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…).
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: http://goo.gl/is3kU
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.
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: http://goo.gl/r6ACG (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: http://goo.gl/jvRbu