"Both the National Cancer Institute and several pharmaceutical companies declined to pay for the research. Neither applicants nor funders discuss the reasons an application is turned down. But good guesses are the general shortage of funds and the concept tried in this experiment was too novel and, thus, too risky for consideration."
The keywords are "experiment was too novel, and thus, too risky for consideration". Unfortunately, this seems to have become the rule rather than the exception in scientific research. All the young folks (pre-tenure) are on a treadmill to get funding and publish something in the top journals/conferences. I wish there was some accountability in the system ... the geniuses who rejected funding this research will go about completely unaffected.
Several years ago I read an article in Fortune about cancer research. Government funding is extremely conservative. They're happy to pay you to nail down another tiny detail about some kind of cancer, but try an innovative cure and they balk. They also take three years to get the money to you, by which time your research might be obsolete.
When Michael Milken got out of prison, he was diagnosed with prostate cancer, and started a foundation to fight it. He asked researchers for the crazy ideas they really wanted to work on, and got the money to them in three months. In return, he insisted researchers immediately share what they learned. No patents, no waiting for prestigious publication.
According to Fortune, people in the field generally credit Milken's foundation with cutting prostate cancer death rates in half.
"Criminal" is a good qualifier for the way most medical research decision are done. I was once told, out of the blue, by a commercial in the field, that research for a HIV cure was a very low priority given how profitable the tritherapy was.
I've probably got him to thank for still having a stepfather - he was just treated for prostate cancer and it seems to be working. (In his case, they put tiny radioactive beads into the tumor to kill it over six months, which is really pretty damned awesome.)
The problem here is one of sampling bias. You're seeing an example of one "outside the box" idea that worked out of thousands that didn't. The NIH and pharmas are constantly getting pitched on (and funding) ideas for innovative new therapies. The problem is that cancer therapies under development fail at a rate >95% in the clinic. Therefore, most new approaches to cancer treatment turn out to be abject failures. The fact that funding agencies and pharmas passed on this specific one isn't surprising. They just can't fund everything.
The good news is, these guys should have no trouble getting funding now.
This is an important point. Medical innovation can be perilous. I'm reminded of the setbacks for gene therapy in the 1990s. The case of Jesse Gelsinger was emblematic:
Every time I recall this, I mean to lookup whatever became of this guy. Finally did. A bit of a follow-up here:
A test of his protocol, published in 2009, found that Gonzalez's patients died faster than those treated with conventional chemotherapy, and had significantly worse quality of life.
Just to update this since there was so much interest, I looked into the literature a bit more closely.
First off, at the end of the article it says "Supported in part by grants from the National Institutes of Health (K24 CA11787901, 1PN2-EY016586, and R01CA120409), the Alliance for Cancer Gene Therapy, and the Leukemia and Lymphoma Society (7000-02)." So the funding system was working as expected.
It also looks like people have been trying this for a long time and for various reasons it has actually not worked very well. This paper is noteworthy not because they used the HIV modified T-cells. It's noteworthy because people have been banging their heads against this specific brick wall for a long time and coming up empty-handed. The key innovation appears to be the addition of extra machinery that made the T-cells more potent and also the ability for the T-cells to hang around and expand in the patient.
Also, to give people a sense of how much time something like this takes. This specific viral mechanism was tested by the same group in mice first and published in August of 2009 (open access: http://www.nature.com/mt/journal/v17/n8/full/mt200983a.html)
My guess is they balked when the researchers decided to use a modified HIV virus to implant the new genes. If it went badly everyone would point to the HIV and the bad press could be devastating.
Thankfully these researchers were able to take some big risks and prove out the theory in a fairly successful trial.
HIV is used to insert the genes into the white cells in-vitro. They are not inserted in the patient. So I guess the risk of HIV infection is quite small (how small, I have no idea).
