# 4-Demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN: A Phase II Clinical Trial in Adolescents/Young Adults (AYA)with CNS Malignancies

> **NIH NIH R44** · DEKK-TEC, INC. · 2020 · $451,382

## Abstract

Abstract – The goal of this Phase II clinical trial application will be to evaluate 4-demethyl-4-
cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) as anticancer therapy for adolescents and
young adults (AYA) with cancer involving the brain in a Phase II clinical trial.
DM-CHOC-PEN (Fig. 1) is a polychlorinated pyridine cholesteryl carbonate (Fig. 1) that is lipophilic,
electrically neural, is able to cross the blood brain barrier (BBB), localizes in CNS tumors with MOAs –
alkylation of DNA @ N7 – guanine and is not transported out of the brain via Pgp (p-glycoprotein). The
drug fulfills all the criteria to be a CNS active anticancer agent and recently received Orphan Drug
Designation approval as therapy for NSCLC involving the CNS.
The drug will complete a Phase I clinical trial in AYA subjects with advanced cancer 1st quarter – 2018
- IND 68,876. Objective responses, acceptable toxicities and no evidence of neurotoxicity have been
noted in the AYA Phase I trial involving subjects with cancers involving the CNS. Hepatic toxicity that
was observed in the adult Phase I trial has not been observed with the AYA subjects.
Novelty and Potential Clinical Advantage – Current reviews report that almost 700,000 people in the
US are living with brain or CNS tumors. Nearly 15% of these tumors involve the adolescent/young adult
(AYA) population, aged 15-39 years of age. It is predicted that 10,617 AYA individuals will be
diagnosed with brain or CNS tumors resulting in 434 deaths this year. For males and female individuals
<20 years of age, primary brain and central nervous system tumors are the most common causes of
death from cancer and in the 20-39 year age group the first cause of cancer-related deaths in males
and the fifth cause of cancer-related deaths in females. However, the incidence and histology of cancer
types vary according to subject age. Trends in CNS tumors have sharply increased since 1989 for AYA
individuals with a history of cancer, who appeared to have ‘beaten the odds’ to have a reoccurrence
from cancer, only to develop involvement in the CNS after years of remission.
The aims will include:
1) Initiation of a Phase II trial with DM-CHOC-PEN administered IV to AYA subjects with cancer
 involving the CNS and/or spinal nervous systems (SNS) and varication that the proposed doses
 are acceptable, monitor pharmacokinetics/pharmacodynamics and toxicities.
2) Monitor tumor responses per imaging/examinations using RECIST guidelines.
3) Electronically store/analyze clinical data for responses, toxicity, and PK relationships.
4) Continue to bank blood and tumor tissue from pre- and post-treated patients when possible tumor
 tissue assays for tumor - drug/metabolite content and bio-markers; for future studies.
5) Prepare a FDA Orphan Drug Designation (ODD) presentation/application for DM-CHOC-PEN as
 therapy for AYA subjects with malignancies involving the CNS/SNS.
6) Manage a platform to provide support information for AYA subjects with CNS/SNS mal...

## Key facts

- **NIH application ID:** 9882878
- **Project number:** 5R44CA203351-05
- **Recipient organization:** DEKK-TEC, INC.
- **Principal Investigator:** Lee ROY MORGAN
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $451,382
- **Award type:** 5
- **Project period:** 2016-02-01 → 2021-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9882878

## Citation

> US National Institutes of Health, RePORTER application 9882878, 4-Demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN: A Phase II Clinical Trial in Adolescents/Young Adults (AYA)with CNS Malignancies (5R44CA203351-05). Retrieved via AI Analytics 2026-05-28 from https://api.ai-analytics.org/grant/nih/9882878. Licensed CC0.

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