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Gene therapy, the new cure for blindness?

20
Oct
2017

Have we really found a cure for blindness?

Earlier this month, a gene therapy that treats an inherited form of blindness was approved unanimously by the U.S. Food and Drug Administered (FDA) panel.  If they get final approval in January, this will be a major breakthrough in gene therapy, as it will be the first time in history that a genetic treatment for a genetically inherited disease will become available to the U. S.

More importantly, it might offer a cure for blindness.

The gene therapy is being developed by a biotechnology group in Philadelphia, called Spark Therapeutics. The treatment product, is named Luxturna, and is used to treat those suffering from Leber Congenital Amaurosis (LCA).

What is LCA?

blurry vision

LCA is an inherited eye disorder causing blindness, which occurs as a result of a mutated gene known as RPE65.  This gene tells the body how to produce a protein that is absolutely essential for normal eyesight.   If it is faulty, it hinders our ability to see entirely.

Here are some of the symptoms of LCA:

  • Night blindness
  • Tunnel vision
  • Nystagmus – a condition which causes constant uncontrollable movement of the eyes

Whilst LCA is rare – affecting around one in 80,000 people  – it is the most common cause of inherited sight loss in young people.

What is gene therapy and how will it help?

gene therapyGene therapy looks at tackling illnesses, viruses and diseases head on, tackling the root cause directly.  This differs greatly from drugs, which are designed to get out any illnesses from the body currently, not permanently.  With that in mind, gene therapy focuses on replacing the missing or faulty DNA that is causing the illness in the first place.  Although the field is still in its early stages, treating the source of the problem offers some incredible exciting treatments for other genetically driven conditions that have had little or no treatment options, such as cystic fibrosis and Parkinson’s.

Whilst there is still a long way to go in the field of gene therapy, the unanimous approval from the FDA for Lacturna to treat LCA provides a beacon of hope for not only improvements to vision, but quite possible a cure!

 

What is the Luxturna treatment for this inherited eye disorder?

children's eye

Luxturna delivers a correct copy of the RPE65 gene to a patient’s retinal cells.   It essentially bears the correct version of this gene, carrying billions of modified, harmless viruses.  These allow the protein so pivotal to normal vision to be produced, enabling healthy vision.

The treatment is a one-off and involves the treatment Luxturna being injected into the retina.  Don’t worry though, you will be put to sleep before the operation takes place and the results so far seem to be worth it, as clinical trials have shown patients experience improvements within just a month after the procedure!

What are the limitations of the treatment?

This new breakthrough is not without its limitations, however, as it faces challenges in the lack of data on the duration of its effectiveness, alongside the cost factor too, which is expected to significantly impact how accessible it is.  With no guarantee of 20/20 vision after spending thousands of pounds/dollars, work will need to be done for Laxturna to reach those desperately in need of it.


Sources

https://theconversation.com/new-gene-therapy-gives-hope-to-people-born-with-inherited-eye-disorder-85501

https://www.ft.com/content/9c4fab50-b38f-11e7-a398-73d59db9e399

http://sparktx.com/

See more of our posts on eye health, laser eye surgery and vision, here

By: David

If you are interested in vision correction at Focus Clinic please call us on 0207 307 8250 and book a free consultation.

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