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

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.

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.

What is functional precision medicine?

Treating cancer is like navigating a gigantic maze. Now, imagine a way to see a clear path forward toward better treatment options. That’s the goal of functional precision medicine.

In the wake of the relapse, Logan’s oncologist Dr. Maggie Fader — who led the clinical trial with Azzam — along with his entire team of doctors started searching for a more tailored treatment. They quickly enrolled Logan in the clinical trial.

“What’s unique is it’s easy and fast to get results about what would be the most optimal regime, so we could act quickly,” says Fader who is employed by KIDZ Medical and cares for patients at Nicklaus Children’s Hospital. “That’s important because we’re talking about a child with cancer getting worse day by day.”