b'REALIZING FUTURES USING INNOVATIVE PEDIATRIC PATIENT EXPERIENCE RESEARCH IN ACTION: TWO PATIENT SUCCESS STORIES PROGRAMMING TO ELEVATE CARE A 4-month-old boy presented with a large spleen and made his way to a local hospital in Virginia. The doctors there thought this wasOur continued investment in Hope for Henry juvenile myelomonocytic leukemia (JMML) but couldnt find a mutation in the boys leukemia cells. To aid their diagnosis, they called Dr. Elliotdemonstrates the power of behavioral economics in Stieglitz, a PCRF-funded researcher at the University of California,guiding kids to adhere to their medical plans so they can San Francisco Benioff Childrens Hospital and a JMML authority. Theheal and thrive. In July 2023, PCRF generously awarded family agreed to participate in a research study with Dr. Stieglitz andthe Hope for Henry Foundation with a $25,000 grant to send him the boys blood. Dr. Stieglitzs lab sequenced his leukemiasupport the growth and delivery of its groundbreaking by reading all of the letters of DNA. To their surprise, they foundpatient incentive program, Super Rewards, at Childrens a mutation in a gene called SH2B3. They had never seen a patientof Mississippi and Childrens Hospital of Orange County with only SH2B3 mutations before but they knew this was JMML.(CHOC).During the grant period, Hope for Henry helped 1,015 young patients at the two institutions to complete They recommended a new type of treatment called ruxolitinib that17 difficult but necessary procedures to stay on track with specifically counters the ill effects of the SH2B3 mutations with fewtheir healthcare goalsand to do so quickly, seamlessly, other side effects. The treatment worked like a charm. His spleenwith fewer healthcare risks and lower costs to their shrunk to a normal size and he was able to get a stem cell transplant athealthcare systems.Johns Hopkins. The boy is now in remission three years later. Dr Stieglitzs ability to make the diagnosis and select the right treatment was made possible because of a new diagnostic test he and his team have developed with funding from PCRF. This test uses DNA profiling to predict which patients will benefit from certain new treatments, helping to improve outcomes. The test also predicts which patients are most likely to avoid requiring intensive treatments like a stem cell transplant, thereby reducing side effects. At the age of 4, Gavin was diagnosed with acute lymphoblastic leukemia (ALL), an unexpected conclusion to endless colds and recurrentSURVIVOR SCHOLARSHIPS ear infections so severe that surgery was required. Following this surgical procedure, a pediatric oncologist told Gavins parents that 75 percentIn 2023, we proudly awarded over 100 scholarships to of the cells in his bone marrow were leukemia cells. Chemotherapy treatments began the next day. The prognosis was very good, but the treatmentsurvivors of pediatric cancers for their undergraduate and/ I forced myself to make a process included 6-8 months of chemotherapy, followed by three years of maintenance chemotherapy.Gavin relapsed at ageor post-graduate studies. These scholarships reflect our 9 and then again at 12. In the end, it was CAR T plus therapy treatment that enabled him to go into remission. It is nowcommitment to improving quality of life for those affecteddecision that changed my seven years that Gavin has been cancer-free and this happy teen is heading to college in the fall.by pediatric cancers and are one more way we help thesefuture reality. I chose notsurvivors realize happy, healthy productive, futures.to allow the diagnosisAs Kim Rowe, Gavins mom, explains:William Jetton is an example of the inspiring recipientsto define me. I chose to Traditional cancer treatment got Gavin into remission twice,we are able to support with this scholarship. define my diagnosis.but those remissions did not last. It was only through cutting- As a healthy cross-country runner, William never expected to receive a cancer diagnosis at 14. OnemonthI wanted to become the edge research and treatment that Gavin attained a lasting cure.after he did, William underwent a six-hour surgery at MDfastest runner on my team.He spent seven years battling three cancer occurrences. ThanksAnderson Cancer Center to remove 42 cancerous lymph nodes and his entire thyroid gland. The surgery left oneI wanted my lifeto breakthrough developments like the ones PCRF supports, helaryngeal nerve paralyzed and half of Williams airwayto be defined by success,is cancer-free and about to graduate from high school.permanently closed.not mediocrity.The Pediatric Cancer Research Foundation is pleased to have supported the early exploration of In spring 2020, William ran a minimum of eight milesI wanted to be known as an CAR T cell therapy that was helpful for Gavin. We continue to fund ongoing science examiningand biked 20 miles daily to make a disciplined effortovercomer, not a quitter.its use in treating high-grade gliomas and other brain tumors, understanding which genes aretowards his objectives. He is now studying accounting at the involved in a phenomenon called CAR T cell exhaustion and how to build better CAR T cells toUniversity of Florida, asserting the same focus and effort he~ William Jettonachieve more robust antitumor immune responses.applied to his athletic performance to his academic goals.13'