# Translational Studies toward Cell Therapy for Treatment of Primary Open Angle Glaucoma

> **NIH VA I01** · IOWA CITY VA MEDICAL CENTER · 2024 · —

## Abstract

Decreased visual ability in veterans quickly leads to functional impairment and with it, reduced quality of
life. In the year 2016 eye care was the third busiest clinical service in the VA Health Care system and 490,926
veterans receiving care at Veterans Affairs medical centers were given a glaucoma-related diagnosis. The
most common type of the disease is primary open angle glaucoma (POAG), and the major risk factor for
POAG is high intraocular pressure (IOP). Medical treatment is life-long and poor patient compliance presents a
significant obstacle to successful care, motivating the need for novel safe, effective and sustained IOP control
therapies. This project is designed with the long-term goal of regenerating the natural ability of the eye to
regulate IOP.
 The trabecular meshwork (TM) is a key regulator of IOP and has been shown to undergo significant
changes in POAG, including a loss of cells. This, in turn, causes failure of the tissue to carry out its functions
and a subsequent rise in IOP. This motivates the use of cell therapy to restore TM function as a potential long-
term treatment for POAG.
 Our studies have demonstrated that induced pluripotent stem cells (iPSC) can be differentiated into a cell
type that resembles primary TM cells morphologically, compositionally, and functionally. Transplantation of
these cells, designated iPSC-TM, into the eyes of mouse models of glaucoma causes re-celluarization of the
TM, decreased IOP, and prevention of vision loss. This approach has also been shown to reliably increase TM
cellularity in ex-vivo organ culture of human eyes obtained from older donors. These findings are extremely
encouraging and suggest that restoration of TM function through iPSC-TM transplantation is possible.
However, crucial aspects of iPSC-TM cell therapy require further development before translation into clinical
practice can be considered. These include: (1) Increased targeting efficiency of transplanted material to the
TM, which will reduce the number of injected cells and decrease off-target effects; (2) evaluation of the
effectiveness and safety of allotransplants to reduce, which could reduce costs and increase quality of
transplanted cells; and (3) direct demonstration that iPSC-TM cell therapy is effective in human eyes with
POAG maintained in organ culture. Successful completion of our studies would provide a very strong rationale
for future clinical trials.
 The proposed studies are highly innovative since they investigate a completely novel approach to
permanently repair, rather than treat, glaucomatous damage in veterans. These studies will have an important
impact since this unique and novel approach has the potential to profoundly alter clinical practice. Lasting
functional restoration will reduce clinic visits and costs for the VA health care system, provide physicians with a
new treatment option, and will help veterans through better vision and increased quality of life.

## Key facts

- **NIH application ID:** 10995780
- **Project number:** 1I01RX005008-01A1
- **Recipient organization:** IOWA CITY VA MEDICAL CENTER
- **Principal Investigator:** MARKUS H. KUEHN
- **Activity code:** I01 (R01, R21, SBIR, etc.)
- **Funding institute:** VA
- **Fiscal year:** 2024
- **Award amount:** —
- **Award type:** 1
- **Project period:** 2024-09-01 → 2028-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10995780

## Citation

> US National Institutes of Health, RePORTER application 10995780, Translational Studies toward Cell Therapy for Treatment of Primary Open Angle Glaucoma (1I01RX005008-01A1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10995780. Licensed CC0.

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