FDA Advisers Endorse Gene Therapy To Treat Form of Blindness (cbsnews.com)

An anonymous reader quotes a report from CBS News: A panel of U.S. health advisers has endorsed an experimental approach to treating inherited blindness, setting the stage for the likely approval of an innovative new genetic medicine. A panel of experts to the Food and Drug Administration voted unanimously in favor of Spark Therapeutics' injectable therapy, which aims to improve vision in patients with a rare mutation that gradually destroys normal vision. The vote amounts to a recommendation to approve the therapy. According to Spark Therapeutics' website, inherited retinal diseases are a group of rare blinding conditions caused by one of more than 220 genes. Some living with these diseases experience a gradual loss of vision, while others may be born without the ability to see or lose their vision in infancy or early childhood. Genetic testing is the only way to verify the exact gene mutation that is the underlying cause of the disease.

Comments

Sneaky

[rtb61]

Genetic testing only way to find out, well, to make every one safer, every one needs to be genetically tested. A little bit suspect one to go for in the first instance, the health insurance companies will love that test, I wonder how far they will stretch it. The best way for insurance companies to manipulate genetic test pilfering. Wait for you to get health insurance, sneakily grab your genetic test and find you have genetic predispositions, hope you forget to say anything about your genetic test record and provide health care coverage, as you are the best possible person to cover, as soon as you try to make a major claim, whamo. After decades of collecting insurance premiums, they say you have a pre-existing genetic condition that you didn't declare and tell you to fuck off and drop dead, hah hah.

Re: Sneaky

[Anonymous Coward]

You forgot the goat.

Re:Sneaky

[Humbubba]

I take your point that genetic testing can be used against us by insurance companies. This is a very powerful tool with awesome benefits and consequences. Genetic testing, medicine and genetic engineering has the potential to make a "better" human race. And therein lies the problem.

In Brave New World, Aldous Huxley foresees genetic engineering used to subordinate the lower classes by making them stupid and purging creative and destructive thought processes. Yuval Harari, in Homo Deus, predicts the rich and powerful will use genetic engineering to give their offspring immortality, happiness and even more power, and in the process create another kind of human. At some point these super humans will integrate with AI in a singularity that renders unnecessary current human affairs - and current humans.

Even if the bleak predictions of Huxley or Harari do not come true, once we tamper with our genetic code, there's no turning back. We lose the nature's evolutionary sorting in favor of designer genes. I'm not sure which best for the survival of the species.

This is HUGE

[virtualXTC]

Considering that what this drug is treating isn't a life-threatening condition, the vote to approve this drug shows just how confident regulators are that this form of direct gene modification is likely to prove to be safe in the long-term. Because of this, we can expect a flood of applications for new gene therapies in the next few years.

Re:This is HUGE

[Mashiki]

More then huge. This also means that unless something weird happens in the next few years, people who suffer from specific forms of red-green colour blindness will be able to be treated as well.

It could be a life saver

[Anonymous Coward]

For people suffering from retina detachment and other retina diseases, current form of eye-surgery is simply insufficient

Patients undergone retinal surgery know that one day they will still go blind, because their retina will still detach / degrade, and no doctor in the world can do anything about it

If this form of genetic treatment can 'repair' the retina (or somehow re-attach the retina back to where it should be) then it would be a life saver to many

Re: It could be a life saver

[Anonymous Coward]

I've had both retinas tear over the years, the right one detached losing a quarter of my field of vision.
Painful surgeries just to delay and prevent further damage, with one having nearly a year recovery time before I could go back to work.
I plan on asking my opthamologist about this when I see him next month. He has only been able to guarantee me three years of a self sufficient level of vision, and that was two years ago and before my cataracts started - I'm 32.

Re: It could be a life saver

[lhowaf]

Sorry to hear of your predicament. Here's hoping the science comes through for you. I guess the best you can do is to see all you can while it is assured, prepare for the dark if it comes and hope for the best from your ophthalmologist (and maybe get a second opinion). Hang in there.

Is MLB paying attention?

[FrankHaynes]

Because they should test this on Jerry Layne FIRST!

People who could see before only

[Anonymous Coward]

There are a number of theories about what would happen if you gave people who were born blind the ability to see. People who are born blind repurpose their visual cortex for things like interpreting braille. Direct stimulation fails to produce visual information, too.

Innovative new

[Anonymous Coward]

Every time I read "innovative new" I feel the urge to smack someone in the face.

Advisory Committee Materials & Presentations

[drunken_boxer777]

The FDA Advisory Committee materials and presentations are here, for those interested.

Note that this is for a very specific genetic cause of blindness, where a mutation in a gene for an enzyme results in effective loss of that specific enzyme. The drug is a retroviral vector encoding only the missing gene. Other causes of blindness, genetic or otherwise, wouldn't benefit from this treatment.

Pretty amazing, and a long time coming for gene therapy, since Jesse Gelsinger's death prompted a long close look at using adenoviral vectors for gene therapy.

At what price?

[Anonymous Coward]

Cost info?

Leber's congenital amaurosis (LCA).

[mpoulton]

The disease this is intended to treat is Leber's congenital amaurosis (LCA). It's buried in the article. Seems like the kind of important specific technical information that definitely belongs in a Slashdot summary.

Re:Advisory Committee Materials & Presentation

[Anonymous Cow Ward]

The drug is not a retrovirus. It uses adeno-associated virus (AAV) as the delivery platform; unlike a retrovirus, AAV does not integrate into the genome. Note also that despite having similar names, AAV and adenoviral vectors are very different.