Articles, Blog

How a Virus Restored This Boy’s Sight

February 5, 2020


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.

48 Comments

  • Reply marax February 3, 2020 at 8:36 pm

    well technlogoy is improving

  • Reply LaMoustache February 3, 2020 at 8:37 pm

    forth

  • Reply Bloomberg February 3, 2020 at 8:37 pm

    Learn about more ground-breaking health tech in the Prognosis playlist: https://www.youtube.com/watch?v=kaFiwC1xh2Y&list=PLqq4LnWs3olVmUA_we9gKP6d3zfEwfVug

  • Reply The Modern Investor February 3, 2020 at 8:38 pm

    Watch the documentary called "Unnatural selection" On Netflix, we can already reverse aging and cure most diseases in humans….but… it costs money, lots of it. And not everyone will be able to afford perfect health.

  • Reply Harry Chen February 3, 2020 at 8:39 pm

    I feel sorry that this family is going to be broke

  • Reply Mr Da Vinci February 3, 2020 at 8:42 pm

    This is just awesome…πŸ‘πŸ‘

  • Reply Gregory Glass February 3, 2020 at 8:45 pm

    They been done this since 2009 and tested done on Federal prisons they always lying on their discoveries

  • Reply blackandcold February 3, 2020 at 9:04 pm

    This is science. No gods or prayers can help little fellows or other patients like him, only science and technology can. This is beautiful! For a better world for coming generations!

    PS: USA health System is a joke. Oh dear

  • Reply Nubnewb February 3, 2020 at 9:06 pm

    Well, where's the mutant super soldiers?

    Oh there you are 117!

  • Reply Prateek Bhuwania February 3, 2020 at 9:11 pm

    added some shares of CRISPR therapeutics to my portfolio

  • Reply B. Allen February 3, 2020 at 9:11 pm

    Think of the trillions wasted by the MIC and wars…..when that could go for life. DAMN the US Government

  • Reply Vincent February 3, 2020 at 9:14 pm

    Going blind is my biggest fear

  • Reply Oli Gilpin February 3, 2020 at 9:16 pm

    Beautiful!

  • Reply Z Xy February 3, 2020 at 9:17 pm

    Man Iβ€˜m glad I donβ€˜t have to put up wit the US health system, are they serious about you having to pay first before the insurance pays?

  • Reply garyjsimm February 3, 2020 at 9:19 pm

    The question is why is it that expensive to begin with?!

  • Reply Jamily din malinda February 3, 2020 at 9:48 pm

    They can change the world

  • Reply Fe King February 3, 2020 at 10:04 pm

    We were learning about these new treatments for retinitis pigmentosa in a lecture literally a couple of months ago, truly amazing to see it in action

  • Reply Nicholas Nelson February 3, 2020 at 10:10 pm

    I want to invest in the companies working on this now. It’s so cool

  • Reply Pyriphlegeton February 3, 2020 at 10:13 pm

    Watch "Unnatural Selection" in Netflix.
    It covers this exact technology and does it very well.

  • Reply Salty Box Gaming February 3, 2020 at 10:13 pm

    what's the company? wanna invest haha

  • Reply John Galois February 3, 2020 at 10:16 pm

    Meanwhile in good, OLD germany: "We now have officially banned any research on stemcells/gen-therapies."

  • Reply Dean February 3, 2020 at 11:38 pm

    My teen has stargardt. Great hope

  • Reply Mister006 February 3, 2020 at 11:38 pm

    Be prepared to pony up $850,000 for the one-time treatment with Luxturna.

  • Reply Black Label February 3, 2020 at 11:56 pm

    How many aborted Fetuses did they use?

  • Reply BLACKLIGHT February 4, 2020 at 12:43 am

    7:23 that is some nice editing and camera work

  • Reply NC Styles February 4, 2020 at 12:47 am

    I have bad eyesight & astigmatisms. -4.50 & -5.00 -3.25 Cylinder!

  • Reply Steven Kramer February 4, 2020 at 2:25 am

    Now know who has all the virus technology.

  • Reply R.Haque February 4, 2020 at 4:57 am

    such a happy moment πŸ™‚

  • Reply TheLittleAdventure Crew February 4, 2020 at 4:57 am

    Amazing

  • Reply κΉ€λ“ν™˜ February 4, 2020 at 6:28 am

    Wonderful !

  • Reply Shakib Ali February 4, 2020 at 6:30 am

    Thanks to scientist for their undeniable contribution for better life of others!

  • Reply Nairobi Street TV February 4, 2020 at 7:38 am

    she still insists it's a miracle instead of technological breakthrough. What's wrong with humans

  • Reply Samuel Escobar February 4, 2020 at 7:44 am

    ❀️❀️❀️❀️❀️

  • Reply Inesophet February 4, 2020 at 8:00 am

    Honestly. I love the new direction Bloomberg has been taking. A straight forward positive approach to the Future. Whoever is in charge actually deserves a Raise.

  • Reply nerdy1701 February 4, 2020 at 9:21 am

    Gattaca here we come!

  • Reply Nishchal Siddharth Pandey February 4, 2020 at 9:45 am

    That's why I watch Bloomberg! Positive news makes my day.

  • Reply Classified February 4, 2020 at 10:25 am

    Can we use gene Therapy to cure Blood synthesis problem too?

  • Reply Espooi VANTaa February 4, 2020 at 10:30 am

    Amazing

  • Reply iamdmc February 4, 2020 at 10:58 am

    It's not a miracle – it's SCIENCE. When it comes time to vote – VOTE FOR SCIENCE. Vote for the candidate that embraces science and doesn't deny or reject it.

  • Reply artended February 4, 2020 at 2:25 pm

    I feel like cali is another dimension flying to hell with the best medicine and biggest tech on board.

  • Reply vansh chhabra February 4, 2020 at 7:02 pm

    Me and my brother have this too
    I’m so happy for maverick
    He truly lives up to his name

  • Reply o_o February 4, 2020 at 8:00 pm

    i was feeling relly fuzzy and warm throughout and then saw that its 1/2 a Mil for treatment on one eye..
    imagine those family that had that hope but no money..
    health care should be human right.

  • Reply Alberto Rodriguez February 4, 2020 at 9:49 pm

    πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ‘πŸ»πŸ™πŸ»πŸ™πŸ»πŸ™πŸ»πŸ™πŸ»πŸ™πŸ»πŸŽ–

  • Reply T W February 4, 2020 at 11:01 pm

    *for the rich

  • Reply Phan Phan February 5, 2020 at 3:41 am

    this is resident evil done right.

  • Reply Aud ience February 5, 2020 at 4:40 am

    demonic virus healing his sorry eyes around this time… demon bless.. maybe..

  • Reply TigerMonthlyHits February 5, 2020 at 6:30 am

    Y’all done changed the title enough already

  • Reply Allah cezanΔ±zΔ± verecek February 5, 2020 at 11:18 am

    Tüm dünyanın canı cehenneme Allah sadece gerçek insanları korusun

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