A GENE VICTORY FOR THE NEW YEAR
Before the New Year, I am looking for something positive, but it found me flashing in the news.
For the third time in the past three months, the FDA has approved gene therapy, the path to which is difficult and circuitous in the American market. The first such treatment was Kymriah for leukemia, and then Yescarta – a treatment for one of the forms of lymphoma.
Perhaps gene therapy for a number of diseases, or even a complete cure, is becoming mainstream right before our very eyes.
The third has to do with the eyes – Luxturna. It is used to treat a progressive loss of vision, which usually begins in childhood, but ends in blindness during adulthood. Thousands of people inherit two false copies of a gene, one from each parent. Luxturna replaces these copies with normal versions of the gene, which erases the impact of the negative mutation.
This procedure will be expensive, but that’s common for gene therapy. Kymriah costs $475,000 for each patient, and Yescarta – $373,000. The annual turnaround from the sale of Luxturna will reach $478 million.
Alas, if the prices weren’t so high (including prime cost), then these treatments wouldn’t have come about. The procedure itself appears to be an injection into the eye area adjacent to the retina. The correct gene uses a denatured cold virus as transport, so the patient doesn’t get sick.
And if the patient still has viable retinal cells, then the treatment restores the production of that very wonderful protein that converts light into an electrical signal, thanks to which we can see how beautiful this world is.
Until recently, it was believed that this method of transferring genes was deadly, and at best caused such side effects that if we were to call it a success, it would be a Pyrrhic victory. But the next breakthroughs in genetic engineering allow for using a virus as a “transport”, while also avoiding viral infection.
The company that created Luxturna isn’t resting on its laurels, though, sipping cocktails on a beach in Hawaii, but is instead working on methods to fight hemophilia and neurodegenerative disorders.
And now, friends, we are entering the New Year with good news!
Photo by Marina Vitale on Unsplash