Vanessa and Brian are typical first time parents. They adore their two-year old daughter Adeline (Addie for short), and take much pride and pleasure in watching her learn to speak and assert her personality.
“Addie is very friendly,” said Vanessa. “She loves being around other kids, spending time at the park, and playing princess dress-up.” In fact, in most ways, Addie appears to be a normal toddler.
However, Addie’s young life is far from typical. At five-months old Addie was diagnosed with Embryonal Tumor with Multilayered Rosettes (ETMR), a serious brain tumor primarily affecting children under four years old. ETMR has a less than a 10% survival rate.
The first few months of Addie’s life were uneventful, as she developed normally. Then, in December 2016, Vanessa and Brian found themselves at the Joseph M. Sanzari Children’s Hospital Emergency Department with baby Addie. She was unable to stay awake, vomiting and severely dehydrated. After performing several tests, including a CT scan, the physicians discovered a 5 centimeter mass in Addie’s brain.
Emergency surgery was performed, and Addie spent the next 12 days in the Pediatric Intensive Care Unit.
“I had a feeling that something was wrong. When the news came it was devastating. I didn’t stop crying the entire time she was hospitalized. We really didn’t know what to do,” recalls Vanessa.
Vanessa and Brian first met Derek Hanson, M.D., section chief of pediatric neuro-oncology, in the PICU. “He visited us every day that we were there. He stayed and spoke to us. He made us feel as comfortable as possible.” Once the tumor was identified as ETMR, Dr. Hanson knew he had to think outside the box.
“Looking back on previously reported cases of ETMR, it was clear that aggressive treatment alone would not lead to success. Instead of hitting Addie’s tumor hard, I knew that we needed to hit it smart and attack the underlying biologic mechanisms that were key to the tumor. In reviewing the published research on ETMR, I found a pre-clinical drug screen that identified the most effective chemotherapy agents used to treat an ETMR cell line. I then created a modified treatment plan for Addie using surgery, chemotherapy, and stem cell transplant.
If this new treatment regimen were to work, Adeline would still be at very high risk for relapse. Therefore, following the completion of Addie’s up-front treatment, she was started on an 18-month maintenance chemotherapy regimen with DFMO, a drug we are currently using in a clinical trial to combat neuroblastomas.”
Dr. Hanson’s new protocol proved to be successful. After undergoing four rounds of chemotherapy and a stem cell transplant, and receiving the experimental drugs, Addie received her first clean scan in September 2017. To date, her scans remain cancer free.
Vanessa and Brian are incredibly grateful to Dr. Hanson and his perseverance. “We could tell that he was personally invested and really cared,” said Vanessa. “When we were first diagnosed we wanted the best treatment. Ultimately we stayed at Hackensack because Dr. Hanson found the best treatment for us. Dr. Hanson was very willing to collaborate with us, and make us part of the care team. He gave us options and consulted with others.”
It’s obvious that Addie is a fan too! Vanessa recalls coming to the hospital for Addie’s speech therapy, and she insisting on visiting Dr. Hanson. “He came and gave her hug. It made her day!”
Recently, Addie finished her drug trial, and now her family is taking more steps towards having a typical routine. “It feels scary – it’s not real yet. After 26 months of chemo and all of the treatments, it is scary to think we are done. We are hopeful that this will be over.”
Addie does have some side effects from treatment. Most significantly is a hearing loss that is treated with hearing aids and speech therapy. She is delayed in some of her gross motor skills, but recently learned to run.
“After being given the news that your child is most likely going to die, every single day and every single milestone is that much more important and special. We can’t take anything for granted. It taught us to be happy with the small things and value family and friendships,” Vanessa said.
Dr. Hanson is utilizing Addie’s response to create an ETMR protocol for other children diagnosed with the same disease.
“Adeline’s response to the chemotherapy treatment showed that a biology-based treatment approach can be successful in ETMR. Now we are looking to make this biological approach available to more children with ETMR.”
“Through our Tackle Kids Cancer funding we will be launching an international registry and research platform for children with ETMR. The registry will collect clinical information from ETMR patients from across the globe and available tumor specimens. This information will serve as a resource for physicians and researchers to improve our understanding of ETMR and develop better approaches to treatment and overall care of children with ETMR.”
“In addition, the tumor specimens collected from the registry will be used to produce multiple ETMR cell lines, which can then be used for translational research exploring new therapies for ETMR. It is our hope that this comprehensive clinical and pre-clinical research platform will vastly expand our understanding of ETMR biology and set the new standard for ETMR treatment,” explained Dr. Hanson.
Vanessa stresses the importance for research funding. “When we received the prognosis, there was no treatment protocol for ETMR. There are so many rare tumors that need more research. It is not acceptable in this day and age that a child is given a less than 10% chance because there is not enough research. Cancer is not as rare as we all like to think it is. It is so important because these kids deserve a chance. Our kids deserve better than that.”