"The Food and Drug Administration on Wednesday approved the second in a radically new class of treatments that genetically reboot a patient's own immune cells to kill cancer.
The new therapy, Yescarta, made by Kite Pharma, was approved for adults with aggressive forms of a blood cancer, non-Hodgkins lymphoma, ,who have undergone two regimens of chemotherapy that failed.
The treatment, considered a form of gene therapy, transforms the patient's cells into what researchers call a "living drug" that attacks cancer cells. It is part of the rapidly growing field of immunotherapy, which uses drugs or genetic tinkering to turbocharge the immune system to fight disease. In some cases the treatments have led to long remissions."
The Food and Drug Administration allowed sales of the treatment from Kite Pharma. It uses the same technology, called CAR-T, as the first gene therapy approved in the U.S. in August, a treatment for childhood leukemia from Novartis Pharmaceuticals.
"In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer," FDA Commissioner Dr. Scott Gottlieb said in a statement.
The treatment, called Yescarta, will cost $373,000 per patient, according to drugmaker Gilead Sciences. Kite became a subsidiary of Foster City, California-based Gilead this month.
A second new personalized treatment for cancer has won approval from the Food and Drug Administration — a clear sign that such treatments will become more widely available for patients with no other options.
The treatment is called CAR-Tand can have dramatic effects in some patients. But it is both grueling and expensive and will remain a last-ditch treatment for a few, specific cancer types.
The company that will market it, Gilead Sciences, has priced it at $373,000 and the FDA warns it can cause severe side effects.
The therapy is called Yescarta — the generic name is axicabtagene ciloleucel. It’s described as gene therapy because the patient’s own immune cells are removed and genetically engineered to better fight off the cancer.
It's a little different from similar approaches in which a cancer patient's immune cells are harvested and numbers boosted in the lab. And it's different from other gene therapy treatments for inherited diseases.
“Yescarta, a chimeric antigen receptor (CAR) T cell therapy, is the second gene therapy approved by the FDA and the first for certain types of non-Hodgkin lymphoma,” the FDA said in a statement.
For myself, this sounds like a fantastic step forward, and I hope that it leads to an eventual cure, not just 'long remissions'. No, not against genetic therapy when it comes to improving the health of people, though once insurance is brought into it, can't help but think they're going to find some way to really fuck it up.
The cost sounds staggering, as always, but given this is looked at as a last chance ... dunno. Have no idea the costs of developing it, and what might be needed to continue to attempt to streamline and improve it. That's completely outside my realm of expertise, unfortunately. As for the risks, well ... these folks are already at the point of 'we can't do much more for you'. My cousin lost her husband to a long, painful battle that she documented on a blog for friends and family, but primarily for herself and her children. Wish they would have had this available way back then, and I'm certain they would have jumped at the chance, however slim it might be, to beat that beast back for a little longer.
So what do you folks think? Good, bad, dangerous, any real hope or another tool of Big Pharma to suck us dry? For or against gene therapy, and why? Let's hear it. Anything for a chance at something positive, and a break from our usual Circus of Idiots out there in DC, neh?
