A Miracle Tech Could Reverse Blindness

A Miracle Tech Could Reverse Blindness


Defects in retinal genes cause a devastating from of childhood blindness. Gene therapy is really,
I think at the forefront of treating retinal degenerations. There are now over 20 clinical trials addressing various diseases in ways that we didn’t really think possible before. Come on. Having to make a decision
on his behalf has been a real struggle. This treatment could change our lives, this could stop a truly terrible disease. As we tackle the problem of childhood blindness, this is
going to be a real breakthrough in our ability to change
the course of the disease in a way we never thought possible. Maverick is my firstborn
child, he has been the most amazing child to raise from the day that he was born. I didn’t have a lot of
experience with babies before I had him, but I just noticed that he had a blank stare. Checked his tracking vision
and it was non-existent. We went through all the genetic testing and it came back positive for
Leber’s congenital amaurosis with a specific genetic marker of RPE65. The cones and the rods in
the eyes don’t allow for light perception so he is legally blind with specifically low-light blindness. He also doesn’t have peripheral vision or vision below his waste. It’s almost tunnel vision. Ahh, I need a parachute now, help me! Up here is my room, and these are my pillows. This is my eye patch. It has to be the right
lighting for him to see. Once the sun goes down,
his world is very dark. We take him inside, turn on all the lights until it’s time for bed. Maverick’s condition is degenerative. There is no timeframe
of when the degeneration will take place, but
it is a well-known fact that he will be completely
blind in later years. It took his thoughts
quite some time to subside into sense of fire and
sulfur, a torched landscape. In December of 2017, the
FDA approved gene therapy for Maverick’s specific
condition and so we went and talked to Dr. Nagiel
shortly after it was approved. Oh, mine goes right behind you. Maverick was six years
old when I first met him, and this family, for a
long time had struggled with getting a diagnosis,
and then we’re very excited finally to hear that
there was a treatment. Luxturna is a gene replacement therapy whereby we’re delivering
a normal health copy of the RPE65 gene to patients
who have two defective copies. This medication is a virus
which has essentially been modified so its only job is to deliver the genetic material. We deliver this virus
with a very fine needle underneath the retina and
then we have a healthy copy of a gene that’s been missing in the cells that normally make it. Unfortunately with this disease, the cells that absorb the
light, the photo receptors, are constantly degenerating
and there gets to be a point where there simply aren’t
enough cells to function even if we were to deliver that gene back. So it makes a lot of sense to treat earlier pediatric patients,
young patients when they have those cells there and
just need the normal copy of the gene to make the
protein functioning again. So treatment early in children
is really the way to go to achieve the best results. Here at Children’s Hospital Los Angeles, we’ve treated 17 patients, so 34 eyes. We have data that the
treatment effect is stable without any loss in function. Five years ago, there were no
treatments for this disease and for the bulk of retinal
generations, these patients, if they were lucky, they got a diagnosis with genetic testing but
beyond that, there wasn’t much that we could do aside from get the most out of the
vision that they have. Since the FDA approval of
Luxturna, that’s really opened the door to not just
treating this relatively rare retinal degeneration, but essentially any retinal degeneration. This has been probably the
most difficult decision that I’ve ever had to
make in my entire life. It makes me very nervous
because it is so new, if it doesn’t go well, it’s my fault because I chose to do this to my child. I honestly feel like I hit the jackpot when I had Maverick. I don’t know any other seven-year-old kid that could handle everything
that he has been through with such appreciation for life, you know? He is willing to do or try anything. All right, here we go. After today, our life could
potentially change forever. I love you.
I love you, buddy. He might go back home
and for the first time have fun with his friends by
the pool as it’s getting dark, ride his bike at dusk,
go trick-or-treating, these are the sort of
improvements that really change a child’s life
and the motor confidence that comes with ambulating and interacting with the environment in a way
that they couldn’t before, I think really has life-long consequences. It takes seven days for the corrected DNA to be absorbed so at seven days, he could potentially see again. It just comes in very small
doses and it could take up to four months, they said. Maverick had his two
surgeries, and we are home. The first day that I really noticed that something was different
was riding in the car, and he said mom, I’m just looking outside and I’m seeing what is outside the window, like I’ve never been able
to see through the windows and it seemed so small but
it was really life changing for him to be able to
look out of a window. Okay, listen to me.
One more game of tag. Maverick, sit.
Ready? One more game. One, two, three, four,
five, I’m coming out! I see you guys! In the past seven years,
we don’t even come in the backyard at night. This is a true miracle. You can not stop me! Okay, I’ve got Maverick. Willow, come here, get him. He has a whole new level of independence and he is ready to conquer
anything that is given to him.

100 comments on “A Miracle Tech Could Reverse Blindness

  1. Human of Augustana Post author

    Hope the kid doesn't reproduce like his mother did. We don't need these kind of genes in our future society

    Reply
  2. alphavortex1 Post author

    we have a cure! but YOU can't have it…
    unless you give us everything you have + will ever have… then maybe.

    Reply
  3. cristoff3 Post author

    Such a brave, incredible kid.
    Nobody should have to go through life without sight. Please like this video to help it trend and get Luxturna the attention it deserves

    Reply
  4. Garrett Szilard Post author

    If Health, Science and Spirituality dominated the world…World could be a cleaner place for sure.

    Reply
  5. Mr Anderson Post author

    Bloomberg, cures blindness so everyone can see how stupid "gun control" really is.

