Home Again

Greetings! Home again and it seems as if we were never gone. That nearly two weeks just flew! The only way I can be sure we were gone is I am freezing! There is about a 40 degree difference in temperature between the Caribbean and home. If I ever disappear you are all welcome to come looking for me. You can start with Costa Rica.😎

Because a WiFi connection was something like 79 cents per minute from the ship, I have been very out of touch. I just did a little research and it appears the second half of the Lampalizumab study also failed in phase 3. That was the Chroma study. Spectri was the failure announced back in September. Or maybe it was the other way around. Anyway, it appears they both failed to meet the desired endpoint in the study. In an article in Healio.com Jeffrey Heier said they have collected a great deal of information they still have to analyze. They are hoping to be able to use this information to better understand geographic atrophy and also to develop new ways of fighting it.

Personally I am still perplexed as to why lampalizumab did so incredibly well in phase 2 trials but failed in phase 3. According to what I have read three-quarters of treatments that pass phase 2 pass phase 3 with no problems. Tis a mystery. Maybe the folks at Genetech can find the answer to that one.

I have already written a bit on BEST disease. That is the AMD disease that is not really AMD. I know at least one of our readers has BEST. October 25th News Medical published the results of a “disease in a dish” study done at Columbia and the University of Rochester. They took skin cells from people with BEST disease and regressed them to become pluripotent stem cells. They then took these stem cells and ‘nudged’ them into becoming retinal pigment epithelial cells.

As expected, these RPE cells growing in a dish (meaning they had no outside influences on them) did the same ‘bad things’ that happen in an eye with BEST disease. Once again this is a strong indication the problem is in the genetic coding.

So far this is old news, but the rest of it is not. The researchers found they could actually reverse early damage through gene therapy. They introduce healthy copies of the faulty gene using viral vectors. Once their codes were corrected, the cells started to recover and to perform like healthy RPEs.

Ready for prime time? Not hardly. This research is still happening in a petri dish. However, the rate at which this sort of research is progressing is startling and if I had to take a shot in the dark, I would say clinical trials may only be a few years away.

Hang in there and stay optimistic. We are making progress daily. Me? I have to make progress on vacation laundry!

written November 11th, 2017

Next: coming soon!

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