Diffuse Intrinsic Pontine Glioma
DIPG
Diffuse intrinsic pontine glioma (DIPG), is a lethal CNS cancer with a dismal prognosis. While some cases occur in young adults, most occur in children, and the median overall survival is less than one year from diagnosis.
Patients with DIPG can present with a wide variety of neurological symptoms reflective of the anatomic localization of the lesion. Thus, in over 50% of patients, cranial nerve palsies (facial asymmetry and diplopia), long tract signs (hyperreflexia, upgoing Babinski), and cerebellar signs (ataxia, dysmetria) are present.
The current mainstay of therapy is palliative external beam radiotherapy, and there are no long-term survivors. While clinical trials are underway using a range of different therapeutic approaches including small molecular inhibitors, immunotherapy, and convection-enhanced delivery, no treatment has demonstrated a benefit to overall or progression-free survival in this tumor. The extremely poor prognosis and lack of efficacious, targeted therapy demand a novel approach to its treatment
Koru has been able to comprehensively characterize the druggable cell-intrinsic vulnerabilities in DIPG and identify promising therapeutic agents. On the basis of this dataset, we intend to perform several further studies using the drug candidates and Convection enhanced drug delivery system (CED)
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