FDA Advisers Endorse Gene Therapy To Treat Form of Blindness

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.

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 a

Re:

[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

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:

[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.

Re:

[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.

Is MLB paying attention?

[FrankHaynes]

Because they should test this on Jerry Layne FIRST!

Advisory Committee Materials & Presentations

[drunken_boxer777]

The FDA Advisory Committee materials and presentations are here [fda.gov], 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 [wikipedia.org] death prompted a long close look at using adenoviral vectors for gene therapy.

Re:

[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.

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.