Gene therapy transforms eyesight of 12 born with rare defect

[SoCalDem]

http://www.latimes.com/news/nationworld/nation/la-sci-gene-therapy25-2009oct25,0,2334183.story


By Thomas H. Maugh II

October 25, 2009


Pennsylvania researchers using gene therapy have made significant improvements in vision in 12 patients with a rare inherited visual defect, a finding that suggests it may be possible to produce similar improvements in a much larger number of patients with retinitis pigmentosa and macular degeneration. The team last year reported success with three adult patients, an achievement that was hailed as a major accomplishment for gene therapy. They have now treated an additional nine patients, including five children, and find that the best results are achieved in the youngest patients, whose defective retinal cells have not had time to die off.

The youngest patient, 9-year-old Corey Haas, was considered legally blind before the treatment began. He was confined largely to his house and driveway when playing, had immense difficulties in navigating an obstacle course and required special enlarging equipment for books and help in the classroom. Today, after a single injection of a gene-therapy product in one eye, he rides his bike around the neighborhood, needs no assistance in the classroom, navigates the obstacle course quickly and has even played his first game of softball.

The results are "astounding," said Stephen Rose, chief scientific officer of Foundation Fighting Blindness, which supported the work but was not involved directly. "The big take-home message from this is that every individual in the group had improvement . . . and there were no safety issues at all." The study "holds great promise for the future" and "is appealing because of its simplicity," wrote researchers from the Nijmegen Medical Center in the Netherlands in an editorial accompanying the report, which was published online Saturday by the journal Lancet.

snip...

[FirstLight]

1. wow - is it only being used in the retina?

my daughter has ONH (optic nerve hypoplasia) - wonder how close we actually are to generating nerve tissue?

[mopinko]

7. imho, the pentagon

wants that technology. so, i am betting that it is not far away.

[glitch]

2. This is incredible!

My sister is legally blind with macular degeneration, this would be a miracle for her.
Thanks for posting!

[SoCalDem]

5. I hope she talks to her doctor.. maybe she could get the treatment

I know how wonderful it is to have sight restored..

after a failed surgery, they were able to restore sight to my right eye. It;s not great, but is a whole lot better than before.. I have uveitis.

[glitch]

13. She's horrible about doctors, as am I. On the other hand, I am a terrific nag about other people

seeing their doctors so I think I can make something happen. :evilgrin:

Thanks again for posting. I am sorry about the uveitis, that's very painful isn't it? :hug:

[SoCalDem]

14. Felt like I was stabbed in the eye..

My opthalmologist likened it to a stroke or heart attack of the eye..and silly me.. I was resigned to having no vision in one eye, because I still had one good eye..until..

yup got it in both eyes now...but he's pretty on top of my care.. so far I am still seeing:)

[glitch]

16. Owwww! How the heck do you get that, guess I'll hit the googles.

I am glad you are still seeing. You sound like you have a good doc. This gene therapy sounds so promising! Good luck to you. :)

[SoCalDem]

18. I have non-specific uveitis, but there are some things that can cause it

thankfully none of those reasons fit my case:)

[goclark]

3. Yes! Yes! Yes! K and R

This gives hope to so many people.

[Me.]

4. Music To My Eyes

[Hekate]

6. "Astounding" indeed! I once had a friend with RT, a lovely bright young woman...

This therapy is 30 years too late for her, but what a miraculous thing for all those to come.

Hekate

[Statistical]

8. I believe we are at the Comodore 64 stage in genetic research.

Even today's breakthroughs will seem mundane compared to the capabilities in next 20 years.
Compare a computer today to one 20 years old as a analogy.

We are at an inflection point where genetic, stem cells, and bio-drugs open up whole new avenues of medicine.

I read an article about how nanoparticles (metallic) are used as transfer agents for chemo therapy. Using magnetic fields the chemo can be directed to the tumor site which means rest of the body gets a smaller dose = less side effects.

[SoCalDem]

9. If only people can afford it.

or there are enough altruistic doctors to donate their services.

[Statistical]

10. Hopefully just as computing power has fallen by a factor of 1000x we will se a similar

fall in cost of treatment.

If we don't then likely finances will be the single largest determining factor to life expectancy. Given that both genetics & money can be inherited within a couple generations we will see a permanent over and under class form.

[Barack_America]

12. It's the next dominant industry.

And here's hoping we maintain our edge.

[Posteritatis]

17. re: C64 stage - that's a *fantastic* way of wording it! (nt)

[Barack_America]

11. We still lead the world in the biomedical sciences.

And yet the NIH only gets $30 billion a year. Imagine how many jobs and cures could be created if we invested more!

[KamaAina]

15. His first game of softball?

People who are totally blind play softball, with an adaptive ball that beeps.