Friday, May 17, 2013

Stem Cells in Ophthalmology Update 25: ACT Patient in Dry AMD Trial Goes from 20/400 to 20/40!

The story started innocently enough. On Wednesday, May 15th, the journal Cell reported on a study that claimed biologists had finally created human stem cells by the same technique that produced Dolly the cloned sheep in 1996. They transplanted genetic material from an adult cell into an egg whose own DNA had been removed.

OK, an important story but what followed boggles the mind. Many science reporters wrote about the discovery which got picked up by several news sources. However, a sharp-eyed member of the Investor Stemcell Forum (iCell), a group originally started by investors in Advanced Cell Technology, noticed a quote at the end of Sharon Begley’s writeup. Sharon is a science reporter writing for Reuters. She had obtained quotes from several people working in the stem cell field, including at the very end of her story, the following quote from Dr. Robert Lanza, the Chief Scientific Officer of Advanced Cell Technology (ACT), “The most promising human study is ACT's. It is two years into clinical trials using stem cells derived from human embryos to treat two forms of blindness, including macular degeneration, with encouraging results. One patient's vision went from 20/400 to 20/40, said Lanza.

By Wednesday afternoon and into the evening, the iCell bulletin board went wild. There are more than 3000 members of this board and most are investors in ACT (or one of the other many stem cell companies discussed). Editor’s note: I am not a shareholder/investor, along with a few others, like Paul Knoepfler of Univ. of Cal. Davis, who are invited members because we write about the field. In the evening when I entered the board, there were several hundred comments all speculating whether the quote was accurate or, somehow, a mis-quote. This was the first time any of us had heard that a patient had achieved that level of correction.

Apparently, the SEC also took notice, because the next morning (Thursday, May 16th), the company was forced to acknowledge that the quote was accurate. In its statement, the company said, “Advanced Cell Technology today confirmed that the vision of a patient enrolled in a clinical investigation of the company's retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs) has improved from 20/400 to 20/40 following treatment. The improvement was first reported on May 15, 2013, in a news article published by Reuters.”

"We continue to be encouraged by the progress we see in our ongoing clinical investigations, though the results included in the article were confidential and not intended for publication at that time," commented Gary Rabin, chairman and CEO of ACT. "Our plan is still to publish additional results from the clinical investigations when we have a significant aggregation of data."

Now we knew that a patient in one of the company’s clinical trials (there are three of them – two for treating Stargardt’s Disease – one in the U.S. and the other in the UK; and one for the dry form of AMD in the U.S.) had achieved unparalleled improvement. So, which clinical trial?

I speculated that it was probably in the dry AMD trial, because in the SMD trials, the patients treated had quite damaged photoreceptors which I didn’t believe could recover to that degree, but confirmation was still needed.

That evening, I asked the iCell board if anyone had confirmed which clinical trial had produced the significant results – and I got a private message providing the answer. I was right, it was a patient in the dry AMD clinical trial.

So, what is the significance of this development. It is significant because it shows that, for the first time, a person suffering with the dry form of AMD (90% of all those with AMD) can obtain improved vision, going from legally blind (20/400), to normal vision (20/40), good enough to obtain a driver’s license in most states. Yes, this is just one patient, and early in this clinical trial, but hope prevails. If this much improvement can be obtained with one patient, and that patient with very poor vision, than think what can be obtained starting with people with much less a degree of poor vision, 20/100, for example, which is part 2a of the Phase II clinical trial.

I am hopeful that ACT is on the right track.


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