There are only so many things a medication can do for a patient. That certainly was the case for young Piper Diamond and her mother, Rhonda, after Piper entered a clinical trial for maintenance therapy following surgery and treatment for metastatic neuroblastoma.

The trial medication could not return the kidney Piper lost because of the tumor that had been in her stomach. It could not repair the hearing damage related to her post-surgery chemotherapy. And it certainly could not do anything about the passing of her father, who died in a car crash while Piper still was undergoing treatment not long after her second birthday.

What the medication could do was help Piper start down the path toward having a somewhat regular childhood once again — and that is exactly what the drug difluoromethylornithine (DFMO) has done.

Following a nine-month stay at Children’s of Alabama – during which, Piper received chemotherapy, radiation, and autologous stem cell transplant – and then several more months of treatment, Piper was enrolled into a clinical trial through the Beat Childhood Cancer Research Consortium to see if the medication could help prevent recurrences of the cancer. She took the drug twice a day from November 2022 through November 2024.

"The trial has helped her progress. It helped her grow. She's able to do things that a normal six-year-old can do. She's starting to live life like a normal child."

Rhonda Diamond

Nearly two years after completing her participation in the trial, Piper remains cancer-free. In fact, the trial was successful enough that DFMO has received FDA approval and is now offered as a brand-name medication called Iwilfin that reduces the risk of relapse for patients with high-risk neuroblastoma.

“The trial has helped her progress. It was helped her grow,” Rhonda Diamond says. “She’s able to do things that a normal six-year-old can do. She’s starting to live life like a normal child.”

UAB pediatric hematologist and oncologist Elizabeth Alva, M.D. – who has been closely involved with Piper’s care – says the primary goal of the trial was to produce a medication that could help prevent the numerous side effects that often occur during standard-of-care treatment for high-risk neuroblastoma patients.

“This is one of most common solid-tumor malignancies in childhood, but when it presents as metastatic, it requires intensive treatment,” Alva says. “We kind of throw the entire kitchen sink of oncology care at these kids, and these treatments can be very toxic with a lot of side effects. There is a lot of cost to the cure.

“This new medication is great because it’s well-tolerated. It’s just an oral medication, so it doesn’t require being in the hospital or a clinic. It’s a way to potentially improve outcomes without a lot of additional burden. It’s improving care without worsening toxicity.”

While Piper still has to deal with the aftereffects of her medical ordeal, her mother says that all signs point toward continued recovery and a productive life.

“She has some challenges, but we’ll take it because she’s alive,” Rhonda Diamond says. “It’s really amazing, everything she has been through and overcome. She’s a blossoming child now. She still gets sick easily, but overall, she’s progressing well.

“I know it’s a scary thing when someone offers a clinical trial treatment on your child. In a way, it was a gamble. But sometimes, gambles are what change you. It definitely changed and saved Piper, and because of that, it’s also able to save others.”