More thoughts on Open Access and JQ1…

I know all about disappointments. I’ve tested on myself quite a few promising, non-toxic substances that showed strong antimyeloma effects both in vitro and in vivo. But they had no effect on me. Or, shiver!, as in the case of BCM-95, they made my MM markers worse…

I am also quite aware of the fact that substances that perform acrobatic cartwheels in a Petri dish or inside a sick mouse may end up tripping and falling flat on their faces when tested in human clinical trials. We’re not cells or mice, after all, are we?

But what I find extraordinary about the JQ1 discovery is the fact that it is really something new. NEW with capital letters. And exciting. As one blog reader wrote to me, “the image of that ‘scanned’ myeloma-free mouse will be imprinted on my retina for quite some time.” On mine, too…

This is not just what I call a “shuffling-cards” combination of third or fourth generation drugs…those derived, I mean, from thalidomide or bortezomib, e.g. If you go to the Clinical Trials website, as I did this morning, you will find heaps of myeloma clinical trials…1426, to be exact…

But let’s have a look at those  numbers for a sec: bortezomib (Velcade) is being tested in 289 of those trials. Its derivative, carfilzomib, in 22 trials. Now for thalidomide: 225…Its derivatives:  lenalidomide 168; pomalidomide 13. Dexamethasone: 311.  Melphalan: 249. And then we have those combination studies, that is, two, three or even four drugs tested together. For example: Dex plus lenalidomide plus doxorubicin or even bortezomib plus melphalan plus Dex PLUS thalidomide…I don’t have the time right now to go through and examine all of these studies and combos…But I recognize that it would be a very interesting exercise…For example, I saw and was reminded of TBL-12, the sea cucumber extract that I reported on some time ago (two trials now, with SMM patients). And there are a few other promising studies there, too…a handful, perhaps… 

However, and here we get to my first point, which drug or which combo can really give us cause for excitement? I mean, real, body-quivering excitement? Even more, which clinical trial can really give us hope that a CURE is on the horizon? 

I’ll leave it at that… An open question that cannot be answered, really…(But you can imagine what I think…).

Next topic…

I took a few days off to sit back and try to figure out what it was about the JQ1 study that got me so enthusiastic and sobbingly emotional in the first place. Hey, I’m not saying I’m not enthusiastic about this molecule anymore. Hah. Not at all. I still think JQ1 is a fabbbbulous discovery with shiploads of promise and potential. But okay, I have to admit I’m disappointed that it will take a couple of years (!) to get it into a clinical trial setting…And that did dampen my enthusiasm just a wee bit. Not much, though… 

But now I would like to discuss what Dr. Bradner said about OPEN ACCESS. Now this is exciting pioneering stuff indeed, and it pushes my enthusiasm levels right back up to the SKY HIGH setting. 🙂

Let’s take a look at the transcription of the TED Talk. By the way, the joke’s on me: a few days ago, with my flying, typing fingers I transcribed some of the most relevant parts of the TED talk…only to discover, once I’d finished!, that a transcript already exists…online…freely accessible, goes without sayin’. ARGHHHH! 🙂 Well, without further ado, here is the transcription link:

Let’s start with what happened after the Harvard team discovered JQ1’s effect on myeloma and other cancers: So we started to wonder, what would a drug company do at this point? Well they probably would keep this a secret until they turn a prototype drug into an active pharmaceutical substance. And so we did just the opposite. We published a paper that described this finding at the earliest prototype stage. We gave the world the chemical identity of this molecule, typically a secret in our discipline. We told people exactly how to make it. We gave them our email address, suggesting that, if they write us, we’ll send them a free molecule. We basically tried to create the most competitive environment for our lab as possible. And this was, unfortunately, successful.

I repeat: We gave the world the chemical identity of this molecule, typically a secret in our discipline. A secret. A SECRET.

And here let me state the obvious. Drug companies aren’t on our side. I don’t care how nice and warm and friendly and sympathetic their representatives are or how many patient-doctor meetings they organize and finance. That’s fluff. A smoke screen. 

Drug companies exist to make a profit. Nothing else. Mind you, theoretically, there isn’t anything wrong with that. After all, these are businesses. And businesses have to make profits. A no-brainer, that one. Unfortunately, though, while they’re busy making profits and possibly/probably being secretive about potentially life-saving discoveries, cancer patients are dying…But that makes me crazy, so let’s keep going…

To be super honest, none of this stuff–secrets and Big Pharma and so on–comes as a surprise to me. But this is the first time I’ve actually heard a researcher, an MD!, come out and say it openly and very matter-of factly…And therein lies the “surprise!” element. For me, at least. 

Now, as we know, the Harvard research team mailed their molecule to 70 labs around the world. Probably more, by now. For free. And they received feedback from those labs. 

And this is where things get rrrrreally interesting: “…the science that’s coming back from all of these laboratories about the use of this molecule has provided us insights that we might not have had on our own. Leukemia cells treated with this compound turn into normal white blood cells. Mice with multiple myeloma, an incurable malignancy of the bone marrow, respond dramatically to the treatment with this drug.”

