Calvin’s illness was something we never could have anticipated. One weekend everything was normal. He was a healthy kid, skating and doing gymnastics. Then on Sunday he started complaining about a pain in his leg. We thought perhaps he had fallen while playing so we went to the hospital in the early morning, but his pain had subsided and we left without doing any tests.
The next day and night he screamed in pain again and we took him to our family doctor who sent us directly to The Hospital for Sick Children. He obviously had his suspicions but ironically, even though my husband and I both work in healthcare, cancer never crossed our minds. We thought for sure it was a broken bone or worst-case scenario a crazy virus.
On January 31, 2017, we heard the words no parent is ever prepared to hear. Calvin was diagnosed with very high-risk leukemia and given a four-year aggressive protocol.
As you would expect, everything in our family’s world turned upside down. Calvin went from being an active little boy to a very sick patient. My husband Michael and I made drastic changes to our work and home lives to take care of our son. And Calvin’s siblings, Quincie and Payton, had to make many sacrifices as they learned to navigate this “new normal.”
It is impossible to be prepared to tell your 5-year-old he has cancer. He kept asking, “What is happening to me? What did I do wrong?” We are both scientists and it was complicated for us to figure out what to say to him in a way that he would understand. We told him, “There was a mistake in your blood and now we have to get rid of all the bad blood. It was random, like an accident.” For a while that answer wasn’t good enough for him. He wanted to know what caused the accident, which there is no good answer for.
And then of course our other kids had their concerns. When our 7-year-old Payton had a pain, he asked, “Do you think I have cancer?” He heard at school about cancer being hereditary. And the other kids at Calvin’s school had their own questions as well; they wondered if cancer was contagious and why he was losing his hair.
This is where POGO came in. We are lucky here in Ontario because every family is assigned a specialized community cancer nurse when their child is diagnosed with cancer. Tina, our POGO nurse, came to our home to help our family adjust. She visited our kids’ school to address the concerns of the other children and talked to them in age-appropriate terms so they could understand what was happening to Calvin and what the effects of his treatment would be.
These past 12 months have been intense. Helping a family member with cancer is hard; managing cancer for your child breaks you apart. Despite this, we’ve actually been very lucky. I didn’t know how to navigate the healthcare system for my child with leukemia, so our nurse Tina became an important part of our team of support. And while most families have at least one parent who must give up their job, Michael and I have been able to work because our employers have provided us with the flexibility we need to manage Calvin’s treatment.
I work at LifeLabs and my colleagues have been unbelievably supportive of me. In fact Lifelabs is the presenting sponsor of POGO’s Pajamas and Pancakes program, a relevant theme given how much time kids with cancer spend in their pjs. Thank you to my colleagues for supporting our family and for supporting the cause. And thank you POGO for everything you do for kids with cancer and their families.
It is surprising, but the world keeps spinning even when your child is going through something like this, and our family really appreciates all of the help and encouragement.
In November 2017, LifeLabs employees had a Pajama Party and invited Joby MacKenzie to talk about her son Calvin’s cancer diagnosis, treatment and support from POGO. Meet them both in this inspiring video Calvin Visits the Lab.
POGO-FUNDED RESEARCH: An Economic Evaluation of CAR-T Therapy
Acute lymphoblastic leukemia (ALL) is the most common form of childhood cancer and for most patients, the standard chemotherapy protocol works very well with an overall cure rate of 90%. But, there is a subset of this population that is treatment resistant. For these young patients, a relatively new therapy called chimeric antigen receptor T-cells (CAR-T) therapy may be the answer.
CAR-T therapy uses the patient’s own immune system to do the work of destroying the ALL cancer cells. It starts with harvesting immune cells called T-cells from a patient’s blood. Millions of copies of these cells are grown in a lab—engineered to recognize, target and destroy the cancer cells—then infused into the patient’s bloodstream. Once back in the patient’s body, the cells divide and increase in number, creating a massive army against leukemia cells. Importantly, unlike an infusion of a drug, these cells can remain in the body indefinitely, providing constant surveillance against ALL’s return. The results to date are promising, but not without significant risk.
