# Development of a novel pan-cancer CAR-T product against AML and Glioblastoma

> **NIH NIH R43** · KURE. AI, INC. · 2024 · $400,000

## Abstract

Despite therapeutic advances outcomes are still poor for patients with most relapsed/refractory
malignancies. Recently CAR-T cell therapies have demonstrated significant potential to markedly
improve the outcomes of patients with relapsed refractory B cell and plasma cell malignancies.
Unfortunately to date, CAR-T cells have not demonstrated similar efficacy for patients with solid
tumors or other blood cancers. A major challenge for the development of CAR-T cells for these
malignancies is the paucity of targets on cancer cells that do not exhibit cell surface expression
on normal cells leading to significant toxicities that can correlate with efficacy. We recently
identified the non-classical MHC molecule, MR1, is a promising target for CAR-T cells. MR1 is
typically found in the cytoplasm in the vast majority of cells and was recently reported to traffic to
the cell surface in a wide variety of tumor cells when bound to cancer cell specific metabolites.
We validated the cell surface specific expression of MR1 protein in a wide range of malignancies
including pancreatic, prostate, brain, melanoma, breast, lung and AML. Further we developed a
CAR-T cell product that utilizes a non-traditional CAR design to enable it to be efficacious against
the low levels of cell MR1 found on the surface of tumor cells. Pilot mouse tumor studies
demonstrate the potential safety and efficacy of this approach. In this proposal we will focus on
the development of MR1 CAR-T cells for a blood cancer, AML, and a solid tumor, glioblastoma,
that both urgently need new therapeutic options. Here we will 1) Assess the cell surface
expression of MR1 in primary patient AML and glioblastoma samples and normal controls 2)
Perform mouse efficacy studies using AML and glioblastoma models and 3) Assess the safety of
MR1 CAR-T cells using mouse and in vitro models. It is hoped that this work will lead to an MR1
CAR-T cell product that can improve the outcomes of patients with cancer.

## Key facts

- **NIH application ID:** 11007100
- **Project number:** 1R43CA295241-01
- **Recipient organization:** KURE. AI, INC.
- **Principal Investigator:** David Soler
- **Activity code:** R43 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $400,000
- **Award type:** 1
- **Project period:** 2024-09-16 → 2026-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11007100

## Citation

> US National Institutes of Health, RePORTER application 11007100, Development of a novel pan-cancer CAR-T product against AML and Glioblastoma (1R43CA295241-01). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/11007100. Licensed CC0.

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