CAR T-cell therapy – Novartis and the question of ‘how much?’

CAR T-cell therapy – Novartis and the question of ‘how much?’

October 29, 2017 0 By Michael
Reading Time: 3 minutes

How much? I imagine that question came up eventually at one of the meetings of whichever team decides pricing strategies at Novartis.  And they came up with an answer: 475000 $. Four Hundred And Seventy Five Thousand dollars for a one shot therapy that can potentially cure a rare type of cancer, acute lymphoblastic leukemia (ALL). In other words, it’s half a million to save a life. Is that morally reprehensible and extortionate or a smart move? A restraint pricing strategy because analysts expected more? Depends who you ask of course. But still it’s way too easy to make Novartis out as the villain here, to blame big pharma once again for gambling with people’s lives. If you really must blame someone, blame us all because somewhere along the way we, as a society, decided to make healthcare a private enterprise. Novartis is a publicly traded company so nobody should be surprised really that they put their shareholders first and everybody else second, some might even argue it is their responsibility (technically they have to put their creditors first but hey, it’s for dramatic effect). Still, we don’t want to call the revolutionary forces to arms just yet and anyways we are here to inform rather than point fingers so let’s have a closer look at what all the excitement is about.   

Novartis just got FDA approval for a cancer “drug” against ALL and priced it at 475000 $. Another cancer drug with a huge price tag, so far so normal. Only that this is not a drug in the traditional sense and one shot of it can potentially cure a patient. It’s a therapy that trains your own immune system to fight the cancer cells. Really, it goes far beyond simple training. It’s called a chimeric antigen receptor T-cell therapy (CAR-T) and it is the first of its kind to be approved (Gilead will likely get approval for their own version in November after acquiring Kite Pharma in September). The therapy employs two components of your immune system, antibodies and T-cells. T-cells are the shock troops of the immune system, their task is to eliminate foreign invaders like bacteria or viruses. Once primed for a particular target they will circulate through your body, find it and kill it. Naturally, you would not want your T-cells turn against your own cells. If they do, they are called autoreactive and autoimmune diseases illustrate the detrimental consequences this can have. 

T-cells need to be excellent at distinguishing between “foreign” and “self”.  And they are usually, it’s one of the things that keeps us alive in a generally hostile environment. The problem with cancer is that it is not a foreign invader, it’s you own cells causing havoc. Since it is your own cells your immune system is a bit reluctant to fight it at first. And once it starts it’s faced with a formidable opponent. The cancer will use every trick in the book to hide from, mislead and shut down your immune system. So, your immune cells could really do with a nudge in the right direction. This is where the antibodies come in, to show the T-cell what exactly it is they should attack. Antibodies can be produced to very accurately and selectively recognize basically any kind of pattern (usually part of a protein on the cell surface of the target). Such a pattern is called a specific antigen. In CAR-T therapy antibody fragments are designed against a unique antigen (CD19 on B-cells if you need to know precisely) on the target, the cancer cells. In the course of the therapy your own T-cells are harvested and genetically manipulated to produce the cancer specific antibody and link it to their cell-surface. That gives the T-cell the ability to recognize the cancer cell. Simple recognition is not enough though, the T-cell needs to be activated to kill the cancer cell. This activation is achieved through a neat little trick. Essentially the antibody fragment facing the outside is linked to an “on-switch” inside the T-cell. That switch sends the T-cell into overdrive, killing the cancer cells. Sounds simple but the whole process is rather elaborate and time consuming, which is one of the reasons put forward to justify the high price. 

That’s it, CAR-T therapy in a nutshell and overly simplified. If you want to know more about CAR-T therapy, cancer or the immune system pay us a visit at the Ivory Embassy. We have detailed explanations for those and many more cutting edge scientific topics.  

But wait, what about big pharma? Are they off the hook? Not entirely, there is one thing we should demand: Nobody dies because of restricted access to a possibly lifesaving therapy. Novartis let the genie out of the bottle, so to speak, and presented a revolutionary therapy to treat cancer. The others, Gilead, Pfizer and the likes, will soon follow suit. This is only the first therapy of its kind and it certainly won’t be the last. Realistically, they won’t get much cheaper either. But we, as a society, can demand that everyone will have access. Let them charge the people who can afford it but at the same time make it the companies responsibility that nobody dies because he/she could not.     

 

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