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AVOIDING UNNECESSARY TOXICITY AND IMPROVING SURVIVAL OF CHILDREN WITH LOW GRADE GLIOMAS by Simone Treiger Sredni, MD PhD

AVOIDING UNNECESSARY TOXICITY AND IMPROVING SURVIVAL OF CHILDREN WITH LOW GRADE GLIOMAS

Led by Simone Treiger Sredni, MD PhD at Ann & Robert Lurie Children’s Hospital

Pediatric Low Grade Gliomas (LGGs) are the most common form of childhood brain tumors. Children usually have good prognoses after complete surgical excision. However, as tumors grow within vital brain areas, complete surgical removal is not always possible. In patients with residual tumors, it is expected that tumor remnants will progress. For this reason, patients are also treated with radiation and/or chemotherapy, which can have devastating long-term side effects. These effects include significant drops in intelligence quotient (IQ) scores resulting in intellectual and academic shortfalls, a significantly increased risk of developing second malignant neoplasms, hearing loss, precocious puberty, short stature, cardiotoxicity, hemorrhagic cystitis, peripheral neuropathy, nephrotoxicity, ototoxicity and coagulopathies. It should be noted, however, that LGGs may stop growing or even regress over time. In fact, about 48% of LGGs present with spontaneous arrest or regression of the residual tumor. We are proposing to perform a study integrating high throughput molecular analyses with pathological, clinical and epidemiological data on children with LGG that had surgery at our institution. The proposed study takes advantage of state of the art technology applied to an invaluable resource: human frozen tumor samples with clinical follow-up. We are aiming to develop a molecular test that will potentially enable the identification, at the time of the diagnosis, of tumors that will be more likely to regress or remain stable after incomplete surgical excision. The identification of such patients will allow adequate intervention and will allow physicians to treat only the tumors that are more likely to relapse/progress with adjuvant therapy. This spares patients with tumors that will not relapse/progress from unnecessary toxicity, allowing these children to grow up and live a normal life. We believe that this project is aligned with the mission of The “Voices Against Brain Cancer” Foundation and we would deeply appreciate your consideration to support this significant study.

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Voices Against Brain Cancer is a not-for-profit public charity recognized by the IRS under 501 (c) (3)