Clinical trial to test immunotherapy for hard-to-treat leukemia

The treatment was seen to be effective in 70 percent of patients in a smaller study in 2014.

By Stephen Feller

LOS ANGELES, March 29 (UPI) -- Doctors at the Children's Hospital of Los Angeles announced the start of a trial for immunotherapy treatment in children with acute lymphoblastic leukemia, or ALL, one of the most common cancers found in children.

The treatment, called KTE-C19, will be tested on 75 children between ages 2 and 21 with treatment-resistant forms of ALL following significant success in a previous, smaller trial.


Immunotherapy is one of the most promising forms of treatment because it uses the body's own defenses to fight cancer by teaching it to target susceptible molecules on the surface of cancer cells.

"This approach has ushered in a whole new era of cancer immunotherapy," Dr. Alan Wayne, director of the Children's Center for Cancer and Blood Diseases at Children's Hospital, said in a press release.

Researchers have started recruiting patients for the study, which will enroll a total of 75 patients and be comprised of two phases: An initial 30-day phase judging toxicity and safety, and a second phase focused on eight-week remission rates. The participants will then be followed for 12 months, the secondary measure for remission linked to treatment with the drug.


For each patient, researchers will take immune T cells from a patient, modify them to expressed chimeric antigen receptor, which motivates them to seek the CD19 antigen, a protein expressed on the cell surface in most ALL cases.

A previous study by Wayne, published in The Lancet in 2014, found 70 percent of children in the study showed complete response to the treatment, where previously they had either relapsed or their cancer was resistant to chemotherapy.

The new study is expected to conclude in June 2017.

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