Seattle Children’s Reports Promising Early Results With Immunotherapy in Relapsed ALL

39 of 42 Patients Achieve Complete Remission in Phase I Clinical Trial


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Now that we know we can harness the power of the immune system to successfully get patients into remission, we are working to ensure the T cells remain a long-term defense against cancer for all of our patients.

—Rebecca A. Gardner, MD
Seattle Children’s announced that 39 of 42 patients treated in a phase I clinical trial using genetically reprogrammed T cells to treat relapsed or refractory acute lymphoblastic leukemia (ALL) have achieved complete remission, showing no detectable leukemia cells. Phase II of this trial, known as the Pediatric Leukemia Adoptive Therapy-02 (PLAT-02), is expected to be launched shortly, with the aim to enroll 70 patients in the next year.
 
Rebecca A. Gardner, MD

Rebecca A. Gardner, MD

“Seeing 93% of our patients achieve complete remission is incredibly promising,” shared Seattle Children’s oncologist Rebecca A. ­Gardner, MD, lead investigator of the trial who recently presented these phase I results at the ASCO Annual Meeting. “We have patients who are still in remission 2 years after receiving this therapy. While we still have a lot of work to do, our findings give us tremendous hope, as this therapy offers children, who are otherwise unlikely to survive, a real chance at achieving remission.”

The PLAT-02 trial includes patients with ALL who have relapsed after a bone marrow transplant or who are unable to achieve remission to proceed with a bone marrow transplant. The research team, led by Michael Jensen, MD, at the Ben Towne Center for Childhood Cancer Research at Seattle Children’s Research Institute, is using T-cell immunotherapy to reprogram patients’ T cells to destroy cancer cells. 

Dr. Gardner, who is also an investigator in the Center for Clinical and Translational Research, said he and his team have been successful in reprogramming and growing T cells for all patients who have enrolled in the trial—a promising finding, as similar investigations have experienced difficulty producing T cells for all ­patients.

Tip of the Iceberg

Another key finding from the phase I clinical trial is that of the patients who achieved initial remission, about 50% are still in remission 1 year after therapy. For the patients who relapsed, researchers have found that their reprogrammed T cells are no longer present or the cancer has evolved to circumvent the T cells.

“Now that we know we can harness the power of the immune system to successfully get patients into remission, we are working to ensure the T cells remain a long-term defense against cancer for all of our patients,” Dr. Gardner explained. “Our ultimate goal is to fully develop this therapy so we can offer it to newly diagnosed patients, reducing the need for toxic therapies and minimizing the length of treatment from months or years to only weeks. We also believe ALL is the tip of the iceberg: Work is already underway to apply immunotherapy to other forms of pediatric cancers, like ­neuroblastoma.”

The T-cell immunotherapy trials at Seattle Children’s are funded in part by Strong Against Cancer, a national philanthropic initiative with worldwide implications for potentially curing childhood cancers through immunotherapies. ■



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