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At just two years old, following a visit to urgent care for a rash that had appeared suddenly, Ailani was diagnosed with a rare form of leukemia. As a high-risk patient, Ailani’s doctors recommended bone marrow transplant as the first line of treatment. However, when the transplant failed to get her into remission, her parents sought out CAR T-cell therapy. Ailani and her family were able to enjoy 9 months of remission before she relapsed again. Over the next few years, Ailani participated in several CAR-T clinical trials, and the Emily Whitehead Foundation funded her participation in the most recent trial. After receiving her fourth round of CAR T-cell therapy in April, Ailani and her family are currently in a “watch and wait” period.

In her own words, Ailani’s mom Princecine shares the story of her daughter’s diagnosis, relapses, treatment journey, and the road to CAR T-cell therapy.

 

Ailani was 2 years old when she was diagnosed.

On March 2, 2019 while visiting family in Texas during our relocation from California to Florida, we noticed a red rash on Ailani’s belly and on her scalp. We later learned it was petechia, a common symptom of leukemia.

At this time she did not appear to be ill, she had no fevers, and she was playing and eating as she normally does.

We took her to urgent care to check out the rash, and was told it was petechia and that she was fine but if I wanted her to have blood work done to take her to the ER, which I immediately did.

The ER did blood work and told us she needed platelets right away. They immediately put her in an ambulance to be transferred to Baylor Scott and White Children’s Hospital, which was 45 minutes away.

Later that evening we were told she has leukemia, but they had to determine which type. They also told us it was standard leukemia and she began a treatment protocol immediately.

Four weeks after diagnosis, we discovered that her leukemia was not standard, but a rare form of leukemia that had a genetic mutation called MLL-r or KMT2A. This changed her diagnosis to very high risk with a poor prognosis. This also meant we needed to go to a larger, more capable hospital. In May of 2019, 10 days after learning of the KMT2A mutation, we relocated to Baltimore and began treatment at Johns Hopkins. The prevailing recommendation from Hopkins was a bone marrow transplant and on July 3, 2019 Ailani received a haplo-identical bone marrow transplant from her dad.

We lived in the Ronald McDonald house in Baltimore for 7 months until it was safe for Ailani to live further away from the hospital. At the end of June 2020, just 3 days before Ailani’s one year transplant anniversary, we found out she had relapsed.

We immediately began bridging therapy in preparation for CAR T-cell therapy. We didn’t have many more options available.

In July 2020, Ailani contracted a fusarium fungal infection in her knee from a tiny scrape. Within days, the fungal infection had spread through out her entire body. She surgery on her knee and had to have a wound vacuum for a couple of weeks. She also had a few fungal lesions removed from the back of her head, leg, and arm. She was incredibly sick and in tremendous pain. We feared we wouldn’t get to bring her out of the hospital alive.

After receiving four granulocyte transfusions, and once her neutrophils returned, she started to improve and was able to receive her CAR T-cells on August 24, 2020.

Ailani enjoyed a wonderful remission. She got healthy and was able to enjoy being a kid for awhile, but unfortunately it lasted only 9 months until she relapsed again in May 2021.

In June 2021, Ailani started 24/7 infusion of immunotherapy as a bridge to a second transplant. In July 2021, we left Maryland to go back to California so Ailani could participate in a bone marrow transplant clinical trial at Stanford Children’s Hospital.

In August 2021, Ailani received a clinical trial haplo-identical stem cell transplant with her mom as the donor. She had many complications afterwards, and unfortunately relapsed again just 2 months post transplant in October 2021.

In January 2022, Ailani was enrolled into Seattle Children’s bi-specific CAR T-cell therapy clinical trial, but due to an FDA pause, Ailani had to wait a few months to receive her CAR T-cells.

In March 2022, Ailani received her bi-specific CAR T-cell therapy. The therapy resulted in some major neurotoxicity that landed her in the PICU for a few days. Fortunately, the neurotoxicity only lasted about a week. Sadly though, the CAR T-cells only lasted 2 months before they wore off and Ailani relapsed again.

In June 2022, Ailani was enrolled in another clinical trial for a menin inhibitor. This was only a bridging therapy and is not considered curative. 

In April 2023, she received her third CAR T trial which was the Humanized 19 Prodigy CAR T cell. She did fantastic and was in remission for 6 months before relapsing again.

In April 2024, Ailani received her fourth CAR T-cell therapy which was a CD 22 clinical trial. Unfortunately, at the 28-day BMA she was MRD positive. However, after another month her BMA showed very little disease. In September 2024, we received her six month bone marrow biopsy, and there was No Evidence of Disease (NED)! This is the first time she’s been  NED after receiving CD22 and the first time in a year since having NED. We are still in a watch and wait period, and her next BMA will be at the nine month mark in December 2024.

Ailani is now 8 years old and feels healthy and normal! She has a sweet and outgoing personality, is very charismatic and a true joy to be around. She’s a very caring and friendly child, who loves making new friends and loves to sing and dance. She really does dance to the beat of her own drum!

We receive messages from patients and families around the world with experiences similar to Ailani’s family. We do whatever we can to be a resource for these families to help them get enrolled in a clinical trial or find a treatment center where they can access CAR T-cell therapy or other advanced therapies.

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