Tag Archives: trials

Incredible News About Stargardt’s Cure

So, I was posting a summary for my book on the NaNoWriMo Facebook page, thinking that it sounded over the top… stem cell-based cures for horrible incurable diseases within 2-10 years, that kind of thing… well….

There’s no link for this, because it is insider info from shareholders who went to Ocata’s quarterly meeting in Boston, which was not open to the public. (There is a LOT about Ocata in the book– they’re the biotech company that just changed their name from Advanced Cell Technology.) Ocata is the company developing the first cure (or even treatment!) for age-related macular degeneration, a disease that slowly causes blindness for 30 million people in the U.S. and Europe alone. The interim published results for the clinical trials have looked great, but as far as when this treatment might actually come to market… it’s everybody’s guess.

But in the quarterly meeting… several attendees have all said the same thing. The drug already has orphan treatment approval for a genetic disease caused Stargardt’s, which has exactly the same effects and eventually causes blindness. But it attacks much younger people, including children as young as six. (Yep, six years old.) AND… the CEO of Ocata said that the drug will be available in the U.K. in 2018. We don’t have absolute confirmation of this yet. But it’s apparently what was officially said at that meeting.

One of the lines of thought on the forums is that the date might be earlier for AMD approval in the U.S, even quite a bit earlier if the drug is fast tracked here… we just don’t know. But this is INCREDIBLE!!!~ There has never ever been any kind of treatment for Stargardt’s before, and now there will be, and it will be STEM CELL BASED. As of now, that will be the first drug approved anywhere in the WORLD that is embryonic stem cell based.

YES! IT’S THE START OF A NEW ERA IN MEDICINE!!! And it will be in the book!
(sorry about the capslock…

The Harvard Type I Diabetes stem cell thing isn’t going so well…

Here we go again! Yet ANOTHER piece of stem cell research that was insanely overhyped until somebody discovered a fatal flaw. The short version: there’s been incredible excitement this past year because a new hormone was supposedly created through stem cell work, and it would create an incredibly efficient treatment for Type I diabetes. Here’s a good quote from the Harvard press release:

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Yamanaka on the new iPSC trials for wet AMD in Japan. Part 1.

Head on over to Paul Knoepfler’s blog and check out hisYamanaka Interview on Clinical Use of Pluripotent Stem Cells.

I don’t know. I’ll be posting more about this tomorrow, but… suffice it to say right now that I wish I could get more excited about this new study with iPSC’s. I think they’re rushing wayyyyyy too fast to use these techniques on a human subject, especially when you look at all the pre-clinical problems and questions. And there were A LOT.

 

And I think we could have all lived without this quote:

 

At the same time, the ethical issues that hESC possess mean that as iPSC technology improves, hESC will be less needed. Still, iPSC is a new technology, and its safety and efficacy still needs to be confirmed.

 

Um, hello, EARTH TO DR. YAMANAKA. There ARE no “ethical issues.” Seriously, you don’t know about Robert Lanza’s blastomere technique, which doesn’t destroy the embryo?. And yep, no kidding that iPSC’s safety needs to be confirmed, much less their efficacy. Maybe we shouldn’t be blithely testing these cells on human beings right now.

 

That’s enough of a rant for today, but there will be more tomorrow!