(Another thing I really like about this guy is his humility…”insights that we might not have had on our own”…)

And then, wham!, we are hit with can only be seen as a revolutionary approach to medicine and science. In Dr. Bradner’s own words, this was a social experiment, an experiment in what would happen if we were as open and honest at the earliest phase of discovery chemistry research as we could be. This string of letters and numbers and symbols and parentheses that can be texted, I suppose, or Twittered worldwide, is the chemical identity of our pro compound. It’s the information that we most need from pharmaceutical companies, the information on how these early prototype drugs might work. Yet this information is largely a secret. And so we seek really to download from the amazing successes of the computer science industry two principles: that of opensource and that of crowdsourcing to quickly, responsibly accelerate the delivery of targeted therapeutics to patients with cancer.

The “sharing for free” approach. The open access approach. To get stuff streamlined and down to patients more quickly and responsibly…

If a cure for cancer, a cure for myeloma, is to be found, labs all around the world are going to have to share their findings and work together. I’m convinced of that now. 

And, mark my words, this will take place only thanks to researchers such as Dr. Bradner, researchers with open minds, or rather, with open access minds!… 🙂

No more secrets! No more secrets! No more secrets!

P.S. I’ve begun following what the online media is saying about JQ1. You might be interested to read about the research being done at Notre Dame, e.g., on JQ1 and NUT midline carcinoma, the cancer mentioned by Dr. Bradner in his TED talk: (Please note that Dr. Bradner and Harvard are not mentioned here…Odd, eh?).

And here is a Science Daily report, which I totally missed back in September…better late than never, I guess:


  1. Hey Margaret, I kinda thought that Kathy Giusti and the MMRF were pioneers in “open source” for the MM community. Acting as a clearing house for the latest research seems to help promote an acceleration of clinical trials. But you are right … there is very little NEW about that research. Coming from an IT background, I appreciate the concept of “open source” and have seen that Linux can coexist with Microsoft and provide expanded avenues for earnings and profits for everyone involved. My fear is that BigPharma will somehow take Dr Bradner’s research and make the products derived from it obscenely expensive for those of us who suffer from the disease.

  2. Hi Margaret,
    You are bang on with this post. The only point that you have missed is that the big pharma companies will not really be interested in developing a cure for myeloma because they wouldn’t make any money out of it! You can sell a cure only once and MM is a relatively rare disease. Therefore big pharma will prefer to develop products that keep us alive and dependant on them.

    I have always thought that big breakthroughs like this would come from university labs and we need to find a way of turning their research in products. This is the expensive part and big pharma won’t fund it if it’s not profitable.

    Dr Bradner will need all our support in the coming years both morally and financially. His open source approach will be seen as very threatening to the status quo. However, as I said before, this movement promises enormous benefits to mankind. What if it could have cured Steve Jobs for example? We also need to consider the potential savings to our overstretched health services if we produce more cures and fewer products that create dependancies.

    Maybe we should start lobbying the Nobel Institute now and hopefully they will reward the altruism of Dr Bradner and his team and give them the recognition that they deserve. To me, their open-source approach is more important than the JQ1 compound itself.

    We could also try lobbying governments and charities to get them to provide more support for developments like this. Although governments are strapped for cash as a result of aging populations and rising healthcare costs, their future savings are potentially enormous. In the end it is a simple investment decision. A stitch in time saves nine. For example, I have seen studies that suggest that if we could find a cure for diabetes we could reduce national healthcare costs by 10%. I wonder if a similar calculation has been made for the benefits of curing cancer.

    Best wishes,

  3. Kent, you took the worsds right out of my mind. Kathy G was supposed to speed up the process but I am not sure what sharing means to them, or if they ran into road blocks that required them to change their model without letting us know.

    Paul, excellent points as well. I don’t understand why there is not a consortium of rich investors, like the business world that has venture capital (I admit I used to call them vulture capitals), or government etc to fund these. I think the Alberta government is doing just that to fund trials for DCF.

    I am unsure whether insurance companies would want to fund it. I used to think they would, but maybe stats prove more money in death?

    Margaret, I cannot say enough about these posts. I wish I could help you, but I love standing back and watching your mind work.

    Remember everyone that trials take a couple of years because the studies need to be done on people for sufficient time to find the right dose, find the right groups to qualify, check for side effects, document it all, and so much more. Before open source it might have taken 10 years. If you think about Don, I think he has been participating in the study for over one year now.

    Thank you Margaret.

  4. Hai ragione Margareth, hai ragione al mille per cento. Loro sono là con i loro bei sorrisi da commessi di Mediaworld ed io sono qui a 32 anni col mieloma, dopo aver visto un mio amico conosciuto in degenza l’anno scorso morire di leucemia.
    Mi viene da piangere.
    Ciao e grazie per il lavoro che fai.

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