Access to a clinical trial means more time for tea with her stuffed bears

Naomi was a very healthy 2-year-old, who loved to spend time with her big sister Nicole and play with her friends at her local daycare. In December 2017, she came home from daycare with a bump on her forehead. Her parents thought it was a typical toddler bump but became concerned when it didn’t go away after a month. By late January 2018, Naomi visited her pediatrician, who booked an ultrasound, the soonest of which was two months later.
During the two months wait, the bump remained and about a month before Naomi’s scheduled ultrasound, it began to grow along with new swelling around her eye. Her parents rushed Naomi to the emergency at the Hospital for Sick Children (SickKids), where she received a prompt ultrasound and x-ray and was admitted to the hospital. The next day a large team came into the family’s room and delivered the devastating news that it was most likely cancer.
A biopsy of the bump was performed along with other tests including an MRI which showed many other small lesions on Naomi’s bones, but the biggest and most severe was the visible lump on her forehead. Her oncology team diagnosed Naomi with Langerhans cell histiocytosis (LCH), a subset of cancer that can damage tissue or cause lesions to form in one or more places in the body.
Dr. Whitlock and Dr. Abla, oncologists at SickKids, shared that Naomi’s LCH was at this point low risk, as it was only affecting her bones and not yet her organs. She began a chemotherapy protocol for six months with good results. However, an MRI was performed in late June 2018 and unfortunately the neurologist noticed some changes on her pituitary gland and on other areas of her brain.  Naomi’s LCH had relapsed.
After the relapse, her oncologists tried to treat Naomi’s LCH with different types of chemotherapies. However, the second chemotherapy resulted in sever fevers that were difficult to control, and the third chemotherapy was not very effective. Naomi’s LCH was under control but still worrisome for the family.
At this point, Dr. Abla suggested a clinical trial using a combination of two targeted treatments, dabrafenib (a BRAF inhibitor) and trametinib (a MEK inhibitor). When BRAF and MEK molecules are unchecked, they allow cancer cells to grow out of control so by inhibiting them, it stops or slows their growth.  After learning about the clinical trial, Naomi’s family decided that they would give it a try. In January 2019, Naomi was approved for the clinical trial and began receiving the treatment in February. Thus far, Naomi has done very well on the new treatment. Her results are good, and her MRI does not show any further progression of her LCH. They have seen very little symptoms and her forehead has healed. Naomi will be on the trial for at least another year and a half, but so far, she is doing very well.

Naomi’s family is grateful for SickKids and their Care Team. They feel the dabrafenib and trametinib clinical trial is a much better fit for Naomi, over the chemotherapy treatments. They were told of possible symptoms to watch for from the treatment, but so far, they have only seen rashes on her limbs and legs, with no other major side effects. Naomi’s energy level is good, and she is more like herself again.
When Naomi was on chemotherapy, the doctors told her family to keep her home as chemotherapy weakened her immune system. However, with dabrafenib (and before COVID-19) Naomi was able to return to daycare, as her immune system was much stronger. Naomi had good support from the teachers at her daycare and was happy to be back with her friends. Because of the clinical trial, Naomi could return to the life that preceded her diagnosis.
Naomi is still on an intense treatment schedule. She takes her medicine early in the morning and at night before bed on an empty stomach. But her family finds the treatment manageable and appreciates that it doesn’t compromise her immune system. It allows her to get back to a regular routine, which is important for a four-year-old. Naomi’s Care Team removed her port as she no longer needed it to receive chemotherapy, which means her family doesn’t have to worry about a port infection and her overall treatment is a little less complicated.
Naomi is known as extremely brave by the nurses and doctors who treat her. Because she doesn’t have a port, an IV was necessary to put her to sleep for her MRIs, which she would tolerate without tears. Recently, Naomi promised that she would stay very still so that anesthesia wouldn’t be necessary for her MRI. She did very well for a child of only four, keeping still for the entire hour, impressing the MRI team.
In September 2020, Naomi enrolled in virtual JK due to the pandemic. She misses her daycare friends and teachers, however, thanks to the new treatment, she has her full energy back to play with her big sister and her favourite stuffed animals at home.  The family appreciates the supports they received from the SickKids’ teams, researchers, and volunteers through a tough two years and are happy to see Naomi feeling more like herself.