PreviousKelsey is still in remission after receiving an innovative new treatment for infant acute lymphoblastic leukemia
Childhood cancer is thankfully rare, and infant leukemia is even rarer, with only about 90 cases diagnosed in the U.S. every year.
That’s why it took several months for Kelsey, now two and a half years old, to be diagnosed with acute lymphoblastic leukemia (ALL).
Kelsey’s parents said they came home from work one day and noticed that Kelsey, who was 11 months old, had what looked like a bruise on her forehead. When it didn’t get better, they took Kelsey to her pediatrician, who recommended ice. The lump kept growing, and after several more visits to Kelsey’s doctor, she was finally referred to Lucile Packard Children’s Hospital Stanford.
By that point, Kelsey had turned 1 and the lump was about the size of a golf ball. A biopsy confirmed that Kelsey had ALL.
“When we heard Kelsey had cancer, it felt odd because we didn’t know what was going to happen—how she was going to be treated and for how long,” said her dad through an interpreter.
That was when the family met Tanja Gruber, MD, PhD, who is an internationally recognized expert in infant ALL. Dr. Gruber and her laboratory team have spent years developing a new treatment for infant ALL, which traditionally had a five-year survival rate of just 35%–50%—much lower than the 95% cure rate for older children with ALL.
Dr. Gruber’s treatment is called TINI (Total Therapy for Infants With Acute Lymphoblastic Leukemia). The first clinical trial testing this new approach, TINI I, used two drugs that were approved by the U.S. Food and Drug Administration (FDA) but had never been used to treat infants with ALL.
“We took a library of drugs, all of which were FDA approved, and screened samples from patients with infant ALL to see which ones were sensitive to these drugs,” said Dr. Gruber. “We looked at over 1,300 FDA-approved drugs, and two classes of drugs rose to the very top.”
The first was a proteasome inhibitor. Proteasomes are like tiny recycling centers in the cell, breaking down proteins and using the leftovers to make new proteins. Proteasome inhibitors cause the cell to stop breaking down proteins, which ultimately kills the cancer cell because it gets too full of old proteins and doesn’t have material to make new ones.
The other drug is a histone deacetylase inhibitor, which makes DNA in the cells uncoil and can trigger the death of cancer cells.
“These two types of drugs were very active against infant ALL but weren’t being used in standard chemo regimens,” said Dr. Gruber. “The standard of care at the time was to use the same chemo that’s given to older kids with ALL, but those drugs weren’t sufficiently active against infant ALL cells, so there were a lot of relapses. TINI was able to lower the disease burden much more and get patients into remission.”
Now, Dr. Gruber is aiming to improve upon TINI I with a new trial, TINI II.
TINI II combines the TINI “chemo backbone” of a proteasome inhibitor and a histone deacetylase inhibitor with another drug, called blinatumomab. Blinatumomab acts like a connector, bringing healthy immune cells (T cells) to cancer cells so the patient’s immune system can kill the cancer.
Blinatumomab was first piloted by the Interfant Consortium, an international group of doctors and researchers who treat and study infant leukemia. The results of this group’s study showed a roughly 50% reduction in the number of children who still had detectable leukemia at the end of one cycle of blinatumomab.
“I’m hopeful that combining these two new approaches in TINI II is going to be even better than TINI I,” said Dr. Gruber. “When you’re using both, it mitigates the risk that resistance will develop to blinatumomab—blinatumomab works better when there is less residual disease to clean up.”
High-risk infants in the study will also get a new drug called a menin inhibitor that directly targets the infant leukemia cells. TINI II is the only study in the world that is giving menin inhibitors to newly diagnosed infants who are at a high risk for relapse.
By the time Kelsey arrived at Packard Children’s, she was more than 12 months old, making her too old for the TINI II clinical trial. But because Dr. Gruber knew that Kelsey’s ALL symptoms had started before she turned 1, meaning she had infant ALL, and because the TINI chemo backbone and blinatumomab are FDA approved, Kelsey was able to be treated with the drugs anyway.
Dr. Gruber said this individualized, research-backed approach to caring for patients really sets Stanford apart.
“A main focus of our program is to translate lab findings into the clinic, and we have been successful at that,” she said. “We really think about the mutations the patient has, how that affects how they respond to various drugs, and what’s the best therapy for that patient. We give patients state-of-the-art treatment that’s informed by science.”
Kelsey spent two months in the hospital while she received the drugs. Dr. Gruber and Kelsey’s parents said that Kelsey didn’t seem to be fazed by the treatment.
“She did phenomenally,” said Dr. Gruber. “It didn’t matter how intense the chemo was, she just had this very sweet disposition. Kelsey loved everybody, and of course everybody loved her. She was a very happy baby—she has a very bright, wonderful personality and she loves to interact with people and play.”
Dr. Gruber also enjoyed partnering with Kelsey’s family.
“Her parents were very attuned to when she was having a side effect, and they would always bring up important things related to how she was feeling or her development,” Dr. Gruber said. “It was easy to take care of her because I could count on them to tell me when something was off.”
The drugs wiped out Kelsey’s immune system, so her care team and parents worked hard to prevent infections and catch any symptoms early.
“There’s a lot of intensive supportive care to make sure babies get through the treatment,” said Dr. Gruber.
Another source of support for the family was a nonprofit organization named Jacob’s Heart, which helps families living in Santa Cruz, Monterey, San Benito, and Santa Clara counties to navigate their child’s cancer journey. Jacob’s Heart provides everything from groceries and transportation to financial assistance, peer mentorship, and more, based on each family’s unique needs.
After an intense first few months, Kelsey was in remission and was able to go home. Her parents were thrilled.
“It’s something we weren’t able to process in the moment,” Kelsey’s dad said. “I don’t know how to describe it; we cried over it. It felt like life was returning to normal, and we would be able to move forward again. We thank God and thank Kelsey’s doctors, especially Dr. Gruber, who was always there for us.”
Kelsey continued to take oral chemo medication—standard for all patients with ALL—for another year and a half.
“She went through chemo as if nothing was happening, with no difficulties,” said Kelsey’s dad. “She has a will to live; she’s really strong.”
Kelsey officially finished treatment this April, and she’s still in remission. She visits the pediatric oncology clinic at Stanford every three months for monitoring.
“Relapses typically occur within a year of diagnosis, so the risk of relapse is very low at this point,” said Dr. Gruber. “Kelsey’s future looks bright.”
Kelsey also has caught up with all her developmental milestones, which can sometimes be difficult for young kids going through chemotherapy and long hospital stays.
Her parents say she’s full of energy and loves to run and play. They’re all focusing on recovering mentally and physically from this chapter of their lives.
“Hopefully she won’t remember any of this, and it won’t affect her,” said Dr. Gruber. “I know her family will help her move on. They’re very attentive parents, just loving her and taking care of her and doing everything they can for her. It’s really a joy taking care of this family.”
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Authors
- Amy Brooks
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