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Seattle Children’s Dr. Nick Vitanza, left, is leading the organization’s BrainChild program. The program aims to create new treatments for children with brain cancer, like Vitanza’s patient and brain cancer survivor Avery Berg. (Andy Rogers Images Photo, courtesy of Seattle Children’s)

When Avery Berg was diagnosed with aggressive, late-stage brain cancer at just 10 years old, she fought hard to stay positive and beat the disease with a smile.

“I didn’t really know what to do next, but I also wanted to beat it and be strong, and meet it with a positive attitude even though I was really scared,” Berg told GeekWire.

Against all odds, Avery did beat her disease. She’s now a healthy 12-year old — but as with most childhood brain cancer survivors, if her cancer comes back, it will be terminal.

Seattle Children’s Research Institute wants to change that, and the organization is launching a new program called BrainChild with the goal of finding better treatments, or even cures, for children with brain cancer. The program is initially focusing on children whose cancer relapsed, but it could expand to include the hundreds of children diagnosed every year with terminal cancers that have no treatment.

BrainChild, which officially launched Tuesday, is a series of clinical trials that will treat these children using CAR T cells, the promising immunotherapy technology that has already led to revolutionary treatments for blood cancers.

“We’re going to be doing this using CAR T cells that are directed towards the molecule on the surface of a lot of brain tumors called HER2,” said Dr. Nick Vitanza, a pediatric oncologist who helped organize the program and is leading the first trial, BrainChild-01.

These treatments work by genetically altering T cells, the immune cells that fight illness like the common cold, to find and destroy cells with certain proteins. The new cells are called CAR T cells and, in this case, researchers will engineer CAR T cells that find and attack cells with HER2 — a protein brain cancer cells have, but normal brain cells do not. HER2 CAR T cells are also being studied as a treatment for breast cancer.

The Berg family shortly after Avery’s diagnosis. Left to right: Avery’s sister Alanah, mom Kristie, father Joe and Avery Berg. (Photo courtesy of the Berg family)

The BrainChild trials are unique for a few reasons. One is the kinds of patients they will enroll: The trial will not be limited to certain types of brain cancer, which is the norm, but will instead enroll any child with recurring brain cancer that expresses the HER2 protein. They will also only enroll children whose cancer has relapsed, giving them no other treatment options.

The trials will also mark the first time this kind of CAR T cell has been directly injected into the brain, a unique and tricky procedure that could make all the difference in how well the treatment works. Some patients will have the CAR T cells injected into the cavity left behind by a tumor, while those with multiple tumors will have the treatment injected into their brain fluid so it can spread further.

Vitanza, who is also Berg’s doctor, said he is optimistic about the trials’ chances of success.

“I am confident of this approach,” he said. He compared CAR T therapy today to the bone marrow transplant, another revolutionary cancer treatment that seemed outlandish when it was first developed. “It was very science fiction-ey, and now it is a mainstay of treatment,” he said.

Dr. Nick Vitanza, right, and brain cancer survivor Avery Berg. (Andy Rogers Images Photo, courtesy of Seattle Children’s)

“Obviously my dream is to cure every single patient we enroll, but it would still be a milestone if we were able to show that any of the tumors are able to get smaller with this treatment,” Vitanza said.

The treatments could spell hope for the hundreds of children in the U.S. who die from brain cancer every year. Some die from cancers that currently have no treatment, but many are children like Avery who survive their first round only to hang on tenterhooks knowing that any relapse is incurable.

Avery’s mother, Kristie Berg, said she worries about her daughter relapsing almost every day.

“If they told us her cancer was back today, her body could not endure the same treatment she’s already gone through. They wouldn’t even offer it to us,” Kristie Berg said, adding that CAR T therapy would be a spark of hope if Avery did relapse. “It’s literally the only option, which didn’t even exist yesterday.”

Vitanza said he hopes the trials will quickly expand into using CAR T therapies for children whose cancer has no known treatments and also using it as a front-line treatment in place of toxic drugs like chemotherapy.

For now, the trials will be based in Seattle, although that could also change. Seattle Children’s recently launched an initiative called CureWorks that will let it expand CAR T clinical trials to patients at children’s hospitals around the country.

Vitanza said his daily work, taking care of sick children and their families, is more than enough reason for him to be excited for BrainChild.

“Every year, I take care of four or five new kids with DIPG,” an untreatable brain cancer, “and every year they all pass away and I go to their funerals and spend time with their families,” Vitanza said. “When you’re in a field with this high a mortality, you end up seeing a lot of young children die.”

“It’s not difficult to be motivated to do this,” he said.

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