At the young age of 2, Gavin Nielsen was diagnosed with a rare and aggressive brain cancer called diffuse intrinsic pontine glioma (DIPG). This type of cancer affects the part of the brain stem responsible for vital functions such as breathing and heart rate. Treatment options are limited, and the prognosis for children with this disease is typically less than a year.
Gavin’s parents, Nate and Ashlee, embarked on a journey to find the best possible treatment for their son. Despite many clinical trials having age restrictions that Gavin didn’t meet, he qualified for a trial led by Dr. Nicholas Vitanza at Seattle Children’s Hospital. Dr. Vitanza was investigating the use of CAR-T therapy, an immunotherapy that reprograms a patient’s own T cells to target and destroy cancer cells.
Gavin underwent radiation treatment followed by CAR-T therapy, where his T cells were modified to attack the specific target in his tumor. The promising results of the trial, which were published in Nature Medicine, showed an extended median survival time for patients like Gavin. Today, at 6 years old, Gavin has surpassed doctors’ initial expectations and continues to receive CAR-T infusions regularly.
His mother, Ashlee, considers Gavin’s progress a miracle, cherishing the extra time they’ve been given together. While Gavin experiences temporary side effects after infusions, such as headaches and nausea, he also enjoys moments of play and activity, showing resilience and determination in his battle against cancer.
Recently, a neuro-oncologist discussed the remarkable case of Gavin, a young boy who has defied the odds by surviving longer than expected with his brain tumor. Gavin’s mother expressed gratitude for the time they have had together, emphasizing the significance of Gavin starting kindergarten last fall.
The neuro-oncologist mentioned that while it is uncommon for patients to live beyond a certain point, there are outliers like Gavin who continue to thrive. He highlighted the need for further trials to confirm the effectiveness of CAR-T therapy, the treatment that Gavin and two other patients received.
Dr. Patrick Grogan, a neuro-oncologist at the Moffitt Cancer Center, cautiously expressed optimism about the potential efficacy of CAR-T therapy for treating brain tumors. He stressed the importance of conducting robust clinical trials to determine the therapy’s effectiveness compared to standard care.
While CAR-T therapy has shown promise in blood cancers, its application to solid tumors, which are more complex, is still experimental. Solid tumors present challenges due to their heterogeneous nature, making it difficult for immune cells to target them effectively.
Gavin continues to receive CAR-T cell infusions every few weeks, experiencing varying side effects from the treatment. The nonuniformity of solid tumors can lead to some cancer cells evading detection by CAR-T cells, posing a challenge to effective treatment.
Moreover, treating brain tumors presents additional hurdles due to the blood-brain barrier, which limits the delivery of therapies to the brain. In Gavin’s case, the modified cells were directly administered into the cerebrospinal fluid to target the cancerous area specifically.
Gavin’s participation in a clinical trial at Seattle Children’s Hospital aims to evaluate the safety and efficacy of CAR-T therapy for brain tumors. The ultimate goal of such trials is to determine the potential of new treatments in improving outcomes for patients.
Working with experimental therapies, especially Phase 1 trials, involves determining a safe and tolerable dose. While the hope is to see benefits, it is challenging to predict outcomes as it is a dynamic learning process,” stated Vitanza. In Vitanza’s trial, only one out of 21 patients experienced a severe side effect – bleeding in the tumor. “I always consider not just Gavin, but also the children who will follow him, those we have encountered during this journey who did not have this opportunity,” shared Nate Nielsen, Gavin’s father. Various early CAR-T trials for DIPG are ongoing in cancer centers nationwide. Despite lingering uncertainties, experts cautiously believe that this therapy could offer a much-needed treatment for DIPG. Children like Gavin play a pivotal role in advancing research. “It’s not just about Gavin, but about the children who will come after him, the ones we have connected with who did not have this chance. It is for them that we must persevere,” Nate emphasized.
