# Experimental Therapeutics

> **NIH NIH P30** · ALBERT EINSTEIN COLLEGE OF MEDICINE · 2020 · $26,996

## Abstract

PROJECT SUMMARY - EXPERIMENTAL THERAPEUTICS PROGRAM
Experimental Therapeutics (ET) is the programmatic home for the design, preclinical development, and early
clinical evaluation of a broad spectrum of new antineoplastic modalities developed within all programs, and for
the conduct of all clinical therapeutic trials at AECC. There are two major areas of research in the drug-
development arena: (1) drug design and preclinical development of novel agents for the treatment of cancer
based upon transition-state principles, chemical library screening, rational agent design, and novel proteins; (2)
new immunotherapeutics based on understanding the mechanisms by which tumors escape immune detection
and elimination; and the preclinical development of agents that activate the immune system. Therapeutics
developments are directed to novel targets identified in this and other programs. This research has resulted in
a spectrum of IPs, the formation of several companies, and the recent approval of a drug for the treatment of
T-cell leukemia/lymphomas in Japan. A number of transition-state inhibitors focused on novel targets are in the
pipeline, including several that impact on epigenetic processes. Based upon an understanding of the structure
and function of BAX, small molecule activators of BAX, developed at AECC, induce cancer cell apoptosis and
are active in vitro and in mouse xenografts, while BAX inhibitors prevent the cardiotoxicity of cytotoxic and
targeted agents. A novel family of immune checkpoint co-inhibitory molecules has been identified, their
expression in human cancers documented, and robust development of inhibitory antibodies is underway at
AECC, and in collaboration with pharma. A new technology in development consists of a specific antigenic
recognition peptide together with stimulatory T-cell signals that expand or differentiate clinically-relevant T-cell
clones but have minimal impact on the general T-cell repertoire, thus ensuring selectivity and avoidance of the
side effects of current immune checkpoint inhibitors. There are robust capabilities in protein production, small
molecule and fragment screening, x-ray crystallography, NMR and mass spectrometry, with bioinformatics and
systems and computational biology support based in this program and in the AECC shared resources led by
members of this program. Drug development efforts are supported by a developing Chemical Synthesis shared
resource. Disease-focused working groups interact with laboratory scientists to foster translational studies.
There is an active Phase I/II program for early clinical evaluation of leads in this and other programs into
investigator-initiated studies by this program along with an established CAR-T program one of only two in the
region. AECC members play important roles in ECOG-ACRIN, NRG, GOG, COG, and the AMC. There are 52
program members from 21 departments. Current NCI funding is 2.2M (dc); total peer-reviewed funding is 5.2M
(dc). There have been 981 pu...

## Key facts

- **NIH application ID:** 9998888
- **Project number:** 5P30CA013330-48
- **Recipient organization:** ALBERT EINSTEIN COLLEGE OF MEDICINE
- **Principal Investigator:** Vern L. Schramm
- **Activity code:** P30 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $26,996
- **Award type:** 5
- **Project period:** 1997-06-01 → 2022-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9998888

## Citation

> US National Institutes of Health, RePORTER application 9998888, Experimental Therapeutics (5P30CA013330-48). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/9998888. Licensed CC0.

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