated nurse navigator to help families make the transition to our institution. We can see patients within a week of referral.”
Bednarski agrees. “Here, we have seamless integration between basic science and clinical medicine. We have a product that can be used immediately — off-the-shelf — so that as soon as children are approved for the clinical trial, we can start therapy,” he said.
Participants in the clinical trial will be hospitalized at St. Louis Children’s Hospital for about a month. They first will receive lymphodepleting chemotherapy and then a one-time infusion of WU-CART-007.
“We will see evidence if the product is working in about a week,” Bednarski said. “In some patients, in the earlier studies, complete remission was documented about a month after infusion, which then allows us to schedule a bone marrow transplant.”
In addition to Pfeiffer, Bednarski and Shenoy, other physicians within the program include Robert Hayashi, MD, and Melissa Mavers, MD, PhD.
For more information about the study or to enroll a patient, click here.