By Jillian Rose Lim
Yael Mossé, MD, remembers meeting Patricia “Pat” Brophy, beloved nurse practitioner at Children’s Hospital of Philadelphia, nearly 23 years ago. Dr. Mossé was completing her pediatric residency, while Brophy was already a seasoned nurse caring for the sickest of children with cancer. Intrigued by the prospect of specializing in neuroblastoma, a cancer of the nervous system, Dr. Mossé reached out to Brophy to learn more about the disease that left children with limited treatment options. What began as a mentorship soon blossomed into a friendship spanning two memorable decades.
Dr. Mossé, who is now an associate professor in the Cancer Center at CHOP, continues to carry the memory of Brophy, who died 11 years ago, both in her heart and through a new endowment named in Brophy’s honor. Last year, CHOP announced Dr. Mossé as the inaugural holder of the Patricia Brophy Endowed Chair in Pediatric Neuroblastoma Research, in the spirit of Brophy’s commitment to helping pediatric patients with small chances of survival.
“It’s hard for me to even articulate what this [endowment] means to me because the depth of it really is immeasurable,” Dr. Mossé said. “It’s wonderful to know that Pat’s memory is still very present. I know she would be really amazed by the progress we’ve made, but she was always one to want to do more for these kids. Her memory, her grit, and her passion for these kids is always with me. And now, to be able to carry her with me as I move forward in a new phase of my career is humbling.”
First introduced in June 2018, the endowment supports the discovery and development of innovative new tools that may improve outcomes for children with neuroblastoma. Funds donated to CHOP in honor of Alex Scott, one of Brophy and Dr. Mossé’s former patients and the founder of Alex’s Lemonade Stand Foundation, established the endowment.
“Pat had this enormous presence,” Dr. Mossé said. “And her thing was taking care specifically of children with neuroblastoma who had suffered a relapse – that was her focus, that defined who she was, the impact she wanted to have, and with this grew the very special relationship she and Alex developed.”
Mentor, Friend, and Inspiration
Dr. Mossé can trace Brophy’s warm and energetic influence back to her days as a fellow at CHOP. While she had always taken a general interest in oncology, Dr. Mossé began to take a closer look at neuroblastoma as a specialty because her very first patient, a young boy, had been diagnosed with the disease.
Meanwhile, at the time, Brophy and John Maris, MD, professor of Pediatrics at CHOP, were building a neuroblastoma clinical program that would provide options for children when curative treatments were no longer available. Brophy was the first person Dr. Mossé reached out to in order to learn more about the concept of “total care” for children with an ultimately fatal disease.
Talking to Brophy, who was beloved by so many patients and families – and who had so much spirit and experience – made a “colossal impression” on Dr. Mossé. Brophy taught her the importance of individualizing therapy to meet the unique needs of a patient and family. Alongside Dr. Maris, who was conducting work on the genetic architecture of neuroblastoma, Brophy became Dr. Mossé’s mentor, close colleague, and dear friend.
And despite neuroblastoma’s relatively low cure rate, there was a lot of room for hope along the way:
Dr. Mossé’s first patient, who had become very ill from his neuroblastoma therapy, managed to defy the odds and beat his cancer.
“With everything we know about the disease, he was supposed to die,” Dr. Mossé said. “We did end-of-life meetings with his parents when he was 3, and he was so ill that there was no way he was going to survive based on what we knew. But today, he has now successfully applied to MD and PhD programs.”
Novel Research Pathways for Neuroblastoma
In the last decade, Dr. Mossé has become a pioneering force in neuroblastoma research and treatment, leading the breakthrough discovery of the gene that causes the inherited form of neuroblastoma, the anaplastic lymphoma kinase (ALK) gene. Dr. Mossé and her team also showed these same mutations in the ALK gene are commonly found in neuroblastoma cells, even if not involved in disease initiation, and they provide an opportunity for development of targeted therapy approaches for a subset of children.
The Patricia Brophy Endowed Chair will allow Dr. Mossé to conduct cutting-edge research projects that are often considered high-risk but hold immensely powerful potential to improve the lives and outcomes for children with neuroblastoma. In particular, Dr. Mossé hopes to advance her team’s development of even more precise ways to diagnose and treat the disease by developing a neuroblastoma-specific blood test to allow for real-time decision-making and alterations in therapy. This more personalized approach will enable each child to get a particular treatment tailored to the exact genetics they inherited, the genetics of their particular tumor, and how their tumor’s genetics evolve over time.
“The cost to develop this as a research test is one thing, but the gap lies in getting this to the clinic,” Dr. Mossé said. “That bridge to the clinic is often underfunded and underappreciated, and so this is really where I think the Brophy Chair is going to allow us to have a big impact. We will be able to not just use this as a research test, but also accelerate this for newly diagnosed patients, patients in remission, and patients who suffer a relapse. Hopefully, we’ll be able to predict relapse before they’re symptomatic and be able to solve the problem more readily.”
Dr. Mossé knows the current project is something that would excite Brophy, in the very vein of the ALK gene discovery several years ago. In fact, Dr. Mossé remembers the day she told Brophy about being on the cusp of that breakthrough and her intuitive reaction.
“I remember about two weeks before [Pat] died, I was at her house spending what I knew were my last moments with her, and we had just discovered the mutations in the ALK gene,” Dr. Mossé said.
“At the time, what did I know? But it was like she knew. She asked the question: This is going to be big, right?”
And just as Brophy might have guessed, it was.
Learn more about Dr. Mossé’s research and the discovery of the ALK gene.