Last month, we met with Professors Jeffrey Goldberg and Joyce Liao of Stanford University. The road to treatment for Autosomal Dominant Optic Atrophy (ADOA) has entered an ambitious new phase. In this extensive meeting, we discussed the important steps being taken at Stanford to combat ADOA. This work, made possible in part by the dedicated financial support from the Cure ADOA Foundation, focuses on actively developing the means to stop and ultimately reverse vision loss.
Current efforts are focused on a two-pillar strategy, namely:
- the preservation of the remaining vision;
- developing technology to restore lost vision.
Maintain remaining visibility
The first pillar focuses on protecting the optic nerve from the "cellular stress" that often leads to vision loss. Joyce Liao's laboratory is currently testing promising compounds that act as a protective shield for the vital cells of the eye, specifically through the use of nicotinamide (a form of vitamin B3).
By improving the optic nerve's environment, researchers hope to create a stable foundation that prevents further damage, allowing patients to maintain their current vision for as long as possible. They have also developed new methods to measure the health of the optic nerve long before actual vision loss occurs.
Technology to restore lost vision
The second, and perhaps most transformative, pillar concerns research into regeneration. While the medical world has long struggled with how to replace damaged nerve cells, Goldberg's laboratory has achieved a major milestone in directing the growth of new cells.
The challenge was never simply "making" new cells, but ensuring they knew where to go. The team has developed a groundbreaking method that helps these cells grow purposefully and in the right direction—toward the brain. This "guidance system" is a crucial piece of the puzzle that was previously missing and brings the goal of functionally restoring vision significantly closer.
Hopeful future for ADOA treatment
We will likely see further advances in both protective therapies and optic nerve regeneration in the coming years. While the scientific process requires time and thorough testing, current developments suggest we can be quite hopeful about achieving major milestones in slowing and curing ADOA in the next five to ten years; and that the road to treatment will thus be significantly shorter.
