I hope that you know that I am NOT trying to say that the results of this study are not valid or not hopeful. Not at all! It’s a great first step but Sue and I and others believe that this treatment won’t be available in 5 years as is being claimed. There is still too much yet to do.
Here are some considerations that we’ve found:
1. This is not a ‘cure’, the person’s vision was not restored to the before-AMD state and this will not help everyone. If a person’s photoreceptors have been damaged, inserting RPEs will not restore vision which is what the photoreceptors do. RPE cells ‘help’ the photoreceptors function. This is for those people who have some functional photoreceptors with RPEs that can be restored or rejuvenated.
2. The scientific article was received by the Nature Biotechnology publishers on November 6th, 2016 (yes, 2016!), accepted on February 28th 2018, and published online March 19th, 2018. This journal is peer-reviewed so it’s highly likely that the delay was because they had questions, asked the authors to explain what they did and how they did something.
3. There were 10 people enrolled and only 2 people got the treatment (stem cells). They can say they have 100% success but what about the others? I read that they said they would be treating another participant but no mention of timeline. Just this morning I saw a video that said that 8 more patients will undergo the procedure but there’s no timeline.
4. The Pfizer company was the sole funding source when the clinical trial started. When 2 of the 10 participants were enrolled, Pfizer suspended funding for ‘strategic commercial reasons’ not related to the trial. We are not sure where the funding will be coming for future research.
5. This is a really early phase of clinical trials. Phase I is the first of the phases. Its purpose is to make sure they’ve got the proper dose and to see if there is any ‘hint’ that it works. Phase II is the preliminary safety, final dose finding and looking at the results according to ‘endpoints’ they set at the start. Phase III is where they really prove if it worked and to make sure it is safe.
6. Before this can be used on the public, it will have to pass through regulators. Consider the phase & this aspect, it’s hard to believe that it could be available in 5 years.
7. They’ve only observed these 2 people for 12 months. They will be followed for 5 years. One thing that can happen with stem cells is that they can migrate to other locations and grow tumors or they can be rejected.. That’s why they give immunosuppresant drugs after surgery. It this case it was prednisone delivered directly to the eye. There’s no information about the long-term use of it. The woman who was treated is diabetic and the prednisone caused a change in her blood sugar (they were able to adjust it).
8. The purpose is to insert a ‘patch’ of RPEs generated from stem cells. It won’t replace all RPEs. It definitely won’t replace photoreceptors that give us sight.
9. There is no sham surgery done so you can’t 100% say that it was the stem cells that improved their vision. Sham surgery would be the researchers using the surgical technique exactly as the treatment technique but without the RPEs.
Yes, we will follow this and report anything we find to you.
Written 3/23/2018