“Like most cancer therapies, CAR-T cell treatment can have severe and potentially fatal side effects,” says Dr. Alexandra Zorzi, pediatric oncologist at Children’s Hospital, London Health Sciences Centre. “The ‘activation’ of your own immune system can lead to the release of too many cytokines, which can result in laboured breathing, high fevers, and potentially life-threatening decrease of blood flow to internal organs.”
But when it works, it works miracles. At least that is what we know in the short term. “CAR-T cell therapy has the potential to be a major game changer in childhood ALL,” says Dr. Paul Gibson, medical officer with POGO. The early results have shown remarkable response rates, even in children who have been heavily treated for ALL previously. Not only is the response rate impressive, but so are the sustained remissions many patients are experiencing. “This is the first true gene therapy in childhood cancer therapy,” says Dr. Gibson. “While very expensive upfront, it may not only save lives, but save children from needing to be treated many more times in the future.”
Currently, this therapy is only offered at the Children’s Hospital of Philadelphia. The cost to send an Ontario patient for treatment is close to $500,000, not including the cost to families who often have to leave work and home for several months.
In 2016, POGO awarded Dr. Petros Pechlivanoglou with a seed grant for his project “Economic Evaluation of CAR-T Therapy for Children with High Risk Relapsed ALL.” Dr. Pechlivanoglou and his co-investigators—Drs. Sumit Gupta, Jason Pole, Paul Nathan, Tal Schechter-Finkelstein and Wendy Ungar, together with PhD student Jill Furzer—are using statistical and mathematical modelling to determine the value of CAR-T therapy from a clinical and economic perspective. What is the trade-off between the treatment’s effectiveness and its cost to the Canadian healthcare system and society overall? Where should we focus our efforts to collect more evidence in the future? And, how will this information be used to inform policy decisions?
“This economic evaluation of CAR-T therapy is only one example of new therapies,” says Dr. Petros Pechlivanoglou. “As new cancer innovations emerge, both in the pediatric and the adult world, we are going to have this challenge of understanding the ‘value-for-money’ proposition again and again. This grant will help us tease out the methods needed to find timely answers to such questions and make prioritization decisions about future clinical, economic and policy research.”
– Petros Pechlivanoglou, PhD
Dr. Petros Pechlivanoglou is a scientist at The Hospital for Sick Children Research Institute and assistant professor at the University of Toronto. His research focuses on methods around the use of health decision analysis, administrative data and prediction modelling in economic evaluation, and the health economics of pediatric oncology and pre-term birth.
The 2017 POGO Multi-Disciplinary Symposium on Childhood Cancer – Cancer in Infancy: Tiny Patients, Huge Challenges – examined clinical and scientific advances in cancer in infancy and the associated physical, psychosocial and medical issues, as well as focus on relevant resources, tools and strategies for a multidisciplinary audience.
Over 230 delegates representing a variety of disciplines attended the conference!
Click on the session titles below to view presentations that POGO has permission to share.
NOTE: The content of each presentation below captures the unedited information and opinions presented by the speakers. Please note that the information contained in the presentations was current at the time it was presented – there may be further information in subsequent literature. Listed speaker credentials were current at the time of presentation.
The Complex Nature of Neuroblastoma in Infancy
Rochelle Bagatell, MD
Developmental Outcomes for Infants with Cancer: Understudied and Underserved?
