Access to a clinical trial got this active kid back to playing the sports he loves

Ashton was an active boy with lots of energy. He had just started kindergarten and hockey and loved being on the ice.
In 2014, at 4 years old, Ashton’s mom Kayley noticed a lump on Ashton’s neck. When the lump did not go down after a few weeks, Kayley took Ashton to their local doctor who sent him for a biopsy and an ultrasound where many more lumps were found throughout his neck.  A biopsy of the lump came back as positive for cancer and a chest x-ray was ordered along with more imaging of his neck, head, and chest to track the progression of the cancer.  One month after Ashton’s 5th Birthday he was diagnosed with stage IV thyroid cancer as the cancer had metastasized to his lungs and lymph nodes throughout his neck and chest, with one tumour wrapped around his heart.
Ashton’s hospital had never encountered a child as young as him with thyroid cancer. Thyroid cancer is an unusual and very rare diagnosis for a child, and especially with the level of progressive disease that was found throughout Ashton’s body. Because of the complexity of his cancer, Ashton’s care team consulted with doctors around the world to construct his treatment plan, including doctors who had treated children affect by the Chernobyl disaster.
In December 2014, Ashton had surgery to remove the tumours at the Hospital for Sick Children in Toronto (SickKids). It was the second difficult surgery Ashton had encountered in his young life, as he had open heart surgery when he was only six months old. Because of concerns with his heart wall and how the previous scar tissue may complicate the surgery to remove the cancerous lymph nodes in his chest, three very specialized surgeons performed the tumour removal, cardiac, thoracic and ENT surgeons.  Ashton’s recovery from the thoracic surgery was long, requiring a month long stay at the hospital.
In February 2015 at 5 years old, Ashton entered the next phase of his cancer treatment, which involved Radioactive Iodine (RAI).  It was the first time that RAI would be administered in a children’s hospital, and the hospital brought in lead shields to create a make-shift RAI room. Everyone who came into Ashton’s room wore lead suits, and the ICU nurses were provided with special training for the RAI treatment. At this point, Ashton’s mother Kayley had just had a baby and was unable to be with Ashton due to the radioactivity.  Shayne, Ashton’s father, stayed with him in hospital for five days until it was safe for Ashton to return home.  When Ashton returned home, his family had to take many precautions due to the small amounts of remaining radioactivity from the RAI therapy. Aside from the dangers of RAI, the side-effects for Ashton were mostly a change in appetite, where his favourite foods didn’t taste the same anymore.
Thyroid cancer releases a protein called thyroglobulin. At diagnosis Ashton’s thyroglobulin was at 16,000 but after RAI it came down to 5,000. However, in November 2015, Ashton’s thyroglobulin began to rise, and RAI therapy was performed once again. After the second RAI treatment Ashton recovered and things mostly returned to normal as he returned to school and was able to get back to playing sports.

In the Winter of 2017, Ashton’s breathing became laboured and his parents knew that something wasn’t right.  He was struggling to play hockey and didn’t have his typical stamina. Ashton’s doctors discovered that the cancer had progress in his lungs and as a result, Ashton had to rely on supplemental oxygen.
Ashton’s care team looked into the possibility of another RAI treatment; however, he had already had two large doses, getting close to reaching his lifetime maximum.  After testing Ashton’s cancer, it was also found that his cancer was no longer absorbing the radioactive iodine, making it an ineffective treatment.
In May 2017, Ashton’s care team began exploring alternate options. Ashton was approved to receive a drug called lenvatinib and he begin treatment with some improvement. It was then that his family heard from SickKids who asked if they would be willing to approve genomic sequencing on Ashton’s tumour through their KiCS program.
Ashton’s Endocrinologist in Calgary received the call from SickKids letting him know that Ashton’s tumour showed an NTRK mutation. Around the same time, his doctor also received an email about clinical trial for a drug called Loxo101, or larotrectinib, which targeted the NTRK mutation. A new lifeline opened up for Ashton. The lenvatinib treatment was stopped immediately to allow Ashton’s body a healing period before he started the new trial for larotrectinib.
Ashton’s Oncologist applied to Alberta Health to access the clinical trial in Seattle; however, the application was denied.  Their Oncologist talked to the pharmaceutical company who agreed to cover many of the costs associated with the clinical trial.
Ashton received his first larotrectinib treatment in July 2017.  His first visit in Seattle was 5 weeks long, after this, the family travelled to Seattle once a month.  Due to COVID-19, February 2020 was their last trip across the boarder. Approval was given to send Ashton’s medication through the mail and Ashton’s visits with his Seattle care team have been conducted virtually since then.  He is able to have all tests required for the study done at Alberta Children’s Hospital which has been extremely beneficial to the family. 
As a part of the clinical trial, Ashton’s cancer is monitored regularly.  In October 2020, the scan showed a dramatic difference and a reduction of his cancer, with many large spots no longer visible.
The clinical trial is five years in duration.  Ashton takes larotrectinib twice a day and has been receiving the treatment for 42 months, with 18 months left. He has had elevated liver enzymes on and off, however this has not required his treatment to be stopped. It’s important for Ashton’s treatment to stay on schedule, if his medication is given a little late he experiences aches and pains throughout his body.
Being on larotrectinib dramatically improved Ashton’s quality of life and his family has noticed a return to his old self.  He can breathe, this energy came back, the colour came back in his face, he could eat, and he gained weight. Prior to larotrectinib, Ashton’s colour was grey, his breathing was difficult, and it was an effort for him to get from the living room to the kitchen.  He had no appetite and was wasting away. The biggest side effect of larotrectinib has been weight gain, but it is a welcome side effect for a kid like Ashton.  Ashton has ADHD and the ADHD medication he takes decreases his appetite, so an increased appetite for his favourite foods is good for his overall health. Ashton’s family didn’t really realize how sick he was until they saw the difference larotrectinib had on his quality of life.
The family didn’t worry to much about the risks of larotrectinib being a clinical trial, as it was his only option at the time. However, his family did their research and heard positive stories about larotrectinib working for others.  It was enough to give them a leap of faith.  
Ashton is 11 years old now and in grade 6. He’s an active kid who enjoys gaming, soccer, archery, camping and horseback riding. His family is elated to see him back to himself again.