Bench to Bedside
A little background on translational research or medicine, often also called “bench to bedside”. In a post by Max Huijgen (http://goo.gl/VNT3Z) there was a lively discussion about an article in the Telegraph about a seemingly miracle cancer drug sitting in a freezer in Sweden. The article stated that because the scientist had published the work, no pharmaceutical company would touch it, to bring it to market. The idea was, no patent means no profit. A large part of the discussion was about crowdfunding for this frozen drug.
There were three things that Rajini Rao some others, and I tried to explain, about the article and the conversation. First, the cost of getting a drug “from bench to bedside” was grossly underestimated by people suggesting crowdfunding. Here’s the reference Rajini provided regarding the cost of clinical trials (http://goo.gl/YXD55). It can be $28k per patient, just to get started, i.e., Phase I. Which leads to the second issue; lack of understanding what is drug development or discovery. How do you get a compound from idea, to bench work, and finally to market. The last issue is the treatment in the story itself.
The figures below are from an excellent overview of the process of drug development. The example is for cancer research but it is applicable to essentially all drug discovery. So when you hear scientist like me, talk about Phase III clinical trials or INDs, you’ll know what they mean. http://goo.gl/ZF88v
There is also a good, albeit a bit old, review on translation research.
Review: Translational science: past, present, and future by S. H. Curry
BioTechniques 44, 2008 http://goo.gl/B5DV6
In that review, they mention acetaminophen being published and therefore the university couldn’t capitalize on the discovery. If the premise of the Telegraph article were true then acetaminophen should not be in every medicine cabinet today. Clearly the inability to directly patent a compound does not preclude getting it to market. I say directly because acetaminophen was patented at some point.
History of acetaminophen or paracetamol
http://en.wikipedia.org/wiki/Paracetamol#History
Getting back to the Telegraph article and the miracle drug. The Swedish group’s current paper, that has in vivo data (the rest are in vitro) has a 40% survival rate at 100 days, using six mice per group. I also find it curious that the virus is supposed to be targeted but they did not inject it systemically. It was injected directly into the tumor.
J Virol. 2011 Dec;85(24):13114-23.
http://www.ncbi.nlm.nih.gov/pubmed/21957304
Although it is interesting and promising work, I wouldn’t put any money towards it with such a small study. Typically, much more pre-clinical work is done before even thinking of putting a therapeutic agent into humans. As others stated in the thread, there are tons of therapies that cure mice but do not work in humans.
The adenovirus that I have worked on is not targeted per se. It has to be injected directly into the tumor. However, the gene is activated only where you irradiate. The tumor gets a double whamy: radiation and damage from the gene therapy. It was in a phase 1 trial, published here: Clin Cancer Res. 2004 Sep 1;10(17):5747-53. http://www.ncbi.nlm.nih.gov/pubmed/15355902?
I want to emphasize that I have nothing against the research done by the Swedish group. I do have an issue with the Telegraph article. The author talks about his keen investigative skills to find the Swedish group but yet doesn’t do due diligence in finding out the true potential of this therapy. Again, it’s interesting work but a 40% survival rate in mice, in a small single study, is not a miracle drug. The author even mentions the Amgen “investigation” (but gives no reference) as a reason to be cautious. It’s actually a commentary, not a peer-reviewed publication. My commentary about it was posted a while ago, here: http://goo.gl/98HmX? One should be cautious because a lot promising pre-clinical studies don’t pan out, not because of a commentary in reproducibility. Reproducibility is certainly important and is a problem in some areas of research. It’s frustrating that the post follows the same tone as the Telegraph article. However, as a proponent of science, I’m happy that there was a lively discussion and there was an opportunity to educate.
#ScienceEveryday when it isn’t #ScienceSunday