The entire pharmaceutical industry is basically going to collapse by the end of 2014 anyway, so it's largely irrelevant. There will be much less funding for everything no matter how promising it is, unless the government steps in and reinvents the entire process. Seems unlikely at this point though:
On the other hand, as research tools are becoming more affordable to smaller institutions and universities, we may witness a shift from big pharma to smaller and more focused independent research.
The article doesn't touch on one aspect of the research likely to have been particularly unpalatable to pharmaceutical companies: Potential cures are bad business.
Of course, there is no conspiracy here. It's just the human tendency to rationalize when money is involved. Imagine you run a pharmaceutical company: Your products save lives, right? You want to be able to continue saving lives, so you have to stay profitable. And to stay profitable, you have to make good investments. Products that are used in long-term treatments are good investments. One-time cures aren't.
I spent 8 years in pharmaceutical industry R&D and this is so ill-informed I feel compelled to refute it every time someone says it. I know it sounds like it makes sense on the surface, but it's just outright false.
I worked in cancer research with some really, really smart people. Every single one of them was sincerely hopeful that the drug they were working on would be a total cure. I know it's hard to believe, but pharma companies are composed of real people who have seen patients, friends, and relatives die of cancer or similar diseases. Researchers want so badly to find a cure for the disease they are working on. The existence of vaccines are proof that when pharma companies can cure something, they do it. I have been in conversations where business factors for a drug were discussed and I can tell you the one thing that was never said was, "Hey guys, make sure it's not an actual cure, or we'll need to kill the project".
Nice to see that not everybody is a greedy asshole :)
A commenter on Slashdot gave another good point on why the industry HAS a reason to develop a cure for cancer:
If any "Big Pharma" company invented a cure for cancer tomorrow, you can bet your ass that they'd be all over it in a heartbeat.
Why? Because, then that company would forever be known as the company that cured cancer. Every new product they make would be a pot of gold. Every ad they put out would be "Muhdikard, a new treatment for erectile dysfunction, from Drugco. We cured cancer.". Every drug company on the face of the planet would kill for that kind of marketing, not to mention the money from selling the cancer cure.
Firstly: You're right; I am ill-informed on this. I haven't any doubt that you or your colleagues had and continue to have strong humanitarian convictions in your work, and I was out of line to speak so broadly.
I recognize that the argument I made earlier is something of an old saw. But you and I both know that businesses on the scale of most pharma companies are not run only by researchers and engineers, and that the explicit reasons for business decisions anywhere often stem from any number of unspoken ones. I don't imagine even the coldest of bean counters ever says "we need to make sure this research doesn't lead to a cure," and I doubt such a black-and-white assessment of a complex R&D process even occurs to them. But incentives are incentives, and humans are masters of rationalization.
I'm heartened to learn that your teams never felt pressure to put business interests ahead of patient interests. I sincerely hope your experience is common.
Thanks for your comments. The pharma industry has a lousy public perception, some of which is quite justified, so it can be hard to separate the reality from the perception.
There is also a practical reason why I can assure you that people are usually swinging for the fences, especially in cancer: most of the time you don't even know if a drug will work, let alone work well. There is no way to predict (with the obvious exception of vaccines which owing to biology are destined to be cures from the beginning) what your drug will really do until it hits the clinic. So there isn't even a mechanism to offer people an incentive to develop a drug someone has to take over and over vs. a cure, especially in cancer. Certainly, there are chronic conditions like diabetes where there is no real "cure" on the horizon because of the biology of the situation. But in the case of cancer, you almost have to try for a total success because you know you're likely to get far, far less than that when you hit the clinic. Because of this problem, one of the bigger issues in pharma is that flawed projects are allowed to linger long after they should have just been scrapped because powerful scientists believed they would work and convinced management to keep funding them despite some of their issues.
Boo! Disagree. JunkDNA gave you a perspective from the Pharmaceutical company side. Here's a perspective from the finance side:
I worked for 2.5 years in a finance firm which specialized primarily in biotech stocks. (We were on the "sell side", meaning our clients were hedge funds and mutual funds, and our product was research reports advising whether to buy or sell the stock of certain biotech companies.)
