This is old news at this point, but I just have not found the time to write about it. Below is the article published in the New England Journal of Medicine regarding the recent gene therapy trial for LCA, a severe, early-onset form of RP.
http://content.nejm.org/cgi/content/full/NEJMoa0802268
There have been press releases issued to major news outlets. You can see the media coverage at the FFB website:
http://www.blindness.org/
It is worth the time to read the academic article. While it is difficult for a layman to understand all of the jargon, it gives a realistic picture of the actual results achieved.
Yes, it is very exciting. But, it is the result of, at least in the study published above, one person's improvement in one aspect of vision, contrast sensitivity. Apparently there is the study above as well as another one using a different set of three adults. This is the only published peer reviewed article I have been able to locate in PubMed (thanks for the share, G.). And, as all of the scientific types are quick to remind us RPers, one person's experience does not usually mean much and we should not invest too much time in being hopeful. Unless it is gene therapy, apparently.
I suppose I must be missing something. It certainly would not be the first time. Maybe the other study, which I cannot locate, had better results.
This is the first gene therapy trial, to my knowledge, done for a condition which is not fatal. I have to admit, I am floored the FDA approved gene therapy for a non-lethal condition. The exciting thing about this trial, based on the little I know about gene therapy, is no one had "adverse events", like dying, from the viral vector used.
A vector is a virus used to carry the normal copy of the gene to the target gene. The tricky part is using a virus that is not harmful to humans. In other (non RP related) gene therapy trials in the past, people have died from immune reactions resulting from the virus. This may be worth the risk to someone dying of a fatal condition. There is also the risk of the replacement gene not just going to the target area and inserting itself in other cells, leading to cancers.
However, by some miracle, these scientists actually were able to get this trial approved in this country for a disease that is not killing people. So, in the scientific community, that must make them rock stars. I am not talking some bubble gum boy band either. More like Keith Richards, able to defy the odds and astonish medical professionals time and time again.
Aging rock star jokes aside, It seems like there are multiple standards at work. Again, I am sure I am missing something. But, take our UCL acetazolamide case study. That is a case of one person having improvements in contrast vision. However, there were no press releases, no buzz or continued funding. Granted, her gains were not nearly as dramatic as the gains of the subject in this study.
But, the results obtained by one young person in the study are very encouraging to me. The increases in his ability to detect contrast are undeniable and unlikely to be explained away. The fact that the dogs who have received the same procedure, eight years ago, have maintained their gains is even more encouraging. And, this is gene therapy's first barbecue for RP in humans. This was just to test safety, and that anyone had any benefit at the most conservative dosing level is encouraging.
My personal belief is gene therapy may be able to help me one day maintain the results of a stem cell procedure. I think it holds an enormous amount of potential for young people who are newly diagnosed and identified. Hopefully the incredibly brave young people who participated in this study will have more improvement and stable general health.
The "subjects" are courageous, pioneering and inspiring. With all of the things I have tried, gene therapy would still terrify me, regardless of who was doing it. Kudos to the researchers as well. Firstly, for getting results. Secondly, for managing to get a trial with this amount of inherent risk done in this country. It has no doubt been a long, laborious road and I look forward to more good news.
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natalie, it does seem odd that these scientists were able to get FDA approval to conduct their gene therapy studies. Twp things come to mind regarding this event and its timing. First, consider all the horrible things were have been hearing in the news about the FDA not doings its job, which has resulted in tainted foods and poisonous products entering our homes and various other dangerous products coming at us from all directions. How much should we be trusting the FDA these days, how do we know that they are really looking after our safety and well-being? Of course, this leads to the issue of credibility too. However, here is my second thought on the issue of these studies getting FDA approval... if you are familiar with Ray Kurzweil, and I am sure you are, then you know what he says about gene therapy. He was on the Glenn Beck show recently, and Mr. Beck introduced him as "the guy" that everyone goes to regarding the future of technology and that nearly all of his predictions have come true over the past 30 or so years regarding technology. Makes one wonder if Mr. Kurzweil perhaps had anything to do with the FDA giving these studies the green light so quickly after the not so distant fatal results. Something to think about...
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