Choroid Plexus Tumor (CPT) therapies based on patient derived cell culture resources


Choroid plexus carcinoma

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CBTN Participants

CBTN Pre-clinical Models Used


Internal funding

About this


Choroid plexus tumors (CPT) are rare intraventricular neuroectodermal lethal neoplasms. Around 80% of choroid plexus carcinoma (CPC) arise in children, in whom they constitute 15–20% of CPT. Clinically, this group of tumors tends to cause hydrocephalus and increased intracranial pressure. Surgical resection is considered to be the most effective treatment for CPCs. The extent of surgical resection remains the most important factor in determining long-term survival in patients with CPC, but patients treated only with surgery have had a very poor outcome: disease progresses rapidly and patients often die within 1 year. Chemotherapy contributes to long-term survival, but it cannot prevent recurrence. Current data strongly support the use of combined chemoradiation but the total amount of necessary adjuvant treatment and the order in which the modalities are to be used are still controversial. Due to their rarity, reports on CPC most often focus on single cases or single-institution experiences with a limited number of patients. Further lack of cell lines has complicated in understanding this disease and design effective therapies.

Ask The


Ask the scientists

What are the goals of this project?

The goals of this project are to establish choroid plexus carcinoma (CPT) cell lines from frozen patient CPC tissue obtained from CBTN, to perform molecular and phenotypic characterization of these cultured cell lines and to conduct high throughput drug screening and additional experimental manipulation to design effective therapies for CPT.

What is the impact of this project?

Our proposed study will assess several molecular targeted therapeutics, which are approved by the Food and Drug Administration (FDA) for oncology, already in clinic for various malignancies. Repurposing and evaluating these molecules in related paired CPC-cells and pre-clinical CPC xenograft models will help in rapidly transitioning them to patients in clinic.

Why the CBTN request is important to this project?

CPCs carry a dismissal prognosis and there is limited clinical data available to describe treatment and outcome of CPTs. Further lack of patient cell lines and clinically relevant animal models have impeded in understanding this disease. Cell lines generated from CPC patient specimen can greatly aid in delineating this disease both at molecular and genetic level.

Specimen Data

The Children's Brain Tumor Network contributed to this project by providing a cell line.