Study Will Test CAR T-cell Immunotherapy in Young People with Brain, Spinal Cord Tumors

Study Will Test CAR T-cell Immunotherapy in Young People with Brain, Spinal Cord Tumors

The Seattle Children’s Hospital is conducting a pioneering clinical trial testing engineered immune cells as a treatment for children and young adults with a kind of brain cancer that produces the HER2 factor.

The Phase 1 trial, called BrainChild-01 (NCT03500991), is now recruiting. To be eligible, patients must be 26 or younger, have undergone surgery to remove a tumor, and have failed first-line therapy.

Patients enrolled in the trial will receive an infusion of cancer-fighting chimeric antigen receptor (CAR) T-cells via a catheter directly into the region of the brain where the tumor was located.

This is expected to improve the treatment’s effectiveness and reduce its side effects, compared to therapies that are injected into the blood.

CAR T-cell therapy involves collecting a patient’s own T-cells and genetically engineering them to bear receptors at their surface, called chimeric antigen receptors (CARs), that recognize and bind to a cancer-specific protein.

Researchers in the trial are testing CAR T-cells that were modified to recognize cancer cells producing the HER2 protein. This protein is found mostly in cancers of the breast, but brain tumors also have been found to produce excess HER2.

“In looking for a common denominator that is present on many types of tumors, but not present in healthy brain tissue, we were pleased to find that HER2 could be a common thread that helps us target several of the brain tumors we treat in children and young adults,” Nick Vitanza, MD, a neuro-oncologist at Seattle Children’s and lead investigator for the BrainChild-01 trial, said in a press release.

Tumors of the central nervous system — in the brain and spinal cord — are the most common in children, and the leading cause of cancer-related deaths in people younger than 19.

While standard therapies are effective in about 70 percent of patients with newly-diagnosed CNS tumors, as many as one third of them will see their cancer return.

“While survival rates have improved, many of the children we care for have no life-saving therapy options if their disease recurs,” said Vitanza. “We have to find a way to give them a life after they recur — and ultimately — be able to offer initial treatments with fewer long-term side effects.”

The trial is planning to recruit up to 36 patients whose tumors are positive for HER2. Patients will be included in one of two groups, depending on the location of their tumors: some will receive the treatment directly into the tumor cavity, while others will have CAR T-cells injected into the spinal cord.

The trial’s primary goals are to establish the therapy’s safety and the feasibility of producing and delivering these CAR T-cells into the brain or spinal cord. Secondary measurements include the distribution of the CAR T-cells in the blood and cerebrospinal fluid, changes in HER2 levels, and disease response.

More information on the trial is available here.

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