The Haystack

Two years ago, and on the other side of the continent, more or less, we were preparing ourselves for our first jaunt above the Arctic circle.  We were also trying to figure out what to do with all of Crust’s luggage, and Richard was earnestly seeking to break the land-speed record on the Ice Trucker highway, but those are other stories entirely…  In my mind, I am retracing our steps and reliving bits of the adventure that it all became.  Revisiting our blog on and the inevitable stream of memories ‘brought to you by FB’ crystallizes those thoughts…and the latter, sometimes daily.

But I’m also reminded that there is a lot of unfinished business.

As connected as I am to other efforts to cure children who, today, are still fighting rhabdomyosarcoma, leukemia, and a host of other just as and some even more horrible diseases – quite a few of which have no known treatment – I regularly see postings and see pleas.  But what can lie ahead when there is no known cure, or the few available therapies have all been tested and failed?

We’ve been exactly here.  Juju faced this, and she—we all—lost.

When the adult population faces a prominent disease (or diseases) for which there is no cure—truly nothing that can confidently be done—does it mostly just shrug and put it on the back burner?  I feel like that is not the case.  There’s lot of private and public investment, the NIH does its share, and lots of therapies ultimately get created.  Obviously, there are many adult diseases that still have no cure.  But the comparatively greater effort that seems to get mobilized behind curing adult diseases always strikes me.

Having been on the losing end of this equation, this has never sat well with me.  In fact, it burns inside.

How do you feel about it?  Would you like to make a difference?

I would.  I do.

Would you like to do something where you know you are literally one step away from people in a lab, working hard to bring cures to children and their families who today don’t have enough hope?

We can make that happen…together!

Last year, Juju’s Journey joined with a multi-national group of family foundations (we call ourselves the Rhadbo Avengers, as we have all been touched by RMS) to sponsor a two-year research project at the Children’s Cancer Therapy Development Institute (cc-TDI).  While every aspect of this has its own story, I’d like to re-introduce Dr. Charles Keller of the cc-TDI.  He was our second scientific interview featured on  (You can catch these in our video gallery, or click on this link:  Dr. Keller Interviews)

We had a chance to visit the lab, meet the team, talk about the innovation his group was driving, and the progress they were making to bridge the tricky gap of taking core research ideas to a point in which viable therapies could be taken into a clinical trial phase.  Dr. Keller and I even geeked out a bit talking about the very clever ways that they had been able to dramatically reduce the cost structure of their operations (i.e., making their funding dollars stretch much farther than in many other settings).  I left a believer.

The Rhabdo Avengers – most of whom already knew Dr. Keller and his lab – unanimously agreed to fund ‘Project Haystack,’ which began with cc-TDI’s review of an entire drug compound portfolio of a major (Swiss) pharmaceutical company.  In an academic-pharma partnership, cc-TDI got access to the entire portfolio of over 640,000 proprietary compounds to screen for efficacy of these drugs against rhabdomysosarcoma (i.e., using a range of cells-of-origin and mutational profiles for primary tumor cell cultures.).  At the end of this process, cc-TDI identified an existing (FDA-approved) cardiovascular drug that exhibits cell-growth inhibitors against both the alveolar and embryonal forms of RMS.  The drug identified (name has been redacted for IP protection purposes) is actually one of a group of four compounds that show great promise and which have never before been tested in the context of RMS.

In short, we’ve found a Needle in a Haystack!

In total, the Rhabdo Avengers have made a $250,000 commitment over two years to support this special project which aims to tee up a drug for adoption into pediatric Phase I/II clinical trials.  Finding ways to reduce the risks of a drug going into these trials is a key goal for any pharma company and can speed the realization of a winning therapy.

While Juju’s Journey has only a very small share of this commitment, we still need your help to meet and ideally exceed these goals.  We’ve included in this post a link to a JJJ-specific donation page and hope to raise at least $5,000 over the next month.  (All funds will go directly to cc-TDI.)  We’d like to do better, as we believe in the cause, we know the lab, and we are honestly excited and proud to be able to be part of this incredibly important work.

And how often do you get to be one step away from the folks creating the cure?  You will directly fund the scientists in white lab coats wearing purple nitrile gloves.  You’ll be that close.

We hope that you will join us and make a donation today!  Your support is so greatly appreciated, and you can’t place a value on the importance of these steps and actions taken towards improving the outcomes and lives of countless children (and their families) everywhere.

Click here to Donate to Support cc-TDI and Juju’s Journey