Your money at work

As you can read in yesterday’s post we sent $20,000 of your money to help fund some extraordinarily exciting research.  The goal is to get that work further down the line so that it can one day be in need of funding to get it into the clinic.   What I wanted to do today is provide you with an update on where the clinical trial we helped fund with $100,000 of your money is headed and what has been accomplished thus far.

The money that we sent along was to help open the phase II clinical trial using DFMO – you can watch this video to learn more about the background and history of DFMO and why it is such an exciting drug.  There were multiple arms on this study in terms of what types of patients to study but eventually it morphed into two trials:

NMTRC003 – Preventative trial of DFMO in patients with high risk neuroblastoma in remission

NMTRC006 – Expanded access trial of DFMO (this is for patients who would benefit from the NMTRC003  trial but don’t meet the entry criteria for that study.)

This in an off itself is remarkable since there are very important scientific questions being asked in NMTRC0003 that require certain entry criteria.  However, the 2nd study was put in place so that this entry criteria does not unduly ‘punish’ kids who could truly benefit from this drug.  They may not meet the entry criteria that is required to ensure that questions being asked in this phase II study can be answered but that does not mean they would not benefit from taking this drug.  Most cancer parents understand that to get access to a drug like this outside of the study criteria would  require access via “compassionate use”.  But instead of having to jump through those hoops the wonderful folks at the NMTRC took this extraordinary step to ensure that as many kids can be helped as possible.

So in March you came to Cure Me I’m Irish and we then sent $100,000 of your money to the NMTRC to get this drug into kids and in June the study was opened and the first patients began to enroll!  So -just three months after you joined us in “Celebrating with a Purpose” that purpose was realized as a child was able to take a drug with the goal of saving their life.

This is amazing.

And what has happened in the six months since that study opened?  For some background the phase I DFMO study just finished up after being open for 2.5 years and the final patient enrollment number over that time frame was 21 children (you helped fund that study too by the way…).  In the six months since the phase II study has been open a total of 29 children have already enrolled!

NMTRC003 (NED study) – 18 kids

NMTRC006 (expanded access) – 11 kids

There are currently 4 kids being screened for entry into NMTRC003 and the trial is open in SEVEN hospitals across the country!  Two more are coming on line next month and 5 more sites will come on board in 2013 which means there will be 14 children’s hospitals across this country where kids can go to be receive this drug!  That is precisely why we fund a consortium and how such extraordinary enrollment figures can be seen.

In the United States each year roughly 700 kids are diagnosed with NB – so lets call it 350 KIDS every six months.  In that same time frame nearly 30 kids have been given access to this drug – I can’t highlight to you how extraordinary these enrollment figures are.

So how does your money help?

This study would not be open without it – along with the $200,000 that came from other NB parent led foundations.  In fact, you are also actually paying for the trial cost AND the drug in these trials since the drug company is not providing the drug free of charge for this study.  Also, the way a consortium works is that someone has to pay each hospital for their work so each time a new hospital opens this trial the NMTRC fund needs to send $5,000 out to that hospital upon IRB approval another $2,000 must be sent each time a new patient enrolls.

This is only possible thanks to the money that was raised and donated by YOU.  This entire study has been paid for by NB parent led foundations – most of which are led by parents whose kids are no longer here.  For me personally this is so incredibly amazing – and sad.

It is amazing to see these wonderful people working so hard to make a difference – it is amazing to see just what has been accomplished with your money this year.  And it is also terribly sad that this is who it falls to to save these kids.  However, we can just sit around and point fingers at the injustice of it all or we can just keep doing our part to direct all of our focus on what is important – and that is saving these kids.

You make this happen with your support and this is not an inexpensive endeavor.  We’ll once again have to send funds from this years event out to the NMTRC to fund this study as there was enough money to open the study but more will be required to keep it going.   So as Cure Me I’m Irish rolls around again this year I want you to come out and have a wonderful time and also know that our volunteer only organization is doing all it can to help make a difference with your  money.

I truly hope that you feel that we are worthy and deserving of your continued support.

Thank you

Patrick Lacey

Founder -Beat NB Cancer Foundation

P: 781-519-9787 E:

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