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are not only more effective but also less toxic for patients grappling with recurrent or progressive ATRT.
Initial Success and Broader Implications
A standout feature of PNOC035 is its innovative, adaptive trial design. This design facilitates a dynamic treatment approach where patients can move seamlessly between various therapeutic arms as they become available. This method significantly expedites the evaluation of new therapies by allowing continuous treatment modifications based on emerging efficacy data, rather than the traditional approach of concluding one protocol before initiating another This structure is intended to reduce the time it takes to bring effective treatments to patients who need them urgently. If an effective agent or combination is identified, it will then be compared to previous treatment regimens by using data from PNOC030 as an external control cohort with the goal of avoiding the need for a randomized trial.
The trial is structured to open its first therapeutic arm in late 2024 This arm will test a novel combination therapy comprising two promising agents, paxalisib and gemcitabine These drugs have been identified for their potent synergistic effects, observed during rigorous preclinical testing In studies conducted in mouse models, this drug duo not only demonstrated significant potential to reduce tumor size but also enhanced each other's effectiveness, thereby improving overall survival rates. The initial focus of Arm 1 will be to assess the efficacy and safety of this combination therapy in patients with ATRT, aiming to establish a new standard of care that could surpass the results of current treatment options.
The strategic inclusion of these drugs follows a carefully orchestrated plan to target specific biological pathways implicated in ATRT, with the ultimate goal of interrupting the aggressive progression of the disease. This approach not only offers hope for extending the lives of patients but also focuses on enhancing the quality of life during treatment by minimizing the adverse effects often associated with more conventional chemotherapy regimens.
To realize these groundbreaking initiatives, substantial funding is required. The initial goal of $150,000 to launch the ATRT Registry was successfully met in late 2023 An additional $250,000 is needed by the end of 2025 to enroll previously diagnosed patients and maintain the registry. Team Beans has generously committed $150,000 towards this goal, leaving a shortfall of $100,000.
Regarding PNOC035, the Rally Foundation and The Truth 365 have awarded us $100,000 to get the study off the ground. With an additional $500,000, we can enroll 16 patients in this study over the next two years When you combine the goals of the ATRT Registry and PNOC035, the total funding need is $1,000,000, with $400,000 already raised, leaving a $600,000 gap
Call to Action
By supporting PNOC's ATRT efforts, donors can be part of a transformative journey to improve and potentially save the lives of children battling this devastating disease The investments in PNOC030 and PNOC035 not only promise better outcomes for today's patients but also pave the way for future advancements in pediatric cancer treatment.
Acknowledgments
We extend our heartfelt gratitude to our partners and donors, including Team Beans, the Rally Foundation, The Truth 365, Cure Kid’s Cancer Now!, Hope4ATRT, Dragon Master Foundation, Kinsley’s Unicorn Squad, the Fox Family and many others. Your commitment is crucial to our continued success and is deeply appreciated by the PNOC team and the families we serve
PNOC FOUNDATION
