Edie was just six months old when she faced a daunting diagnosis: stage four neuroblastoma. This aggressive cancer had spread throughout her abdomen and spine, confining her to the hospital for over four months. Despite undergoing 12 rounds of chemotherapy and multiple surgeries before the age of two and a half, Edie’s journey was filled with moments of doubt and questioning. It was during this challenging time that her family was introduced to the Children’s Hospital of Philadelphia and Dr. Yael Mosei, a beacon of hope and expertise.
Dr. Yael Mosei, a professor of pediatrics at the Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, has dedicated her career to pediatric oncology. Her passion for helping children was ignited when her best friend was diagnosed with leukemia at the age of 21, a revelation that inspired her to pursue a career in pediatric oncology. Dr. Mosei emphasizes the importance of research in protecting children, advocating for breakthroughs that can transform lives, much like Edie’s.
Dr. Mosei’s approach to research is unique; she believes in taking insights from the bedside to the bench, rather than the traditional bench-to-bedside model. This perspective underscores the significance of clinical trials in advancing medical knowledge and translating scientific discoveries into real-world treatments. For Edie, this meant participating in a clinical trial specifically designed for her type of neuroblastoma, which was identified as hereditary through genetic testing.
Upon examining Edie’s medical history, Dr. Mosei discovered tumors on each of her adrenal glands, indicating a genetic predisposition to neuroblastoma. This finding led to the identification of mutations in an oncogene responsible for the inherited form of the disease. Such discoveries pave the way for developing targeted drugs that can deactivate these mutations, offering new hope for patients like Edie.
Pediatric research is a complex field that requires careful consideration of ethical standards and quality. The Pediatric Center of Excellence, a group of dedicated experts, focuses on addressing the unique needs of children in healthcare. Children are among the most vulnerable populations, and the center’s work aims to bring new medicines and vaccines to improve their health and well-being.
Edie’s participation in a phase one clinical trial marked a turning point in her treatment. Despite the challenges, the trial offered a glimmer of hope. The administration of the study medicine was thoughtfully designed, considering the preferences and comfort of young patients. Edie, for instance, could choose a sweet treat like whipped cream or ice cream to accompany her medication, making the experience more bearable.
As the trial progressed, the results were astonishing. Dr. Mosei, initially skeptical, ordered repeated scans to confirm the remarkable outcomes. Edie’s response to the treatment was not only significant but also long-lasting, showcasing the potential of clinical trials to change lives.
Edie’s story highlights the transformative power of clinical trials and the importance of collaboration between medical professionals, industry partners, and families. These trials represent the most scientifically rigorous method to advance cancer care, offering new possibilities for patients worldwide.
Reflecting on her journey, Edie’s family expresses immense gratitude to Dr. Mosei for her unwavering support and expertise. The experience has profoundly impacted their lives, reinforcing the value of research and innovation in healthcare. Through continued collaboration and dedication, breakthroughs like Edie’s can become a reality for many more children in need.
Research the latest advancements in pediatric oncology and prepare a presentation. Focus on how these advancements have improved patient outcomes, similar to Edie’s case. Present your findings to your peers, highlighting the role of clinical trials and genetic research in developing new treatments.
Analyze Edie’s journey through her diagnosis, treatment, and participation in clinical trials. Discuss the ethical considerations and challenges faced in pediatric research. Reflect on how Dr. Mosei’s approach from bedside to bench influenced Edie’s treatment outcomes.
Engage in a role-playing activity where you design a clinical trial for a pediatric cancer treatment. Consider factors such as patient comfort, ethical standards, and scientific rigor. Present your trial design to the class, explaining how it could potentially lead to breakthroughs in treatment.
Participate in a debate on the ethics of conducting clinical trials with pediatric patients. Discuss the balance between potential benefits and risks, using Edie’s case as a reference. Explore how ethical standards can be maintained while pursuing innovative treatments.
Join a workshop focused on fostering collaboration between medical professionals, researchers, and families. Brainstorm innovative solutions to improve pediatric healthcare, inspired by Edie’s story. Share your ideas on how collaboration can lead to breakthroughs in treatment and patient care.
