Look what you did


One of the phase I clinical trials that we funded with YOUR money was just published – you can click on the title below to read it in it’s entirety. We have been funding this trial with your donations and support from the very beginning – between directing $10,000 from one of our events to Hawaii to perform the pre-clinical work – to sending another $80,000 to Dr. Sholler to fund the study opening.  YOU have played a critical role in making this study happen and it’s results have led to even more studies.  There are currently TWO other studies that are open using this drug – and enrolling patients – thanks once again to YOUR donations.  There is another groundbreaking study set to open later this year that will also be using this drug and your support to save KIDS with cancer.

A Phase I Trial of DFMO Targeting Polyamine Addiction in Patients with Relapsed/Refractory Neuroblastoma

Phase I studies are traditionally done with the specific goal of finding out what the safe dose is of a new drug that has never been used before in that patient population.  You start with a very low dose of drug and then enroll three kids – before watching for side effects that would be a reason to stop the drug.  If none are found you then enroll the next 3 kids at a higher dose and you continue until you find the MTD – the maximum tolerated dose.

Think about that.  The goal is to not cure – to not find the most effective dose – but instead to find the HIGHEST DOSE of drug that is tolerable.  The next step?  To open a phase II study where everyone gets the drug at the MTD – usually in combination with an already established cytotoxic chemo agent – to see if it actually does anything.

It is no wonder survival rates are stagnant.

With that as the backdrop it is important to understand that phase I studies – especially in relapsed NB – do not save kids.  What do the kids who enroll on phase I studies look like?  For the most part these are kids who have been told to consider hospice.  These are kids who have tried and failed any known ‘curative’ therapies and whose own home hospitals have given up on curing them.  They then usually go to the one of the know “NB specialists” – fail their therapy – and then try these phase I’s.  This is a HEAVILY pre-treated population with little statistical hope for survival but whose parents are still filled with hope.  For this paper I was incredibly thrilled that they went ahead and created a chart showing just what therapies these kids had endured BEFORE enrolling on this study.

NOTE: this list of therapies does NOT include the upfront treatment they received at diagnosis which usually includes: 5 or 6 rounds of high dose chemo, 1 or 2 rounds of myeloablative chemotherapy followed by stem cell rescue, surgery, radiation, antibody therapy, biologic therapy. Please click on the link below to see just what these kids endured BEFORE this trial:


Did you just look at that?  Staggering.  It is truly mind boggling to consider what these kids have to endure at a chance at beating this disease.  And if they do get it in remission the specter of the crippling side effects from this treatment that lay ahead.

My own child enrolled on this study while a patient in Vermont when the NMTRC was housed there before the move to Grand Rapids Michigan.  The first patient to enroll lived in another country, the second was from Maine, the third from New York and the fourth – my son – lived in Massachusetts.  All these kids found themselves living at the Ronald McDonald House in Burlington Vermont because everyone else – EVERYONE – had given up on them.

Except for their parents.  Except for Dr. Sholler.  Except for YOU.

I understand why so many folks have grown frustrated with what little impact research dollars and support have had on actual patients.  I understand their frustration – I have seen it far too often and now understand that actually funding work that has clinical impact is the exception to the rule.

However, when you find that exception it is so incredibly important to do all you can to support and promote that work.  It is so important to go ‘all in’ so that this exception slowly becomes the rule.

I could sit and bemoan the state of research funding, the peer reviewed process and the lack of progress all day long.

That will change nothing.


Instead – I will do all I can to make sure that Dr. Sholler and the NMTRC can continue to take your money and turn it into clinically relevant trials within 12 months so that kids fighting today can get a chance to enroll.  Can get a chance to live.

This is what we do with your dollars.  This is how we are changing survival – by not complaining about what is broken but instead focussing all of our energy on funding what is working.



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