Gene Therapy and Blindness

by pharmacy on March 7, 2018

gene therapy blindnessWhen we think of blindness, we assume that it is someone who has no vision whatsoever. We tend to forget that blindness comes in different levels of intensity and how it affects their day to day lives. For people who have inherited a defective RP65 gene, it means that they live with color blindness or suffer from retinal degeneration. The FDA recently approved a gene therapy that could help those who have inherited the gene. Today we explore the therapy, the excitement behind it, the reality of the cost, and what changes we could see in the future.

A Simple Injection Could Fix It All

Spark Therapeutics has created an injection called Luxturna that is supposed to heal the defective gene. It simply sends a benign virus into the eye that delivers healthy copies of the gene into the retina for absorption. This type of gene therapy will not affect any offspring because genetics have already been coded in the reproductive system in the body. This treatment is not a cure but can change the effect of the diseases resulting from the defective gene. Many people have indicated that it made their eyesight better overall. Those diagnosed as children often end up with complete vision loss by adulthood. This kind of treatment can help them combat the vision loss early on while research continues.

The Cost Is Beyond What Most Can Afford

Luxturna is not yet available to the public, and the current biggest hurdle is the price tag that could be associated with the treatment. At $425,000 per eye, it can cause even the healthiest person to experience heart palpitations. Spark Therapeutics is currently working on a payment plan to make the treatment easier to access. There have been talks about installment plans and rebates for treatments that don’t react in a timely fashion. Until they can decide on a payment plan, upfront payment will be required. In the current state of healthcare, we will see an influx of fundraisers or people simply not opting for the much-needed treatment.

When Will It Be Available?

There is no date announced for release at this time. We just know that they are aiming to make the treatment available sometime in the first quarter of 2018. We assume that they are trying to develop a payment plan to coordinate with the release. It will be interesting to see what happens with its release and a potential payment option.

One Step in The Right Direction

Despite the hefty price tag and the slow release of the treatment, this is truly a great step in gene therapy. Until now, all gene therapy has been focused on cancer treatments. Now is a great time to be a pharmacist because we get to see treatments evolve and watch our patients heal in incredible ways. We imagine that 2018 is going to see some incredible breakthroughs!

 

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