'Game changer' cancer therapy approved

When you think of drugs you probably imagine pills in a packet. But last week there was a landmark ruling - the FDA (American Food and Drug Administration) approved a new type of treatment for one form of blood cancer. And it could be a game changer. To tell us about it, Chris Smith was joined by Magda Papadaki of the Association of the British Pharmaceutical Industry.

Magda - We had the most exciting piece of news in the war against cancer we’ve had for quite some time. The FDA approved Kymriah  for the treatment of pediatric (that’s children) and young adult patients below 25 years of age in a rare and very aggressive form of beta cell lymphoblastic leukemia (B-cell ALL), which is a very aggressive and rare form of cancer. The treatment would be given to children that are either refractory or relapsed, which means that they’ve stopped responding to any other treatment or that their cancer has returned after everything else has been tried.

Chris - This sort of tumour, ALL, is the most common pediatric cancer isn’t it? About 25% of children who get cancer have this. At the moment, we can treat that and about 80% of people get better, don’t they, but for the 20% who don’t, it’s a pretty grim prognosis. But now we’ve got this drug, how does it work?

Magda - The immune system is the first line of defence our system has against viruses and against diseases like cancer, and the t-cells are it’s primary soldiers. The goal is to target and kill infected or abnormal cells and what cancers try to do is stop or make these soldiers less effective, which is what inspired the scientists to pursue this new line of treatment.

The scientific description of Kymriah is a long chain of buzz words. It’s autologous gene modified t-cell therapy. What this means: autologous means that every dose of Kymriah is a bespoke patient-tailored therapy that uses its own cells, its own immune system to seek and destroy cancer cells.

Chris - So you’re saying you take the patient’s own white blood cells, their own t-cells out of them?

Magada - Exactly. The story starts like that. In the hospital where a safe amount of blood is taken from the patient, and the white blood cells that include t-cells and other types of cells are taken out. These are moved to another research and manufacturing facility where they undergo gene therapy using viruses that have been rendered safe - that means they can’t lead to any further disease.

What the viruses do is that they have the genetic instructions for the t-cells to start encoding an artificial receptor. It’s what Mene Pangalos, to use his analogy, described as a key. This key is fundamental in the ability of these t-cells to identify the leukemic cells in the body and start destroying them. Once this change has taken place, the t-cells are cultured in millions and infused back to the patients where they are released to the blood stream and start their fight against cancer.

Chris - Right. So you take cells that naturally in the patient’s own body are the killers that go out and destroy cells anyway. You reprogramme them by adding a piece of genetic information, which is the instructions this is how to attack your own cancer, put those cells back in and they then go round the body hunting down that person’s cancer?

Magda - Exactly.

Chris - And is it successful?

Magda - Yes. Again, that was the most exciting in terms of the results. The clinical trial that the FDA assessed and gave the green light included 63 children, and the results are that for 83% of them there was complete remission that continued for three months after the initial dosing.

Chris - To put that into perspective, we mentioned at the beginning that these are the 20% of individuals that would not  previously have had any option open to them. They would have failed the primary therapy, the prognosis was grim, and so 80% plus of these individuals are being potentially saved by this?

Magda - These were the children that had no other option; they were dying. The medicine they were taking in our arsenal does not work and the cancer had returned.

Chris - Novartis did get a bit of backlash because when people heard the price - because this is estimated to be costing in the region of half a million dollars to treat each individual - people said that’s too expensive. What do you think?

Magda - These are totally new discussions and the approach that we are hearing that Novartis is taking with the US payers is the value based one. Again new in terms of pricing and reimbursement. The other alternative that these children had, and for some a treatment that had also stopped working was bone marrow transplantations which costs along the lines of 800,000. So again Novartis is offering about half the price but we are yet to see where those negotiations and discussions will lead.

Chris - It’s interesting, isn't it, because your industry started off look at bags of pills and it’s actually going to end up looking at therapies which include cell therapies in future, increasingly probably?

Magda - This is very right and, hopefully, it’s the wave of innovation that came out of the deciphering, the decoding of the human genome about 14 years ago in 2003, so I hope we’re just seeing the tip of the iceberg.