Two weeks ago I re-visited the promising new anti-cancer therapy, JQ1, after learning that JQ1 may also hold the key to male contraception.

If you haven’t been following along,  last summer a group of Dana-Farber Cancer Institute scientists reported that they had “successfully disrupted the function of a cancer gene involved in the formation of most human tumors by tampering with the gene’s “on” switch and growth signals, rather than targeting the gene itself.”

Although these researchers hope JQ1 technology will work to thwart a number of different cancers, much of the early work was done using multiple myeloma cells.

Very few topics that I have written about have provoked as many reader comments as JQ1.  The fact that a single re-engineered molecule could draw such interest is probably the result of an almost perfect storm; the hopeful promise of a yet unproven and mysterious creation, Dr. Bradner’s charismatic appeals for funding on Ted TV and YouTube, all prodded along by my healthy–if a bit irreverent–skepticism.

Reading my last post about JQ1 should bring you up-to-speed:

Say it ain’t so! Has Dr. Bradner’s JQ1 research team just sold-out?

Hopeful followers of JQ1 should be thrilled to learn that Dr. Bradner himself has been following along as well.  I was surprised to read his comments following my last post late Monday night:

Thanks, Pat, for prompting this lively discussion.

My lab and I are blown away by your interest in and support for our work. Make no mistake, our lab is still totally committed to developing the next generation of cancer molecules. In fact, we have already completed the optimization of the drug-like JQ1 molecule, now being developed for clinical investigation as early as next year. And we have enjoyed significant progress on three new classes of drugs this year, owing to the tireless and relentless effort of my lab and collaborators.

Please try to consider this article, which has captured so much attention online (it’s just wild), as an exemplary illustration of open-innovation in drug discovery. We have enjoyed an incredible collaboration with the Matzuk laboratory at Baylor in assessing this activity of JQ1 in reproductive biology. Through collaboration we were able to expand our bandwidth *without* jeopardizing our total commitment to cancer research, enjoyed cross-pollination with a sophisticated community of scientists, and contributed an important observation to a very mature field in need of new approaches to male contraception. The accompanying, generous perspective in Cell by William Bremner, a long-time champion of the field of reproductive biology, really drove home how meaningful this type of interaction can be. And this would simply not be possible without making the compound freely available, providing further evidence that open-source drug discovery is very much a new and promising paradigm. You might know that we recently published a very cool, and hopefully important, story about JQ1 in reactivation of latent HIV with Monty Montano and colleagues at BU School of Medicine. Also very exciting. In fact, the activity of JQ1 on spermatogenesis may actually provide an important biomarker for the Phase I study, for those who can and are willing to provide samples.

Now, you have asked whether we are ‘selling out’. If it’s any reassurance, I am still driving the old Ford Fusion and moonlighting on the weekends – so I assure you that we are not in this for the money. There is only one thing we want – responses in the clinic.

Take care, and thank you so much for following our work.

Fondly, Jay

How cool is that?  I’m sure that my readers appreciate your update, Dr. Bradner.  I know I do!

I responded by thanking Dr. Bradner for his thoughtful response to my posts.  I went on to write, “It is easy to become jaded when you view the myeloma world from a patient’s perspective.”

And it’s true!  Do this long enough and I often find myself rolling my eyes over the next latest, greatest therapeutic invention that most likely will end-up stalled in the lab.

Buried toward the end of one of many articles about JQ1 last September, Dr. Bradner was quoted as saying,

“Yes, like many others, I’m guessing that researchers may be a decade away from developing a patient friendly application for this new anti-myeloma/cancer technology. “

I closed my post that day this way:

Here’s hoping that 1) JQ1 turns-out to have a practical application which can help myeloma patients and 2) We don’t have to wait decades for this important research to work it’s way through the system.

Lives are depending on it.

I’m sure I’m not alone in wishing the JQ1 team the best of luck with their research!  I invited Dr. Bradner to update us from time to time on their progress.  And I will keep my ear-to-the-ground so I can share news about other promising new therapies with you, too.

After all, it’s what I do.

Feel good and keep smiling!  Pat