“As a parent, you never want to hear that your child has cancer,” said Paul Esposito, of Plano, Texas. “It creates an emotion that starts at your feet and takes hold. It’s devastating.”

This was the terrible news Paul and his family received in 2010 when his son, Zane Esposito, was only 7 years old. Zane, now 12, was diagnosed with acute lymphoblastic leukemia (ALL) in June 2010. Zane underwent three grueling years of cancer treatment, including 365 days of chemotherapy, before reaching remission. Two years later, Zane relapsed in January of this year. Their only option: another three years of aggressive chemotherapy.

“I really don’t like chemo, it’s the worst,” said Zane. “My back hurt super bad due to tiny fractures from the chemo. I couldn’t even bend over to tie my shoes. And here I was having to start another three years all over again.”

Not only was the thought of starting over daunting, but Zane faced a major hurdle as he began chemotherapy – his cancer was not responding to the treatment. He had refractory ALL. Zane and his family were desperate for another treatment option.

About 2,000 miles away in Seattle, Wash., they would find that other option. But first, they would learn about it in the most unlikely place: a doughnut shop.

A sweet tooth leads to an answer

Soon after Zane relapsed, Paul and his family went to the local doughnut shop to satisfy a sweet tooth and enjoy the afternoon together. By chance, they ran into acquaintances who had heard about Zane’s situation. Paul said they approached him and mentioned they had recently seen a documentary about immunotherapy. They advised him to look into it.

“At the time, I just said ‘thank you’ not knowing what to expect, but after researching it, it seemed promising,” said Paul. “We found a trial at Seattle Children’s and knew it was our last hope. I called and left a message with Dr. Rebecca Gardner and she literally called back in 30 minutes. A month later we traveled to Seattle to start treatment.”

T-cell immunotherapy trial shows 93% complete remission rate for children with ALL

Zane enrolled in the Phase 1 clinical trial at Seattle Children’s known as Pediatric Leukemia Adoptive Therapy-02 (PLAT-02). The trial includes patients with relapsed or refractory ALL who have less than a 20% chance of survival upon enrollment with current treatments. Using T-cell immunotherapy, the research team led by Dr. Mike Jensen at the Ben Towne Center for Childhood Cancer Research at Seattle Children’s Research Institute reprograms a patient’s T cells so they can hunt down and destroy cancer cells wherever they are hiding in the body.

The trial has seen great success. Today, Seattle Children’s announced that that 39 of 42 patients treated in Phase 1 have achieved complete remission, showing no detectable leukemia cells in the most sensitive tests.

“Seeing 93% of our patients achieve complete remission is incredibly promising,” said Seattle Children’s oncologist, Dr. Rebecca Gardner, who is the lead investigator for the trial and will present the Phase 1 results at the American Society of Clinical Oncology (ASCO) Annual Meeting on Sunday, June 5. “We also have patients who are still in remission two 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.”
For Paul, he said his family’s decision to enroll Zane in the trial was a “no brainer.”

“The traditional treatments were just not working, and chemo takes a tremendous toll on a child’s quality of life,” he said. “This was the logical next step that gave us a new sense of hope.”

Zane becomes a “T-cell explorer”

Zane during treatment

Zane during treatment

The therapy at Seattle Children’s began by removing T cells from Zane’s blood. Gardner and her team genetically reprogrammed his T cells by inserting DNA that tells the cells to recognize CD19 – a protein found on ALL cancer cells. Gardner said they have been successful in reprogramming and growing T cells for all patients who have enrolled in the trial, a promising finding as similar trials have experienced difficultly producing T cells for all patients.

The engineered cells were infused back into Zane’s blood, boosting his immune system and allowing it to find and attack cancer cells. Zane then waited for the sign that the T cells were at work, a sign that presents in the form of a fever.

“They said I should have a fever in about seven to 10 days, but I told them I was an overachiever and I would have mine sooner,” Zane said, smiling. “Sure enough, I had my fever in six days.”

Zane said the best part of the treatment was how “easy” it was compared to what he had previously experienced.

“Going through immunotherapy treatment was much shorter and simpler than all the chemotherapy, steroids and everything else I had before, with far less side effects,” said Zane. “But the best part was not losing my hair again. My hair is a very big thing to me.”

Five weeks after the start of the therapy, Zane and his family received the news they had been hoping for.

“They told me I had no cancer cells at all – zero percent – and that’s awesome!” said Zane.

“It was like a breath of fresh air,” added Paul. “You couldn’t ask for anything better. We are so grateful to Seattle Children’s for giving us the opportunity to be a part of this amazing treatment.”

Zane said it meant a lot to be involved in this emerging treatment that holds immense promise.

“It’s an amazing experience to be a part of a Phase 1 trial that has the potential to help so many people,” said Zane. “I like to call myself the ‘T-cell explorer,’ and I want to do anything I can to help other kids like me get this treatment.”

What’s next for cancer immunotherapy for ALL

Seattle Children’s will launch Phase 2 of the PLAT-02 trial this month, with the aim to enroll 70 patients in the next year. While Phase 1 of the trial established the safety of the therapy and the optimal dose of T cells, Phase 2 will show how this therapy works in a larger group of patients.

As with all research, the findings from the first Phase of the trial are key to informing what researchers will focus on next to develop the therapy. Of the patients who achieved initial remission in Phase 1, about 50% are still in remission one year after therapy. For the patients that relapsed, researchers discovered 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 like Zane into remission, we are working to ensure the T cells remain a long-term defense against cancer for all of our patients,” Gardner said. “Our ultimate goal is to 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 believe ALL is the tip of the iceberg – work is already underway to apply immunotherapy to other forms of pediatric cancers, like neuroblastoma.”

Celebrating remission and a message for other parents

Zane visiting Mount Saint Helens and celebrating being cancer-free

Zane visiting Mount Saint Helens and celebrating being cancer-free

Soon after receiving the incredible news that Zane was in remission, Paul and Zane celebrated by going whale watching in the San Juan Islands. But, the best celebration was going home to Texas.

“I was so excited to see my family and my dog again, and just be back in school with my friends,” said Zane.

In reflecting on their journey, Paul has a message for other parents who may also someday hear the devastating words that their child has cancer.

“As a parent, you will do whatever it takes to heal your child,” said Paul. “I would tell any parent that if they’ve gone through all the other traditional treatments and you’re looking for hope – an opportunity for your child to potentially be cancer free – then Seattle Children’s is where they should go. This organization and what they’ve done for us and our family is exceptional. Without it, Zane wouldn’t be here today, free of cancer.”

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 immunotherapy treatments. If you are interested in supporting the advancement of this medical breakthrough, please visit Strong Against Cancer’s donation page.

For more information on immunotherapy research trials at Seattle Children’s, please call (206) 987-2106 or email immunotherapy@seattlechildrens.org.

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