This drug costs more than 1m euros, learn why

Leber's disease.

It's known as Leber's Congenital Aurosis. This disease affects the retina and occurs 140,000 births. In 10% of the cases, there is a lack of DEA65 gene (retinal pigment epithelium), this gene produces an enzyme that allows retina cells to produce a pigment called rosedopsina, which serves us to look into the darkness. Children with Leber's disease born with the disabled DEA65 gene cannot form radopsina, and they have difficulty seeing in less enlightened environments. They progressively lose their sight and remain blind at the age of 40.

Genetic therapy.

Knowing that Leber's disease is caused by a single gene mutation, some scientists have long tried to find a method to allow them to replace the defective gene with another healthy. In 2007, American and English scientists published their first studies in which they had introduced an MPE65 gene into a virus with the idea of using it as a tool or a vector to insert a healthy copy of the gene into retinal cells, replacing the abnormal gene. The technique of using a virus as a vector is the basis of genetic treatments, and the process of injecting the virus into the tissue acts as a Trojan horse and is called transducing. Because it is dangerous to inject a modified virus that can penetrate normal cells, scientists make changes in the protein of the virus's membrane, making it more specific that they can penetrate changing genes. In 2009, Philadelphia scientists published a study in which children and adults with Leber's disease could recover their eyesight.

A million-dollar drug.

Even though the Sparcs lab instructions, the manufacturer of this drug, say they will set the award next month and that the Clinical and Economic Review Institute (ICER) will set the price on 25 January 2018, Wall Street analysts have estimated this will be the first drug to cost more than $1m. Of course this assumption has caused concerns in both America and Europe, much more if Luxurna's price does not justify its temporary effect. It is estimated that Sparks would earn $78 million in 2018, 238 million in 2019, and 445 million in the years ahead.

Reason.

Since genetic therapy has taken steps from the giant, it has been rumored that there is a cure for hemophilia and that drug prices from Luxturna will be astronomical, the Margolis Policy Centre at the University, gathering scientists, health insurance companies and pharmaceutical industries to discuss how to solve this problem. The question is: Why do they create a drug that no one is able to buy?

There is no doubt that this is a topic that will be talked about for years to come, but now we can't even imagine what would happen if an Albanian child is diagnosed with Leber's disease and needs Luxurna. /The world.al