    A real wtf moment.

    Reply
  6. bluestone best Post author

    the fun part begin at 3:22. but i m thinking…. hey where are the problem in implanting a modified disease that modify your genes into your eyes? can we use it to modify other genomes elsewhere on the body? can we build hulk? or a spartan of halo?

    Reply
  7. The Aequalitatem Post author

    Amazing technology! But more amazing is the love and courage this family has. Your son is a beautiful and wonderful kid! God bless him.

    Reply
  8. TheFreshSpam Post author

    What a shame it cost them a house to get it to him. Its this type of money that should be given to these people instead of seeing the same amount of cash be dropped by a bomb in unneeded situations.

    Just imagine if the US replaced the bombs they dropped by helping every fellow american

    Reply
  9. C Post author

    I thought the Thumbnail was of a robot eye. I was excited that maybe robotic eyes are on the market now. But gene therapy is cool too.

    Reply
  10. Mauricio Graham Post author

    Great job to the developers of the therapy and I definitely hope we can solve blindness all together one day.

    Reply
  11. Gregory Boyle Post author

    This reflects the very simple concept of enjoying the outdoors at night and how it's taken for granted. But this child appreciates it as a new part of life. I really look forward to seeing future genetic manipulation to save our future from these horrible diseases.

    Reply
  12. PizzaDelivery4u Post author

    Um guys… Listen very closely… I'm surprised 92,194 people did not spotted this out yet. They forgot to edit this out. 8:14 The neighbors. 😂🤣

    Reply
  13. Alejandro Flores-Lopez Post author

    great medical advancements are great but it seems the costs are too high. Does it really take nearly 1/2 a million dollars per eye for this treatment? Id like to see why the price is so ridiculously high

    Reply
  14. Владислав Post author

    Great video, world is not such a bad place to live, then we thought. And it’s getting better.

    Reply
  15. Blue Azola Post author

    Whenever i see
    CNN
    FOX
    BLOOMBERG

    i used to just ignore it & move on coz these are reptilian/elite medias use for CONTROLLING & BRAINWASHING the ppl

    Reply
  16. World Peace Post author

    If getting a child in the us cost over 10 grands (what’s already ridiculous)…I don’t want to know what this is going to cost.

    Reply
  17. Penname Bear Post author

    I wouldn't let a needle in my arm and I'd typically retaliate or flee now imagine how much my fear takes over when i realize that a needle is gonna stab my eye.

    Reply
  18. camramaster Post author

    Right on time… Humanity may bypass the first major existential barrier after all.
    Don't give up humanity- some of us are counting on you!

    Reply
  19. Matok Post author

    So only a million dollars to fix your kids eyes.

    Yeah, most families won't be getting anywhere near this cure. Shame.

    Reply
  20. Lord of Nothing Post author

    well, get yourself some bernie sanders for president, and maybe every child that needs it can get this treatment. because of a proper public healthcare. 90% of people, couldn't afford this. if they vote sanders, they will.

    Reply
  21. Luke Smith Post author

    Great story, did you catch the end with how much it costs. Very depressing and a bit disturbing how much that actually costs, I think they call that greed.

    Reply
  22. Frank Nakamo Post author

    There is no defects in vision, or retinal degeneration, is all directly cause by injury in the eyes by human cells who teleport, they all know this, but they just acting like heroes.

    Reply
  23. Javier Aguirre Post author

    How can insurance companies able to cover those costs? Specially in this case where is something that comes since born? I mean I understand crash accidents where everybody who is not in an accident ends up paying for the ones that are but in this case? Can someone explain it to me?

    Reply
  24. Keith Camilleri Post author

    im happy for them, but please the title is clickbait, when you say tech and put a mechanical eye as the thumbnail, you get the wrong idea -_-

    Reply
  25. Normie Peasant Post author

    "We're developing new ways to take your money. We want everyone to have access to our treatment, as long as they can afford 2-3 Ferraris."

    Reply
  26. Johnoè Post author

    Could this used to treat infections diseases? Maybe a harmless virus that’s sole purpose is to replace DNA that isn’t encoded

    Reply
  27. jason bourne Post author

    May Allah bless n help all Docs to research more n more n make everything possible to resolve the disease, disabilities etc….Ameen

    Reply
  28. Matthew Dunstone Post author

    This “miracle” is cutting edge medical science born of hard work. What a great medical outcome for the lad in this story.

    Reply
  29. Zergoug Faycal Post author

    As someone who lives with glaucoma since birth Isee this as a breakthrough fir a lit of people who suffer from eye diseases although I consider myself as one of the few lucky people who have a stable vision but I really wish that I can read or drive like everyone, I hope this will be perfected and made available for everyine.

    Reply
  30. PlanetPresident Post author

    I'm screwed, Poor and going blind 68yrs , Nobody gives a damn about me! when I die I'm taking all my invention with me, you didn't care about me, you don't deserve my gifts

    Reply
  31. Barry Quinn Post author

    It’s criminal that society would have the knowledge to fix a child’s blindness, but then cost it in a way to ensure most children never get treatment. What are our priorities???

    Reply
  32. Peter Roub Post author

    Trump's latest Job Approval rating. Reuters (Nationwide Poll:) 2/18 – 2/19 43% approve 55% Disapprove 55 % disapprove NET -12. There just doesn't seem to be any path forward for trump.

    Reply

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