Melissa Alderfer, PhD
Retinoblastoma: An Eye to the Future
Furqan Shaikh, MD, MSc, FRCPC
In Utero Surgery: Exploring New Dimensions for Infants
Jacob Langer, MD
Anti-Cancer Drug Pharmacology in Infants and Very Young Children
Clinton Stewart, PharmD
Rare Tumours of Infancy
Rajkumar Venkatramani, MD, MS, FAAP
The Art and Science of Keeping Infants with Cancer Well-Nourished
Laura Collins, RD
Debbie O’Connor, PhD, RD
Measuring, Managing and Mitigating Cancer and Treatment Pain in Infants
Anna Taddio, PhD
Jason Thomas Maynes, PhD, MD
Developmental and Psychosocial Aspects of Caring for Infants with Cancer
Melissa Alderfer, PhD
Vanessa Burgess, MScOT, OT Reg. (ONT)
A Fine Balance Infection Control in Infants
Michelle Science, MD, MSc
Dana Devine, PhD
This session will cover unique aspects regarding the presentation and assessment of neuroblastoma in infants. Key features of the natural history of neuroblastoma in infants will be highlighted, as will molecular features of tumours occurring in our youngest patients. Issues pertaining to drug dosing and supportive care for infants with neuroblastoma will also be discussed. In addition, outcomes for infants with neuroblastoma will be compared and contrasted with outcomes in other patient groups.
Rochelle Bagatell, MD
Associate Professor of Pediatrics, Solid Tumor Section Chief, Division of Oncology
Children’s Hospital of Philadelphia, University of Pennsylvania, Philadelphia, PA
Dr. Bagatell is a pediatric oncologist with an interest in extracranial solid tumours. She is the solid tumor section chief at The Children’s Hospital of Philadelphia where she leads a multi-disciplinary team committed to improving outcomes for children with high-risk solid tumours. In addition, she leads clinical research efforts designed to critically evaluate current therapies for children with high-risk and relapsed neuroblastoma, and is committed to conducting studies of new therapies for this population. She is the vice chair of the Children’s Oncology Group (COG) Neuroblastoma Disease Committee, and is chair of the upcoming COG Phase 3 trial for children with newly diagnosed high-risk disease.
It is widely accepted that young age at the time of a cancer diagnosis is a risk factor for poorer neurocognitive development. Tumours and cancer treatments that impact the central nervous system are believed to disrupt the brain growth and development that uniquely characterizes the first few years of life. These concerns have led to clinical trials focused upon modifying treatment protocols to decrease CNS-related insults while maintaining survival rates. However, comparison-controlled prospective studies of the neurocognitive development of infants with cancer are scarce and empirical investigations of interventions to address the poorer developmental outcomes of those diagnosed and treated for cancer during infancy are even more rare. This presentation will provide a review of the current literature regarding neurodevelopmental outcomes and interventions for infants with cancer. It will also summarize current gaps in our knowledge and provide suggestions for moving research and clinical care forward for this special population.
Melissa A. Alderfer, PhD
Senior Research Scientist, The Center for Healthcare Delivery Sciences
Nemours Children’s Health System/Alfred I. duPont Hospital for Children, Wilmington, DE
Associate Professor of Pediatrics
Sidney Kimmel Medical College at Thomas Jefferson University, Philadelphia, PA
Dr. Alderfer completed a doctorate degree in clinical psychology at the University of Utah and a post-doctoral fellowship in pediatric psychology in the Division of Oncology at The Children’s Hospital of Philadelphia (CHOP). After fellowship, she stayed on in the Division of Oncology at CHOP and joined the standing faculty in the Department Pediatrics at Perelman School of Medicine at the University of Pennsylvania. She rose to the level of associate professor before transitioning in 2013 to her current position as a senior research scientist in the Center for Healthcare Delivery Sciences within Nemours Children’s Health System. Under the broad umbrella of healthcare delivery science, her program of research focuses on how childhood chronic illness impacts families, how families adapt and learn to manage illness and the healthcare system, and how we can intervene to improve the experience. Her research has been funded by the National Institutes of Health, American Cancer Society, and various other cancer foundations. She currently has 92 peer-reviewed manuscripts and chapters.