Lily-Mae was barely 4 years old when she was first diagnosed with stage 4 neuroblastoma. Neuroblastoma is a deadly cancer that develops from immature nerve cells and occurs most often in infants and young children. Her parents were given very little hope for her condition.
For two years Lily-Mae endured a brutal regime of chemotherapy, radiotherapy, and a bone marrow transplant after which she was declared cancer-free. But the doctor warned them that there was only a small chance she would remain that way as neuroblastoma has a relapse rate of 50% with a low survival rate for those who relapse.
Her parents looked for ways to keep her healthy and cancer-free and were fortunate to find Dr. Sholler who was doing research into neuroblastoma at the Spectrum Health Helen DeVos Children's Hospital in the US. Dr. Sholler was researching a drug called DFMO (difluoromethylornithine) and was running a clinical trial to see if it could prevent relapse of neuroblastoma.
Lily-Mae was a potential candidate for the trial and her parents enrolled her in the study. To raise funds to pay for the treatment some of Ireland's most popular singers banded together and produced a cover version of Elton John's "Tiny Dancer" to honor Lily's love of dance. It quickly topped the charts in Ireland and Lily-Mae even got to meet Elton John in person.
She took her first pill in November 2013 and continued to take pills every day for two years. Then she continued to receive periodic scans and checkups for another two years.
On her 10th visit, Lily-Mae greeted Dr. Sholler with hugs. Her mother thanked Dr. Sholler who exclaimed “She's perfect. She's wonderful, she's as good as she could ever be.”
Unlike chemo drugs, the DFMO pills did not cause uncomfortable side effects for Lily-Mae, and she happily takes her tablets every day. She lost a little hair at her temples, but that was it.
The DFMO phase 2 clinical trial has demonstrated a 97 percent overall survival rate at two years for the 101 children taking part in the study, Dr. Sholler said.
Lily-Mae has become curious about her disease, asking a lot of questions about her treatments, what neuroblastoma is and why she got it – questions that her parents don't have all the answers to, but they explain the best they can.
Lily-Mae loves to dance and sing, and is working towards becoming a rock star! “She is a little miracle,” her mother says. “We are so grateful.”