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Inquiry in Action

Spearheading New Cancer Therapy

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Seattle Children’s takes a step toward revolutionizing the way cancer is treated with the hospital’s first reprogrammed T cell immunotherapy trial for relapsed leukemia.

In September 2012, Seattle Children’s received the green light to conduct a trial of cancer immunotherapy treatment for children and young adults with relapsed acute lymphoblastic leukemia (ALL) from the U.S. Food and Drug Administration (FDA). The goal is to revolutionize how childhood cancer is treated, and reduce or eliminate the need for chemotherapy and radiation treatments that have debilitating lifelong effects on those who survive cancer.

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“Less than 40% of patients with relapsed ALL typically survive, so it’s imperative that we find new paths to a cure,” notes Dr. Rebecca Gardner, who leads the trial. “Ultimately, we hope to apply this same type of therapy for patients with many types of cancer.”

In cancer immunotherapy, a patient’s own T cells are genetically modified to recognize cancer cells and attack them just as unmodified cells attack a viral infection. Children’s is one of only a few pediatric centers in the U.S. working on an immunological approach to curing cancer – and the only center west of the Rocky Mountains with an open trial and the capability to modify and manufacture the T cells.

Testing for safety; demonstrating viability

Like all Phase 1 trials, the current trial will determine the safety of this therapeutic approach and the dose (in this case, of modified T cells) that can be tolerated. It will also demonstrate that it is possible to genetically modify T cells from relapsed ALL patients who have recently received chemotherapy.

Treatment will consist of two doses of cyclophosphamide followed by a single T cell infusion, and then six weeks of observation and monitoring. Patients will then be monitored for more than a decade to determine long-term treatment efficacy. The treatment sequence is as follows:

• A patient's own T cells are purified, reprogrammed with recombinant DNA and grown to billions of cells.

• The reprogramming instructs the T cells to make an artificial receptor that acts like a Velcro molecule, allowing them to recognize and attack cancer cells as if they were fighting an infection.

• The reprogrammed T cells are reintroduced to the body through a simple infusion – and immediately go to work, hunting down and eliminating the cancer cells.

The reprogramming and manufacturing of the T cells takes place at the Therapeutic Cell Production Core, a GMP (good manufacturing practices) facility in the Ben Towne Center for Childhood Cancer Research at Seattle Children’s Research Institute. The infusion and direct patient care will take place at Seattle Children’s Hospital.

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“Childhood cancers don’t have the same set of biological markers as adult cancers,” says Dr. Michael Jensen, who leads Children’s childhood cancer research center and was the first to describe the specific chimeric receptor for targeting leukemia. “Pediatric cancers must be studied so we can reprogram the specific DNA molecules in T cells that can fight them.”

Philanthropy is funding the work being done in the lab and the research-related patient care costs for this Phase 1 trial. The efforts received a big boost in January 2012, thanks to a $5 million gift from Ben Towne Pediatric Cancer Research Foundation, spearheaded by Jeff and Carin Towne, whose son Ben died at age 3 after a two-year battle with neuroblastoma. In addition, Seattle Children’s Guild Association is providing $2 million to the center as part of its 2012 and 2013 Funding Focus.

 

“Childhood cancers don’t have the same set of biological markers as adult cancers. Pediatric cancers must be studied so we can reprogram the specific DNA molecules in T cells that can fight them.”
– Dr. Michael Jensen