Preteen Saves Her Own Life by Catching Rare Cancer Early On

Stem cell transplant at Stanford Children’s helps Lila achieve remission and celebrate two years cancer-free

Intuition is the reason Lila Fowler, age 12, is healthy today.

A little over two years ago, Lila was living a fourth grader’s dream life. She had a close family and lots of friends, enjoyed school, played sports, and had tons of energy and good health.

“Except I had these little red dots on my arms, which were weird. My grandma died of cancer, which shook me to my core, so I didn’t want to let it slide,” Lila says.

The pinpoint-sized red dots, called petechiae, had been something Lila had lived with on and off for several months. Her mom, Elizabeth Fowler, MD, is a pediatrician at the Menlo Medical Clinic, and she and Lila’s pediatrician thought the petechiae were odd, and they were watching it. They didn’t suspect an extremely rare blood cancer called myelodysplastic syndrome (MDS), which occurs in just four children in a million.

It was during a visit to her mom’s office for COVID booster shots that Lila piped up and asked for a blood test. That simple request may have saved her life.

“What 10-year-old asks for a complete blood count (CBC) voluntarily? I give her credit for saving her own life,” says John Fowler, Lila’s dad.

Lila still gets goosebumps just thinking about it.

Once Lila’s mom read the test results, she knew Lila needed more advanced testing. Sure enough, testing revealed MDS based on her blast (abnormal immature white blood cell) count and a genetic mutation. Other than the red dots, Lila had no symptoms.

“I was blown away. I thought about how I was one in a million. I told myself, ‘Lila, you are healthy and strong and you can do this,’” she says.

Getting cancer care at Stanford Children’s

Lila and her family met Norman Lacayo, MD, Alice Bertaina, MD, PhD, and the Pediatric Oncology and Pediatric Stem Cell Transplantation care teams.

“I’ve known Norm since my residency at Lucile Packard Children’s Hospital Stanford in 1999,” says Dr. Liz Fowler, Lila’s mom. “When he determined that Lila had MDS, the first person he called was Dr. Bertaina, who was in Europe on her honeymoon. While we didn’t get to meet her in person for another two weeks, it was very worth the wait. She is a superhuman stem cell transplant magician who is like a loving fairy princess to all her patients. We all adore her!”

Since MDS is extremely rare in children—and much different than the adult disease—Lila’s doctors convened a “tumor board” to gather input and insights from other top pediatric oncologists in the United States to discuss the best next steps for Lila.

“It was inspiring and humbling to see Lila’s oncology team at Stanford reach out to a nationwide network of MDS experts for input, and then work together to come up with the best possible plan to treat her. They even allowed my company’s chief scientific officer, who is a dear friend and blood cancer drug developer, to join these tumor board meetings,” John says. “Norm led this process in the most thoughtful and expert way possible, which allowed us to focus on Lila and feel 100% confident that we were in the right place.”

Patients with MDS, unlike leukemia—another blood cancer—have a low number of blasts. However, those blasts are highly resistant to chemotherapy.

“You can’t cure MDS with chemotherapy. In fact, too much chemotherapy before stem cell transplant is not recommended and can increase toxicity without benefit. A stem cell transplant (also called a bone marrow transplant) is the best option,” says Dr. Bertaina.

Lila’s type of MDS is super-rare. Of the four in one million kids who are diagnosed with it, only 15% have hers. Usually, children are diagnosed with it in much later stages, when they have severe symptoms of fatigue, infections, shortness of breath, or bruising.

Receiving a special type of stem cell transplant

To get Lila to transplant in a timely manner and to lessen the chance of rejection, Dr. Bertaina planned for an alpha/beta T-cell depleted haploidentical stem cell transplant—for which she is the worldwide pioneer and foremost expert. This revolutionary method of transplant lets doctors use cells from a partially matched donor by eliminating the immune system’s fighter cells (alpha/beta T cells) to reduce the risk of graft-versus-host disease (when donated stem cells attack the body). Dr. Bertaina engineered a unique alpha/beta T-cell depleted formula specifically for Lila.

“What makes the alpha/beta T-cell depleted approach so groundbreaking is that we don’t need a fully matched donor. We can use virtually any partially matched related or unrelated donor,” Dr. Bertaina says.

Lila’s donor was her big brother, Will, who had recently turned 17. “He was 100% there. He said, ‘Yes. Let’s do this!’ It was pretty heroic of him,” she says. Her other older brother, 16-year-old Nate, was right there by her side as well.

The stem cell transplant went smoothly for Lila, but it was a lot for a 10-year-old to endure. She felt very sick as she prepared for the transplant with chemotherapy and radiation to essentially wipe out her bone marrow so it could be replaced by her brother’s.

“It felt like I had nothing left. Like I was whitewashed. I would get headaches and throw up, and my whole mouth would burn from chemotherapy,” Lila says. “But after transplant when my stem cell counts were coming up, I could feel like I was growing into a human again.”

The whole process, from diagnosis to conditioning to transplant to engraftment (when transplanted cells start growing into healthy cells) to discharge after transplant took approximately three months, which included an initial hospital stay of a week, followed by a penultimate stay of just over a month to receive her transplant and await engraftment. “The team elected for an intense course of pretreatment since she was still so healthy when she was diagnosed. They thought she could tolerate it and hoped it would reduce her chance at relapse,” John says. “And Lila didn’t succumb to despair. She was brave and positive and never lost her sense of humor.”

Going above and beyond

Lila’s stem cell and oncology doctors also recommended extra, innovative treatments that may help increase the odds that the stem cell transplant will succeed long-term. “I gave her all of the treatments I would have given my own daughter,” Dr. Bertaina says.

For example, while prepping her bone marrow for transplant with chemotherapy, Lila received total body irradiation from Susan Hiniker, MD, radiation oncologist. “It irradiates all the bone marrow in the body to create more space for the donor’s stem cells,” Dr. Bertaina says. “It gave me reassurance that we could possibly cure her forever.” Lila and her parents were worried about potential long-term effects of irradiation, but Dr. Hiniker and her team delivered it in a very innovative way that dramatically reduces the risk of side effects.

Lila did great. She had zero major complications, and she didn’t experience rejection or graft-versus-host disease. Her cell counts steadily climbed after she was discharged, and five months after discharge, her immune system had recovered enough to allow her to return to in-person school to finish out her fifth-grade year.

“She’s a super-fun, very sassy, brilliant girl,” Dr. Bertaina says.

Lila describes herself as “a people person who loves to make good connections with others and who talks a lot and laughs a lot, sometimes too much.”

Back to living her best life today

Lila is actively living her life to the fullest. She started a power-washing business for the summer, and she and her dad enjoy making music together—she plays the piano while he plays the guitar. Over the summer, she went backpacking in the Sierra and the Tetons and went off to sleep-away camp. This fall, she started seventh grade.

Everything—Lila’s strength, her family’s support, and her exceptional innovative and compassionate medical care—added up to a great outcome. It’s been two years since her transplant, and Lila is still in remission.

“It is a big result, and it is a big deal,” Dr. Bertaina says.