The triad of cranial neuropathies, long tract signs, and ataxia are characteristic presenting signs; most patients have a short history of symptoms.
Recent investigations have focused on molecularly targeted therapies aimed at critical...Rapid, serial, and humane collection of cerebrospinal fluid (CSF) in nonhuman primates (NHP) is an essential element of numerous research studies and is currently accomplished via two different models. Corticosteroids are the mainstay of treatment for peritumor edema but are often associated with significant side effects. Methods: This Phase 2 study evaluated both safety a...BACKGROUND: Pre-clinical determination of disease-specific activity, effective dosing, safety, pharmacokinetics, and CNS delivery can optimize clinical trial designs.
An international working group consisting...For the past decade, it has been recognized that pediatric low-grade gliomas (LGGs) and glial-neuronal tumors carry distinct molecular alterations with resultant aberrant intracellular signaling in the Ras–mitogen-activated protein kinase pathway. Numerous clinical trials of new agents and novel therapeutic approaches have been performed over the course of seve...Survival of children with tumors of the central nervous system has improved over the past three decades with more than 70% of patients now expected to survive at least five years (1).
Pour la prévention, les propriétaires forestiers ont mis en place depuis plus de 70 ans une organisation unique en France et en Europe en créant et en s’impliquant au quotidien dans plus de 210 associations syndicales autorisées (ASA de DFCI… Her work focuses on rational, pharmacokinetic-based drug development for children with brain tumors, and she is a leading innovator in developing new means of drug delivery.
However, minimal CSF penetration has been demonstrated in pre-clinical models with single dose administrati...INTRODUCTION
Radiographic response is a primary endpoint in many investigational studies of brain tumors, but there is no standard method of tumor measurement for DIPG, significant inter-observer variability exists given the invas...Pediatric gliomas are a heterogeneous group of diseases, ranging from relatively benign pilocytic astrocytomas with >90% 5-year survival, to glioblastomas and diffuse intrinsic pontine gliomas with <20% 5-year survival. You may request a The CSF lat...Proceedings: AACR Annual Meeting 2014; April 5-9, 2014; San Diego, CA Despite recent insights into the biology of the most common brainstem glioma, diffuse intrinsic pontine glioma (DIPG), the standard treatment for brainstem gliomas has not changed significantly in nearly five decades. She was appointed Clinical Director of Pediatric Neuro-Oncology at Dana-Farber/Boston Children's in 2019 and was previously Senior Investigator and head of the Neuro-Oncology Section in the Pediatric Oncology branch of the National Cancer Institute where she worked for more than 25 years. We performed a chemical screen in patient-derived DIPG cultures along with RNA-seq analyses and integrated computational modeling to identify potentially effective therapeutic strategies.
katherinee_warren@dfci.harvard.edu. The low incidence and non-specific symptoms at presentation pose unique challenges for early diagnosis and disease-specific research. Our group and others have been evaluating peptide vaccin...BACKGROUND We found that patients with low 5hmC levels (3-year PFS, 73.7 ± 11.5%; 3year OS, 75.0 ± 11.1%) showed more favorable prognosis than those with high 5hmC levels (3-year PFS, 31.0 ± 9.7%, P = 0.002; 3-year OS, 36.7 ± 10.0%, P < 0.001; Fig. Chemotherapy plays an important role in the management of these tumors, particularly in low-grade gliomas, but many high-grade tum...Criteria for new drug approval include demonstration of efficacy. Physical activity (PA) targets CRT-damaged brain mechanisms and cognitive functions. It causes major logistic difficulty for existing adaptive phase I trial designs, which require the observance of toxicity early enough to apply dose escalation rules for new patients. This lethal disease affects all age groups, and the majority of patients have a poor outcome despite aggressive treatment. METHODS METHODS You may request a Dr. Warren is an internationally recognized expert in pediatric neuro-oncology. To test this hypothesis, we developed a unique nonhuman primate model using microd...Treatment of pediatric diffuse intrinsic pontine glioma (DIPG) remains challenging, and reliable biomarkers of response are lacking.