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- W4387049063 abstract "<p class=first dir=auto id=d6757347e129>Diffuse midline glioma (DMG) remain lethal tumors with virtually no long-term survivors and a reported median survival of 11 months. The majority of patients are less than 10 years of age at time of diagnosis. Due to their delicate location and diffuse appearance, resection is not feasible. Standard treatment includes biopsy followed by focal radiation therapy and clinical trial enrollment given the lack of standard of care options. <p dir=auto id=d6757347e131>Unfortunately, translation of promising preclinical results into the clinic has been not successful. Although some genetic markers have been identified that correlate with prognosis, only few approaches make use of precision medicine tools to consult individual, targeted therapies. Key challenges to improve outcome for these hard-to-treat tumors are (1) need for international collaborations given disease rarity, (2) lack of industry interest, (3) access to agents that cross the blood brain barrier, and (4) limitations to conduct trials that allow interrogation of on treatment tumor tissue to better understand impact of new therapies. <p dir=auto id=d6757347e133>In 2019 the DMG/DIPG center Zurich at the University Children’s Hospital Zurich was established with the goal to change the therapeutic approach to DMGs through close collaboration between the clinic and laboratory. Since then, we have established a large international DMG focused network aimed at developing new therapies. One outcome of this network is the implementation of a first DMG platform trial that enrolls patients internationally. The trial treatment is based on combination therapy options offered at different disease stages that have been validated across multiple laboratories. To address key limitations of prior trials we built in a myriad of correlative studies to develop potential biomarkers of response as well as resistance. <p dir=auto id=d6757347e135>Given the dismal prognosis, physician and scientists are caught between the expectations of families, the interests of researchers, and regulatory requirements. This results in a variety of challenges on the ethical, scientific and legal side. It is undeniable that for this constellation of a rare disease in a pediatric population, we need to rethink previous approaches and adopt new strategies to make a difference." @default.
- W4387049063 created "2023-09-27" @default.
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- W4387049063 date "2023-09-26" @default.
- W4387049063 modified "2023-09-27" @default.
- W4387049063 title "Clinical Research for Pediatric Patients Suffering from a Diffuse Midline Glioma: Limits, Challenges and Perspectives" @default.
- W4387049063 doi "https://doi.org/10.58647/rexpo.23012" @default.
- W4387049063 hasPublicationYear "2023" @default.
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