Editorial Comment - Patient Care and the Technical Dilemma of ACTC's 20-100 Patients

I will be on my 3rd Cup of PeaBerry Coffee, thus I have to write LOL!

And yes... the unintended consequence of ACTC's 20-100 Patients will be coming up
very fast before the FDA.

What if all of the handful of early stage patients (whose decades of productive lives are before the
hands of the world's leading retinal surgeons) all have reasonable/exceptional visual acuity improvements? But more importantly, all their diseases are arrested and the deterioration and death of the PhotoReceptor Cells were reversed?

What should be the next step? for the Doctors, ACTC, and the FDA?

But! Do not forget the millions and millions of Patients whose PhotoReceptors are dying everyday!

Here is the Dilemma:

1. Many many Doctors, and Bureaucrats will say: We need at least 400 more patients in the Trials to prove efficacy! Forget about the labels! We need more Patients to prove that RPEs work from an Statistical Standpoint! These are the Drug Paradigm based Science People.

2. Some of the Doctors will argue... that RPEs are not Drugs! These are smart cells and these are Biologics! We have to start putting together a NEW REGENERATIVE BIOLOGICS APPROVAL PROCESS FOR THESE NEW CELLS!

3. My point is: Are the Drug Based Statistical Models being used by Trial Statisticians even relevant or applicable to the RPEs??? Case in Point:
a. The Eye is a reasonably Rejection Free Zone.
b. The purity of ACTC's RPEs is no longer in question because of their Proprietary Assay.
c. There is a blood barrier in the back of the eye.
d. The RPEs do not migrate out of the back of the Eye.
e. No tumors.
f. The Multiple Complex Drug interaction between the locus of the application vs. the other biological systems of the body as used in a Drug Model DO NOT APPLY IN THIS CASE OF THE RPEs! SO WHY ARE WE APPLYING THE SAME STATISTICAL MODEL AND ASSUMPTIONS USED IN DRUGS TO THE RPEs??????
g. And finally, Has any Doctors anywhere around this big wide world of ours found any human eye, or human cells... that are designed differently... with different cells... with different bio-chemical mechanics? CAN ANYONE IN THE FDA... POINT TO A DIFFERENT EYE DESIGN BETWEEN BLACKS, WHITES, BROWNS, REDS? SO TELL ME... IF ALL HUMAN EYES ARE AT LEAST DESIGNED REASONABLY OR STATISTICALLY THE SAME... WHAT IS THE POINT OF TESTING THIS DARN RPEs ON 400 OR 500 OR 1000 MORE PATIENTS!!!!!!!!!!!!!???????

I am not ranting... I am just asking questions?

Sooooooooooooo... if ACTC's handful of 20-100 Patients have arrested diseases and have good visual acuity improvements...

Are there any reasons, any of you Doctors should allow the PhotoReceptors of you Patients to die on a daily basis because the FDA do not know what to do???

OR... Should ACTC and the Doctors propose a Temporary Commercial Approval and Concurrent Phase 3/4 Trials until a set of 2000 Patients are reached?

Best,

Cuenca