Megan was three days away from entering 8th grade when she started to feel an unbearable ache in her legs.

At first, she thought she’d simply slept wrong—but the pain kept worsening. “My mom would wake up to the sounds of me crying in pain,” she recalls.

After over a month of missed school, lost sleep, and repeated hospital visits, her family received the news they’d been dreading: Megan had cancer. The doctors explained that she had B-cell acute lymphoblastic leukemia (B-ALL), the most common type of pediatric cancer. 

At first, her treatment path seemed straightforward. But in Megan’s case, the treatment didn’t work. After four rounds of chemotherapy at her local hospital, Megan's leukemia still wasn’t in remission. That’s when doctors referred her to Children’s Hospital Los Angeles.

Megan was referred to the Cancer and Blood Disease Institute at CHLA, a leader in childhood leukemia treatment and one of the largest and highest-ranked pediatric cancer centers in the country.

Emily Hsieh, MD, a pediatric hematologist-oncologist at CHLA, explains that Megan's genetic testing revealed a rare leukemia mutation resulting in a TCF3::HLF gene fusion.

This subtype of B-ALL is classified as ultra-high-risk.

While standard forms of B-ALL have a survival rate of roughly 95%, Megan’s leukemia type had historically been almost universally fatal.

One option was a bone marrow transplant, which rebuilds a patient’s immune system using stem cells from a matched donor. But for patients to have the best chance at a cure, they need to be in remission first.

Because Megan’s leukemia wasn’t responding to chemotherapy, she wasn’t initially a strong candidate for a transplant.

The CHLA team proposed a newer treatment option. One of the nation’s leading centers for pediatric bone marrow transplantation and cellular therapy, CHLA was participating in a clinical trial exploring earlier use of a type of CAR T-cell (CAR-T) therapy called Kymriah for children with difficult-to-treat B-ALL.

Kymriah works by reengineering a patient’s own T-cells to recognize and attack cancer. 

Megan paints her nails—an activity that helped her cope with long hospital stays.

Even if it couldn’t induce long-term elimination of Megan's leukemia, Dr. Hsieh explained to Megan and her family, the treatment could help put her in the best possible position to receive a bone marrow transplant.

“Very few pediatric centers can provide both CAR-T and bone marrow transplant,” says Dr. Hsieh, Director of CAR T-cell Therapy at CHLA. 

“Having access to both allows us to create more individualized options for children with high-risk cancers.”

“It sounded like something out of a comic book,” Megan says. “The way they explained everything, from collecting my cells to infusing the therapy, helped me feel less nervous.”

Megan hoped that CAR T-cell therapy would clear her cancer completely and eliminate the need for a bone marrow transplant. She hated missing school and was eager to return to her friends and her life. 

When she found out a few months later that she’d still need a transplant, the news was initially devastating.

“My hair was growing back and I was finally starting to feel better, so it was awful to think about going through another long hospital stay,” Megan says.

“My social worker, Sydney, was amazing at helping me cope during that time.” 

Fortunately, three of Megan’s five siblings were perfect bone marrow matches for her. 

The matches were unveiled during an elaborate reveal party, coordinated by Megan’s mom with help from her nurses. 

Dr. Hsieh ultimately selected Megan's oldest sister as her donor.

After nearly two years in and out of the hospital, Megan’s care team delivered the news she had been waiting for: The treatment worked.

Today, four years later, she remains cancer-free.

“From where I am now, it’s honestly hard to picture where I was just a few years ago,” Megan reflects. 

“I was so worried about all the things I was missing out on,” she says. “Now that I’m through it, even the simplest moments are so rewarding.”

Along the way, she's celebrated small joys—eating deli meat again, growing her hair to her shoulders—and major milestones, including returning to in-person classes, getting her first part-time job, and graduating high school in May.

She recently enrolled at a local university and plans to become an MRI tech.

Today, the protocol that helped Megan is widely recognized as an effective approach for certain children with treatment-resistant B-ALL.

Reference:

1. https://www.chla.org/treatments/chemotherapy-treatment

2.https://www.chla.org/cancer-and-blood-disease-institute

3.https://www.chla.org/cancer-and-blood-disease-institute/leukemia-and-lymphoma-program

(Nweswise/AM)