UKC

I don't know if this has been posted yet, but excellent preliminary results in treatment of blood born cancers.

http://www.cancer.gov/about-cancer/treatment/research/car-t-cells

In reply to GregC

I got first hand that the doses they are using in the trails in GOSH are in the ball park £250,000 each, and there was a quote on $1 million cost a year for other treatment combinations.

Don't expect it on the NHS anytime soon

In reply to DrIan:

Won't be costing anything per unit cost to the NHS in a trial. Not unsual for monoclonal treatments to cost hundreds of thousands per patient in early market use. Presuming its not a orphan (where the development and the manufacturing costs are supported and offset by the government) - current cost of treatment chemotherapy, supportive care and procedures such as bone marrow transplant probably cost in excess of a million plus cost to patient in lost earnings, lowered life span and significant deaths during treatment , this has early data > 90% success rate > very cheap.

D

D

In reply to DrIan:

I think the price would come down when the genetic modification of the T-cells has been optimised and automated. I suppose that it is an economies of scale problem.

A number of current cancer treatment are platinum based drugs and platinum will only continue to get more expensive. One of the starting materials for this procedure is the patients blood which is free so I think it should (eventually) be cheaper, although I don't know know the cost of the other materials involved.

In reply to GregCHF:

This is a very exciting result, however while it is effective, it is also risky. I believe there were two deaths and several more acute illnesses as a direct result of the treatment during the trial. Of course, this is far more acceptable when you are trying to treat terminal cancer than when you are trying to treat a cold - however it does mean that for the time being at least this will only be a last-resort treatment when chemotherapy and other currently available treatments have failed.

Hopefully it will become safer as the technique is refined. I am looking forwards to seeing the paper.

In reply to GregCHF:

This is very big.

Not in the treatment itself (although that is big), but in heralding in an era of treatments designed from scratch, with an understanding of how a disease works, how a body works, how the immune system works (or fails) and how they all interact.

There was some strong disagreement on UKC a few years ago when I suggested that the importance of trawling the rainforest for cures is going to decrease in the future as we gradually become masters of biochemistry, but I stand by that.

Mind you none of this would have been possible without HIV and it's like, and it looks increasingly like mammalian life as we know it is only possible because of HIVs ancestor viruses. The real keys to the kingdom were found in diseases.

In reply to DrIan:
That figure is rather blown out of perspective
First GMP batches of drugs for human trials always cost the earth; method development, scale up, characterization, fill-finish, plus all the extra safety bumpf that goes with a brand new ATMP. You also need to include in manufacturing of the vector and CMO's will not have a shake and bake method already established like they do for small/large molecule drug development.

The drug company won't be dumb enough to pitch the price at an unaffordable rate, they won't make their return on investment before they hit the patent cliff. The pharmacoeconomics for this would have been done right before the pivotal preclinical studies and the investment simply wouldn't of happened if they couldn't find a way for it to be affordable.

Give it 5-10years and we will have stepped away from palliative care provided by small and large molecules, we are running out of easy targets for mAbs, gene therapy is the way forwards. Products like this spearhead their development.

In reply to wintertree:
It is obvious that biochemical treatment is becoming more and more important. As a chemist, I am (sad to be) seeing a lot less funding for total synthesis of target molecules. There is a definite trend to biochemical research.

It is also interesting to see the discussion around phage therapy as a way of combating bacterial infections. Again moving away from finding new antibiotic targets to synthesis.

I suppose that chemistry is a bit like other areas and total synthesis of targets will come back in to vogue in 10 years time.

In reply to GregCHF:

Viral vectors have their own issues, mostly to do with who owns the IP, companies own the capsid, plasmid and vector rights which makes it really challenging to develop a complete phage which you will own the full rights to. No one will invest in you when the slice of the pie is so small to begin with. The added complexity is that different viruses have different target specificity, even within the different serotypes there are big differences. The CMO's who will produce modified vectors are mostly university spin outs whom prioritise their own research or are commercial entities which large pharma are going in and setting up exclusivity contracts on particular disease indications.

The problem is there simply are not enough people out their doing this yet.