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New Treatment Approach for Hard-To-Treat Cancers Shows Promise

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Today, Logan Jenner is a healthy, happy 8-year-old who jumps tirelessly on his trampoline, dreams of visiting Hawaii and plans to tackle every sport imaginable — from basketball and soccer to surfing and snowboarding.

Once, he was Patient 13a case of relapsed acute myeloid leukemia.

Logan was part of a small, diverse group of children with various advanced, relapsed pediatric cancers who took part in Diana Azzam’s clinical trial conducted in collaboration with Nicklaus Children’s Hospital and funded by the Florida Department of Health Live Like Bella Pediatric Cancer Research Initiative. Each one of them lacked treatment options and time. They were in desperate need of new alternatives to attack aggressive tumors.

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The hope: A treatment of last resort might be found by Azzam and her team at the FIU Robert Stempel College of Public Health & Social Work.

In a race against the clock, Azzam’s lab became the first to successfully guide personalized treatments for some of the deadliest, hard-to-treat children’s cancers through a unique functional precision medicine approach.

The process involves small samples of a patient’s tumor rush-delivered straight to Azzam’s campus lab. Cancer cells are enriched and processed in the lab in a way that closely resembles how they would normally grow in the body. Then, they're exposed to over 120 FDA-approved drugs, including both cancer and non-cancer drugs like statins and allergy medications. The best cancer destroyers are singled out. The entire process takes around a week.

According to study results, published in Nature Medicine, this approach provided safe and readily accessible, effective and affordable options for patients without other alternatives. Most important, those recommendations impacted the children — 83% showed improvement, including Logan.

“In this feasibility study, we were able to show what works in the lab works on a patient's tumor,” says Azzam, an assistant professor of environmental health sciences and Society for Functional Precision Medicine board member. “I hope the more data we generate we’ll be able to convince more physicians that functional precision medicine should be used — not only as a treatment of last resort, but first choice.”  

A new type of personalized medicine

Typical cancer treatment relies on standard of care — the well-studied, statistically proven regimen that benefits many people.

Cancer, however, is a wild card. The one size-fits-all strategy simply does not work for everyone. A single drug can have radically different responses in different people.

Personalized cancer treatment aims to turn how the disease is normally treated on its head. It’s about getting rid of the guesswork by matching “the right drug for the right person at the right time,” Azzam explains.

One of the most used precision medicine tools is genomics — DNA-profiling of a patient’s cancer. Existing processes can look at thousands of genes but take several weeks. Even then, not everything about how to treat the cancer may be revealed. Recent data suggests only a small minority of adults, around 10%, benefit from therapies guided by genomics alone. The percentage is suspected to be less in children.

Azzam’s approach includes a genomics component. But what’s different is its “functional” ability to see how living cancer cells derived from a patient tumor sample respond in real time to extensive drug testing.

This expands the horizon of possible treatment options. In fact, 88% of patients received treatment recommendations from the drug testing. And those whose treatment was guided by those recommendations showed improvement — like Logan. 

Patient 13

Doctors diagnosed Logan with acute myeloid leukemia, cancer that starts in the blood and bone marrow, at 3 years old. Three lines of chemotherapy were followed by a bone marrow transplant. He reached remission in 150 days.

At age 5, his cancer came back.

“Suddenly the world stopped,” remembers Logan’s mother Diana Jenner.

“Logan was not going to be a little science experiment. They were going to take his cancer cells and test the medications on those.”

Those tests guided Logan’s treatment. Results revealed which combination of drugs would likely work for him and which might hurt his chances. In 33 days, he reached remission — faster than his previous treatment when he eventually relapsed. More than two years later, Logan remains cancer-free.

Jenner keeps the results from Logan’s doctors’ visits scattered within reach — her bedroom, car, even Logan’s coloring books. She also keeps the results from the clinical trial and has a special plan for them. “I want to put them in a frame for Logan because this is what saved his life.”

Other children in this study also received guided treatments.

Recommendations were returned to 19 patients who had both blood and solid tumor cancers. Six received subsequent guided treatments and five, including Logan, showed improved survival outcomes over the course of the study. In contrast, seven out of eight children who did not receive the guided treatment recommendations did not show improvements.

Those other patients will likely stay anonymous to Azzam. Medical studies, like this one, follow strict standards to protect participant privacy — especially important when human lives are on the line.

Azzam is the first to admit she was never meant to know the identity of Patient 13. Only a chance encounter changed everything.

Jenner happened to be at the Live Like Bella Pediatric Cancer Research Symposium Azzam was attending. “I think my son was a part of a study like yours,” she told Azzam, having no clue the person before her played a key role in saving her son. After a brief conversation, Azzam connected the dots.

It could only be one child: Patient 13.

Since then, Logan has visited Azzam in her lab at FIU. The doors are always open for him.

And hopefully many more people fighting cancer, Azzam says.

“This study really breaks the conventional lines of how you treat cancer in children. It’s something that is extraordinarily positive and changing the world,” says Raymond Rodriguez-Torres, chairman of the board of Live Like Bella, a nonprofit founded in memory of his daughter who passed away from cancer in 2013. “When I'm receiving phone calls from families across the country wanting to know how they enroll in a trial happening at FIU, that's when I know we are doing the right thing. That's when I know that FIU is changing lives. And that's when I know my daughter Bella is smiling from heaven.”

The next phase

Azzam’s research is ramping up. Larger personalized cancer treatment clinical trials for children and adults are ongoing. More will be needed to gather additional data.

The study shined a light on another line of inquiry to explore: Minority populations respond to FDA-approved drugs differently from one another and white patients. Based on this, Azzam spearheads a NIMHD-funded research project as part of FIU’s Research Center in Minority Institutions to reduce health disparities in childhood cancer patients by identifying biomarkers expressed in minority populations and finding more effective targeted drugs.

Additionally, Azzam’s lab is set to become the first federally certified large-scale lab dedicated to functional cancer drug testing in Florida with a $2 million appropriation from the State of Florida spearheaded by State Senator Bryan Avila and former State Senator Anitere Flores.

“When I see Logan laughing and playing,” Azzam says, “I just know we're on the right path of helping revolutionize cancer treatment.”

Reference: Acanda De La Rocha AM, Berlow NE, Fader M, et al. Feasibility of functional precision medicine for guiding treatment of relapsed or refractory pediatric cancers. Nat Med. 2024. doi: 10.1038/s41591-024-02848-4

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