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FDA panel considers gene therapy for blindness

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by Marilynn Marchhione, Associated Press, 10/11/2017:

"A girl saw her mother's face for the first time. A boy tore through the aisles of Target, marveling at toys he never knew existed. A teenage singer walked onto a stage and watched the stunned expressions of celebrity judges as he wowed 'America's Got Talent.'

Caroline, Cole, Christian. All had mere glimmers of vision and were destined to lose even that because of an inherited eye disease with no treatment.

Until now.

On Thursday, U.S. FDA advisers will consider whether to recommend approval of a gene therapy that improved vision for these three youths and some others with hereditary blindness.

It would be the first gene therapy in the U.S. for an inherited disease, and the first in which a corrective gene is given directly to a patient. Only one gene therapy is sold in the U.S. now, a cancer treatment approved in August that engineers patients' blood cells in the lab.

Children, parents, doctors and scientists will tell the FDA panel what it's like to lack and then gain one of our most primal senses.

The treatment, Luxturna, is made by Philadelphia-based Spark Therapeutics. It does not give 20-20 vision or work for everyone, but a company-funded study found it improved vision for nearly all of those given it and seemed safe.

The FDA must decide by Jan.12 whether to approve Luxturna."

 

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This is amazing. If only the fda or public cared about us. Or even our own community did. Unfortunately hsv is thought of as a non issue.

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I understand that there is a lot of red tape when it comes to the approval of health treatments and drugs, but why is it that you think that the FDA much less the public doesn’t care about us? 

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27 minutes ago, stronghands87 said:

I understand that there is a lot of red tape when it comes to the approval of health treatments and drugs, but why is it that you think that the FDA much less the public doesn’t care about us? 

Considering there hasn’t been an improved treatment or cure for it yet in almost 40-50 years and just slightly helpful daily pills it certainly seems like HSV is on the tail end of all other viral infections including the yearly flu now which has a universal vaccine coming for it soon.

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Is anyone curious as to why HSV is suddenly getting attention by researchers and labs? There must be a reason why crispr- cutting edge technology is even being considered for HSV, a harmless skin infection. 

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Probably because hsv is becoming too wide spread and people are realising it can cause damage also I'm going to assume hsv will evolve and possibly resist current antivirals ????

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Yep fixme1, I always wonder about whether one or both of the hsv2 strains could become resistant. We are already seeing this with bacterial infections like that strain of gonorrhea that is incurable because it is resistant to antibiotics. 

As we get closer to a cure for hiv, we could see more resistance issues with diseases, syphilis as well maybe on the sti front. 

So this is really is a serious reason to continue researching and developing hsv treatments and get closer to that elusive cure. 

 

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Don't the antivirals for herpes remove the building blocks that the virus uses to replicate? So how can it become resistant

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13 minutes ago, Franticheart said:

Don't the antivirals for herpes remove the building blocks that the virus uses to replicate? So how can it become resistant

I mean influenza can become resistant to antiviral drugs so...despite it being a dead non living thing it still has the capacity to become resistant I believe. It’s still mind blowing how something that isn’t considered living can create so many problems.

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The employees at the FDA are just regular people and I highly doubt they are intentionally blocking any cures or treatments with the intentions of maintaining any kind of disease prevelance within the US population.

 

Viruses are not considered living because of how we define life.

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7 hours ago, fixme1 said:

Probably because hsv is becoming too wide spread and people are realising it can cause damage also I'm going to assume hsv will evolve and possibly resist current antivirals ????

More in more people will have it oral hsv rates are as high as 60-80% genital is as high as 1 in 4 women after 30, maybe they dont cure it because so many people have it soon 1 third of the poplutaion could have genital herpes hopefully not but if theres no cure o vaccine or proper testing it could look that way.

 

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I just want to see people talk about HSV in the HERPES CURE RESEARCH forum.

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Not me, I would prefer it if we could continue to talk about successful medical advances with regards to other ailments and diseases in an attempt to justify our argument that the FDA is intentionally ignoring us. 

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then make a sub-category in the forum for all casual research references UNRELATED to HSV.

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10 hours ago, Franticheart said:

Don't the antivirals for herpes remove the building blocks that the virus uses to replicate? So how can it become resistant

yes but what if the tablets dont work for the certain strain of virus thats replicating ?

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13 hours ago, Franticheart said:

Don't the antivirals for herpes remove the building blocks that the virus uses to replicate? So how can it become resistant

I thought antivirals like Acyclovir and Valacyclovir act as literally throwing a monkey wrench in the molecular gears of HSV.

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There are resistant strains of HSV and the viruses can create recombinant strains meaning HSV1 and HSV2 are combining into one. I don't want to be an alarmist but I do think as they (scientists) learn more I am predicting the virus will get more attention and that has and will spur more research to try and treat it or at least prevent it.  HSV1 and 2 have been hard viruses to conquer mainly because they've been around for thousands of years and its preferred host are humans.  All these animal experiments can't replicate what the virus does in humans...that's one of the reasons why so many trials fail once the treatment or vaccine is introduced to humans. 

That being said, it's a double-edge sword with herpes.  Right now it doesn't kill anyone but  I do think they'll start finding out it is an accomplice in many conditions that do affect peoples quality of life.  

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