# Advancing Treatment for Pediatric Craniopharyngioma (ATPC); in vivo Tocilizumab Measurement

> **NIH NIH R03** · UNIVERSITY OF COLORADO DENVER · 2020 · $77,750

## Abstract

Project Summary
The principal investigator for this Small Research Grant is a pediatric neurosurgeon with specific interest in
improving the neurological and functional outcomes (e.g. quality of life, school performance, activities of daily
living) for children afflicted with Adamantinomatous Craniopharyngioma (ACP). This neurologically devastating
tumor, forces children and their families to face a lifetime of chronic and profound disability. As an early stage
investigator, the PI established North America's only consortium dedicated to the study of pediatric ACP,
Advancing Treatment for Pediatric Craniopharyngioma (ATPC). This award will determine whether a
systemically delivered monoclonal antibody reaches ACP tumor in vivo. As large molecules, these drugs would
not be expected to reach brain tumors, which lie behind the blood-brain barrier. However, work by Dr.
Hankinson's group and others indicates that ACP may be unique in this regard, making it accessible to these
agents. If this is true, a multitude of novel agents may be used with therapeutic intent. Cytokine expression in
ACP indicates a unique proinflammatory profile, raising the possibility that FDA approved medications targeting
IL-6/IL-6 receptor, could control ACP growth and/or invasion. We have considerable clinical experience with
the IL-6R inhibitor, Tocilizumab. Presently, there is no available assay to detect Tocilizumab in human tissue.
Through collaboration between the Hankinson and Reisdorph laboratories, we derived pilot data indicating that
mass spectrometric techniques will allow us to establish such an assay. This collaboration demonstrates that
Drs. Hankinson, Hoffman and Reisdorph work in an environment that is ideal for the completion of the work
detailed in Aim 1. In addition to Dr. Hankinson's appointment in the Skaggs School of Pharmacy, he and Dr.
Hoffman work closely in the clinical management and laboratory investigation of pediatric CNS tumors.
Children's Hospital Colorado has a considerable clinical research infrastructure and a record of successful
study recruitment, facilitating Aim 2. Our key personnel are experts in the methods required for this award, and
our infrastructure is exceptional. Tissue specimens for Aim 1 have already been obtained; we have years of
expertise in mass spectrometry; and we are one of the largest clinical pediatric neuro-oncology programs in the
country. Dr. Hankinson has built collaborative relationships with international leaders in ACP research, such as
Dr. Juan Pedro Martinez-Barbera, who has established the only animal models of ACP. If this project does not
identify Tocilizumab within ACP in vivo, we will proceed with animal studies regarding the efficacy of IL-6
blockade to determine whether these drugs offer adequate promise to merit study using more invasive delivery
methods, such as intrathecal or direct intracystic delivery. If the results of this work indicate that Tocilizumab
does have access to ACP tissue in vivo, t...

## Key facts

- **NIH application ID:** 9861235
- **Project number:** 5R03CA235200-02
- **Recipient organization:** UNIVERSITY OF COLORADO DENVER
- **Principal Investigator:** Todd C Hankinson
- **Activity code:** R03 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $77,750
- **Award type:** 5
- **Project period:** 2019-02-04 → 2021-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9861235

## Citation

> US National Institutes of Health, RePORTER application 9861235, Advancing Treatment for Pediatric Craniopharyngioma (ATPC); in vivo Tocilizumab Measurement (5R03CA235200-02). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9861235. Licensed CC0.

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