Why did ACTC choose the RPE Program?
I thought this e-mail from CEO Caldwell might help some here understand why the program was chosen to hopefully be their first clinical trial with FDA. This was posted here and on other boards in June of 2008. I did confirm/verify the authenticity with the CEO.
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"We have stated publicly the following reasons why we chose the eye as our first disease indication. This is within the context of a specific objective of trying to identify “the lowest hanging fruit” to get into and through the clinical Trial process. It was assumed that whatever embryonic stem cell derived therapy that would be chosen would be proven efficacious and safe pre-clinically.
Our senior scientist, Irina Klimanskaya, identified the Retinal Pigment Epithelium (RPE) cell which is attributed as a factor in almost 200 different types of retinal disease. This cell is pigmented thus distinguishable by the naked eye versus all other cells in a petrie dish. This assists the development process in the identification, extraction and ultimately the purity process for the Development of pure RPE cells. Purity, the fact that the therapeutic derived cells are fully and terminally differentiated, from their original embryonic state, is a huge factor for the FDA. Pure cells address tumor issues. The eye, as you have indicated below, has often been used as a first mover in new therapies because of its unique properties within the body. It is an immune privilege site. This means it doesn’t naturally reject foreign matter. This will either totally eliminate or greatly reduce the necessity for immune-suppressant drugs that are required for the introduction of foreign cells in most other parts of the body. It also allows for a business model of “cells in a vial” which is similar to a traditional vaccine type therapy. The eye is an enclosed tiny organ that requires a very small amount of cells to effectuate a viable therapeutic response. Our adult heart disease therapy requires 600 million cells to be injected into the damaged heart muscle versus 1-1.250 million RPE cells in the vitreous cavity of the eye. This allows us to better track the migration of the cells (if any should occur which has yet to present itself in any of our animal studies). The FDA in evaluating safety is concerned about tumors AND migration of the cells. Where do they go and what do they do when they get there. A small dosage, in an enclosed area helps address this FDA safety concern. Finally, the mechanism of application is a needle. Unlike most appliance applicators, it does NOT have to be approved by the FDA. A needle is recognized by the FDA as a standard mechanism of application and even more importantly every eye surgeon utilizes a needle in surgical procedures dealing with the eye. Therefore, you don’t have to teach the “old dog new tricks” which is a huge barrier when normally introducing new therapies utilizing a delivery device.
We believe that we have a therapy that has multiple applications (retinal disease includes dry AMD, Retinitis Pigmentosa (RP) and Stargardt disease. The latter two may qualify as Orphan indications which could qualify for special treatment through the clinical process by the FDA. This was a large factor weighing in favor of selecting this therapeutic option.
I hope this helps in your understanding of our choice."
