The Path to a Cure:

For years, families facing Coats’ Disease have shared a frustrating reality: there is still so much we don’t understand about what causes this vision-threatening condition or how to stop it.

Coats’ Disease is rare, unpredictable, and devastating. Yet research has been slowed by one major barrier: the lack of a reliable model that truly reflects how the disease develops in the body. Without that model, scientists are forced to study the condition in fragments, making it difficult to uncover its root causes or test promising treatments.

This is where a new study offers real hope.

The Research Opportunity

The Jack McGovern Coats’ Disease Foundation is supporting groundbreaking research led by Leo A. Kim, MD, PhD, Associate Professor of Ophthalmology at Harvard Medical School and vitreoretinal surgeon-scientist at Massachusetts Eye and Ear.

Dr. Kim’s research will investigate whether an existing IGFBP7 genetic mouse model can reproduce the key characteristics of Coats’ Disease and become the first validated animal model for the disease.

If successful, this model could give researchers an essential new tool to:

• Better understand the biological mechanisms behind Coats’ Disease

• Identify new therapeutic targets

• Study how the disease develops and progresses

• Test potential treatments in a controlled research environment

• Explore future approaches, including gene and RNA-based therapies

• Generate preliminary data needed to pursue larger federal and corporate research funding

This isn't simply another research study. It could create the foundation for the next generation of Coats’ Disease research.

What Could Success Look Like?

The goal of this research is not simply to create a mouse model. It is to create a research platform that can support discoveries for years to come.

If successful, the project is expected to result in:

A new Coats’ Disease mouse model: A model that reproduces key characteristics of the disease and can be used for future research.

New insights into disease biology: A better understanding of the mechanisms that cause and drive Coats’ Disease.

Potential therapeutic targets: Identification of biological pathways that could be targeted by future treatments.

A pathway for treatment development: The ability to evaluate promising therapies in a controlled research environment.

A foundation for future funding: Preliminary data that can help the research team pursue larger NIH and other federal or corporate grants.

The ultimate goal is clear: better treatments, better outcomes, and one day, a cure.