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Results: 51

Elucidating the underlying mechanisms of radio resistance at diagnosis and ...

Backgound. Radiotherapy is still the mainstay of the treatment for DIPG. If the majority of children experience an improvement of their neurological condition following irradiation, this effect is not observed in all patients and is universally only transient. Determinants of the response to radiotherapy have yet to be defined. We ...

Targeting DIPG through combining a super-activator (MCB-613) of steroid receptor ...

The objective of this application is to demonstrate that combining MCB-613, a small molecule stimulator of the oncogenic steroid receptor co-activator (SRC), with ionizing radiation would synergistically kill tumor cells of diffuse intrinsic pontine glioma (DIPG) in vivo and significantly prolong survival times in patient tumor-derived orthotopic (intra-brain stem) xenograft (...

Defining the molecular mechanisms of DIPG development and progression to ...

  Diffuse Intrinsic Pontine Gliomas (DIPGs) are devastating pediatric brainstem tumors that lack effective treatment and are uniformly fatal. Patient studies have identified recurrent genetic lesions that drive the development of these tumors. Almost all DIPGs carry mutations in genes encoding either replication-dependent histone-H3 proteins (mostly HIST1H3B) or ...

An open label single arm phase I/II study of ...

We propose a phase I/limited efficacy clinical trial investigating repeated administration of MTX110, a soluble form of panobinostat, given via convection-enhanced delivery (CED) to pediatric patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG). We hypothesize this therapy will be safe and well-tolerated and will be efficacious and prolong ...

Credentialing an improved DIPG mouse model

DIPG is a rare type of childhood brain cancer that is currently incurable. One barrier for progress against DIPG is the development of predictive models, i.e. models in which observations regarding the evaluation of new therapies will accurately predict observations in clinical trials for children with DIPG.   ...