Without fail, the biggest catalysts for moving the stock price were study results, and the better the results, the bigger the jump. Every company absolutely wants that blockbuster drug that cures a disease.
With CLL, for instance, as in the article, there are a number of treatments, in 1st line and 2nd line after relapse, 3rd line, and so on. And doctors prescribe this one or that one, depending on context and what side effects you can tolerate, etc. If your drug is a complete cure and you can jump straight to first line and everyone prescribes your treatment, then that's a gold mine.
A recent example is Cougar Biotechnology, whose cancer drug abiraterone was so insanely successful the company's stock price rocketed and then was acquired shortly thereafter by Johnson & Johnson.
Lastly, if you finished reading the article, you'd see this part:
"With results for the three patients published Wednesday simultaneously in the New England Journal of Medicine and Science Translational Medicine, money for further studies -- not just in this one type of leukemia, but in other cancers -- will likely pour in from both the government and drug companies."
That's why I think medical research should be funded by the insurance companies.
The main impediment: free-loading companies could take advantage of the companies funding the research. One solution would be to start a patent pool, making their inventions freely available to all the companies participating in funding the research.
A senator (Bernie Frank maybe, not sure) recently introduced a bill to tax insurance companies and apply the funds for medical research. That'd be another way to fix the freeloader problem, but then you're stuck with the government choosing the research, and it tends to be overly conservative.
Wrong mind set, they could easily charge 100k+ for the treatment and insurance companies would still jump for joy since it's cheaper over the long run.
Surely a cure to an otherwise generally terminal illness is worth 100k though. I know I would be grateful to pay such a price, the alternative seems to be no cure, and eventual death.
I'm sure there are tons of people who would be grateful to be in a position to be able to pay such a price.
I think a company would be hard-pressed to have a pill that cures cancer -- letting millions die each year, because the pill costs $100k. I think there's a non-trivial possibility that the gov't would take that drug for its own uses.
No they wouldn't there are a lot of procedures that cost more than a 100k (any transplant, open heart, high end implants are more). Really the government and insurance companies would save a fortune if the cure cost between 100k - 200k.
Apparently when the negotiations broke down (Brazil wanted the same rate as Thailand but Merck wanted to charge them more), Brazil declared sovereign property rights in the good of it's citizens.
This was for Cipro for Anthrax. Bayer, at the last minute, gave in to some pricing concessions. There's also been talks about it happening during some H1N1 and avian flu scares.
A $100k pill that cures cancer? I'm not saying it would be taken by the gov't, but I think you'd have to seriously consider it.
Quick research tells me that there ar about 45,000 cases of leukemia in the US per year. Equally quick research tells me traditional treatment easily comes to $400k, and comes with terrible side effects.
So, if you had a fairly reliable (at achieving remission) injection, and especially one without the terrible side effects, you should be able to charge at least $400k.
Please inform me which pharmaceutical company would not like an $18 billion/year revenue stream.
The problem is that this phenomenon (funding research where you know there is a good probability it will work before you even start) is not limited to medical research, it can be found even in fields where there are no commercial interests involved.
Opinions differ as to why that is - I personally suspect the grants system that is used for funding science.
"Both the National Cancer Institute and several pharmaceutical companies declined to pay for the research. Neither applicants nor funders discuss the reasons an application is turned down. But good guesses are the general shortage of funds and the concept tried in this experiment was too novel and, thus, too risky for consideration."
The keywords are "experiment was too novel, and thus, too risky for consideration". Unfortunately, this seems to have become the rule rather than the exception in scientific research. All the young folks (pre-tenure) are on a treadmill to get funding and publish something in the top journals/conferences. I wish there was some accountability in the system ... the geniuses who rejected funding this research will go about completely unaffected.