Here’s a sanitized version of the provided YouTube transcript:
—
[Music] Edie was six months old when she was diagnosed with stage four neuroblastoma. She had tumors throughout her abdomen and up her spine, and she did not leave the hospital for four and a half months. [Music] After 12 rounds of chemotherapy and multiple surgeries, all of which occurred before she was two and a half, there were times when I questioned why this was happening to us and why our child had to endure such challenges. That’s when we were referred to the Children’s Hospital of Philadelphia and Dr. Mosei. Dr. Mosei is amazing; she feels like family. It’s often said that children need to be protected from research, but the reality is they need to be protected through research. One of Pfizer’s commitments is to develop breakthroughs that change patients’ lives, like Edie’s. Dr. Mosei has become part of our family, and I can’t imagine going through this without her. [Music]
My name is Yael Mosei, and I’m a professor of pediatrics at the Children’s Hospital of Philadelphia, with a faculty appointment at the Perelman School of Medicine at the University of Pennsylvania. I always knew I wanted to take care of children, but my perspective changed when my best friend developed leukemia. I didn’t realize that someone could be as young as 21 and have cancer. That experience motivated me to become not only a pediatrician but also a pediatric oncologist. Working in the lab allows me to take questions and formulate experiments. Since I didn’t have a science background, I started by asking what questions arise from the patients. We often say “bench to bedside,” but for me, it has always been “bedside to bench.” This is why participating in clinical trials is so important; it advances medical knowledge and brings science from the bench to the bedside. [Music]
In pediatric research, we need to prioritize many aspects of development. We must ensure that our work is ethically sound and of high quality. I had the privilege of meeting Edie when she was an ill-appearing baby. [Music] Dr. Mosei recommended a blood test to check for hereditary neuroblastoma. Upon taking her history, we discovered that she was born with a tumor on each adrenal gland, suggesting a genetic predisposition to developing neuroblastoma. Four days later, we received confirmation of hereditary neuroblastoma, and we were advised to return to Philadelphia immediately because a clinical trial for Edie’s specific type of neuroblastoma was closing. We identified mutations in an oncogene responsible for the inherited form of neuroblastoma. When there’s a mutation that abnormally activates the gene, it can lead to the development of a drug to turn it off.
That was a significant moment for us, and the trial for the drug opened just over a year after the discovery was published, which is unprecedented. After a transparent conversation, we recommended enrolling Edie in a phase one clinical trial for high-risk neuroblastoma. Pediatric research is challenging, and the Pediatric Center of Excellence is a group of pediatric experts dedicated to focusing on the needs of children in our work. Children are one of the most vulnerable populations in healthcare, so we are deeply invested in pediatric research to bring new medicines and vaccines to them, helping them live healthy lives.
What I remember most about Edie’s experience is when they brought in the first dose of the study medicine. She didn’t even have an appetite yet, but whenever she took it, she could choose something sweet to go with it, like whipped cream or ice cream, to mask the taste. As we developed our clinical trial programs, we began to think holistically about the child and family. We consider how the medicine will be administered to children, whether it needs to be a liquid, suspension, smaller tablet, or capsule. It’s important to involve pediatric subject matter experts from the beginning to ensure everything is well-coordinated.
Edie took the drug for a month, and as is typical in early-phase clinical trials, we repeated scans early on. During an appointment with Dr. Mosei, she mentioned she kept ordering more images because she couldn’t believe the results. Clinical research requires a genuine uncertainty about outcomes, and while not all trials yield positive responses like Edie’s, we began to see that not only did she have a remarkable initial response, but that response was durable, and she is doing exceptionally well.
I didn’t know about the possibility of participating in a clinical trial before this experience, but if I had to make the same decision again, I would do it without hesitation. It has changed my life; without the medicine, I might not be where I am today, and even if I were, I wouldn’t be as well as I am now. I can’t imagine going through this without Dr. Mosei. She is incredibly helpful, understanding, and encouraging, and I am forever grateful to her for her support. Clinical trials are the most scientifically rigorous way to impact the care of patients with cancer, and we need to collaborate with our industry colleagues and families to achieve the impact we desire.
—
This version maintains the essence of the original transcript while removing any sensitive or personal identifiers.
Cancer – A disease characterized by the uncontrolled division of abnormal cells in a part of the body. – The new treatment shows promise in targeting cancer cells without harming healthy tissue.
Research – The systematic investigation into and study of materials and sources in order to establish facts and reach new conclusions. – Recent research has led to significant advancements in understanding the genetic basis of certain diseases.
Trials – Clinical studies conducted to evaluate the effectiveness and safety of medical interventions in humans. – The clinical trials for the new vaccine are expected to conclude by the end of the year.
Genetics – The study of heredity and the variation of inherited characteristics. – Genetics plays a crucial role in determining an individual’s susceptibility to various diseases.
Pediatrics – The branch of medicine dealing with the development, care, and diseases of infants, children, and adolescents. – Advances in pediatrics have significantly improved the survival rates of premature infants.
Oncology – The branch of medicine that deals with the prevention, diagnosis, and treatment of cancer. – Oncology specialists are exploring new therapies to improve patient outcomes in metastatic cancer.
Mutations – Changes in the DNA sequence that can lead to alterations in gene function and may result in disease. – Certain mutations in the BRCA1 gene are known to increase the risk of breast and ovarian cancer.
Drugs – Substances used in the diagnosis, treatment, or prevention of diseases and medical conditions. – The development of new drugs has revolutionized the treatment of chronic illnesses like diabetes.
Healthcare – The organized provision of medical care to individuals or a community. – Access to quality healthcare is essential for maintaining public health and preventing disease outbreaks.
Children – Young human beings below the age of puberty or below the legal age of majority. – Vaccination programs have been instrumental in reducing the incidence of infectious diseases among children.
