A potential game changer for childhood cancer

It’s a parent’s worst nightmare: hearing that your child has run out of cancer treatment options.

Now a professor in the Robert Stempel College of Public Health & Social Work is working to change that.

Diana Azzam, pictured above in her lab, has a $700,000 grant from the Florida Department of Health Live Like Bella Pediatric Cancer Research Initiative to identify potentially life-saving drugs for individual patients.

Even with an as-yet limited number of participants involved in the four-year trial, the findings have already proved promising.

Patients are recruited by Nicklaus Children’s Hospital in Miami, and their tumor cells sent to Azzam’s campus lab for testing against hundreds of drugs approved by the FDA, some for cancers and others not. When a drug is identified as effective in destroying the cells in a sample, Azzam reports the match to the particular patient’s doctor. (Unlike in cases of adult cancer, insurance companies will generally pay for medications for children even when deemed “off label,” or for a use other than originally intended.)

Currently, pediatric oncology patients typically undergo one or more rounds of conventional chemotherapy and/or radiation. For those whose cancers
 are resistant to the treatments, Azzam’s personalized approach—called functional precision medicine—could be the answer. Ideally, one day, it would be used as a first resort, she says, thereby avoiding wasted time on therapies that do not help.

“Let’s work together, researchers and oncologists,” she says. “Let’s have this diagnostic way of identifying the drugs that would work for this specific patient and save the trial and error.”

Should the study results support her approach, Azzam foresees starting a company to provide such testing to clinical centers.