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The Inside Ride
Clinical Trials Awareness
Access to a clinical trial gave this jokester his sense of humour back
Diagnosis: Acute Lymphoblastic Leukemia (ALL)
Age at diagnosis: 3 years old
Clinical Trial: Car T Cell Therapy
Marky is an imaginative and outgoing kid with a sense of humour beyond his years that often makes his family laugh. In May 2017, Marky’s grandparents were watching him while his mom Heidi was on a short business trip to Hong Kong. They became concerned and took him to see a doctor when an earache caused him to become unusually cranky.
During Heidi’s long plane trip home while she was unreachable, Marky’s doctor assessed Marky and instructed his grandparents to take him directly to the hospital after noticing his spleen and liver were enlarged. Heidi’s father met her at the airport and drove her directly to the Hospital for Sick Children (SickKids) in Toronto. Marky had been diagnosed with acute lymphoblastic leukemia (ALL) and was very, very sick. In fact, he was in the critical care unit for five days before he could be moved to the oncology ward.
Marky’s initial treatment included chemotherapy; however, it quickly became apparent that the leukemia cells were not responding to chemotherapy. His white blood cell counts were very high, so the medical team made adjustments to his chemotherapy regimen. After further tests, the medical team sat Marky’s family down for a serious conversation. While Marky’s leukemia was identified as ALL (the childhood leukemia with the highest cure rate), additional testing revealed that the leukemia cells had two mutations that designated his leukemia as high risk, with a greater possibility of relapse. His leukemia is known as Philadelphia chromosome positive with a rearrangement called mixed lineage leukemia (MLL). At the time of his diagnosis Marky was the first child in the world to have ALL with both of these mutations.
To treat this form of leukemia Marky was placed on strong high dose chemotherapy which made him very unwell. He essentially lived at the hospital from the end of May 2017 until the end of December 2017. In January 2018, his treatment protocol changed to oral chemotherapy that was less intense and he was well enough to spend more time at home.
Marky completed the first phase of his treatment in April 2018 and moved into the maintenance phase, allowing him and his family to live a more normal lifestyle, continuing to take daily oral chemotherapy at home with less frequent visits to the hospital. However, three months later, doctors delivered the devastating news that Marky’s ALL had relapsed.
Marky was admitted almost immediately for very intense chemotherapy to prepare him for a bone marrow transplant (BMT). He spent 95 days as an in-patient, including three weeks in strict isolation. Following his BMT, Marky went home in October 2018 but was only at home for a week before complications set in. He battled many infections and fevers and spent more time in-hospital than at home with his family. In mid January they calculated that in the 117 days since his BMT he had only slept at home 7 nights.
In March, 2019, Marky’s ALL relapsed again. The cancer was very aggressive. His bone marrow was more than 80% cancer cells with a collection of cancer cells behind his eye. Another bone marrow transplant was out of the question as he was very weak from the first one, only six months earlier. The only option was CAR T-cell therapy, a form of immunotherapy where T cells are genetically manufactured using the patients own T cells, giving the immune system the ability detect and destroy cancer cells.
CAR T-cell therapy is a precision-based treatment that is used for patients when all treatment options have been exhausted. For Marky, there were only two options, one was to take him home and make him comfortable, the other option was CAR T-cell therapy. Marky was in a group of about a dozen patients being treated at SickKids in Canada for CAR T-cell therapy for the first time, before that patients had to travel to the USA.
Marky’s family was told that it would be a narrow path, with many opportunities for the treatment to fail. They needed to meet five important milestones for him to qualify for the trial. First, they needed to use chemotherapy to get Marky’s cancer under control, but not in complete remission as the T cells would need some cancer cells for the treatment to work. Second, he would need to get off the immunosuppressants he was taking to stop his body from rejecting his recent bone marrow transplant and to prevent graft versus host disease (he would need to be free of graft versus host disease to qualify). Third, his doctors would need to successfully harvest enough T cells for the genetic manufacturing process, and they needed to get him healthy enough to do the collection. Fourth, they needed the genetic manufacturing of the T cells to succeed, which takes approximately six to eight weeks. And last, they would need Marky to stay healthy. He needed to be free from infection and his cancer stable. This last step required keeping Marky in complete isolation at the hospital.
It was not a smooth journey and there were many bumps along the way. Marky didn’t have a functioning immune system, and as a result, he battled many infections including a very scary moment when an infection caused his body to go into septic shock. A crash cart was parked outside of his door, while his team worked fast to flush his system and Marky’s mom thought she might lose him. Luckily, their efforts were a success and soon Marky was back to his happy self.
Once the CAR T-cells were ready from one of the two manufacturing sites (Marky’s came from New Jersey), they were brought to SickKids. It was unclear if they would be able to proceed with the procedure given Marky’s health, but once the decision was made and the CAR T-cells were prepared, his doctors had only 30 minutes to get the manufactured T cells into Marky’s body before the cells would die. Marky was only the 14th patient to receive CAR T-cells at SickKids, so the process was still very new for everyone. The nurses spent all morning practicing.
The team quickly and carefully transferred the cells to Marky’s room and a nurse stood on a ladder repeating an infuse and flush cycle using gravity, as the CAR T-cells were too delicate for a pump. It was an intense 30 minutes and they finished with only four minutes to spare.
Following the infusion, patients who receive CAR T-cell therapy need to be monitored closely for two weeks for cytokine release syndrome (CRS), when cancer cells release a toxin into the patient’s system which can cause side effects that can be as mild as a fever or as serious as multiple organ failure. Marky thankfully made it through the 14 days with only a low-grade fever.
Marky was in the hospital for many months but was finally discharged in the summer of 2019, 16 days after he received his CAR T-cell infusion. He had many complications during his treatment and has many side effects that are difficult to trace, given the newness of CAR T-cell therapy. He lives with permanent lung damage, and it’s unknown if there will be other long-term effects caused by all of his treatment. The very first patient to receive CAR T-cell therapy was a young girl named
Emily Whitehead who received the treatment in 2012
. Emily is now more than eight years cancer free. Marky’s family is thrilled to have him home and understands what a blessing it is that his CAR T-cell therapy was a success.
Marky is closely monitored by his care team at SickKids. Eighteen months after his treatment, he is in grade 1 and living an active life at home with his parents and older sister. Marky is a video game enthusiast! He loves all things Nintendo - especially if it's related to Mario and his gang or Sonic & Friends.
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