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Happy New Years! (and a drug called nitisinone)

Posted on Fri, January 1, 2010 at 06:38PM by Registered CommenterNeil Hutchison | Comments40 Comments | References52 References

New Year’s Day is a time for renewal and my goal for 2010 is to blog every day this year to raise attention and money to help children beat neuroblastoma.

Many have followed my son’s journey (www.teamsam.com) for the last 5 years since his diagnosis of Stage 4 metastatic neuroblastoma in January 2005.  He is a wonderful little boy, the oldest of three boys, who loves life, school, and tormenting his brothers.

If you don’t know much about me, with other parents, I have helped form (2) foundations:  The Penelope & Sam Fund at the University of Vermont Cancer  Center (place link) and Magic Water.  Contributions to-date of Penelope & Sam Fund well exceed $1,000,000 and contributions to Magic Water exceed $985,000.

With this blog, I hope to provide a unique and compelling perspective from a parent who is fighting to save his son’s life.  Neuroblastoma is a mean, evil, nasty cancer and is the #1 cause of death for children under the age of 5.  Much ballyhooed progress in pediatric cancer is skewed by statistics:  pediatric leukemias are much more highly curable than solid tumors and more kids get leukemia than solid tumors as well.  There is much work to do for kids with solid tumors.

The focus of Magic Water is to help kids fighting today.  That means we are interested in doing the hard work —- opening and funding clinical trials based on solid science.  It is very easy to publish scientific and medical papers outlining basic research that speaks of results in mice or in cell lines.  People have built entire careers publishing basic research papers that never have seen the light of a clinic.  The difficult work is navigating the FDA, hospital review boards, and the politics of cancer research to open clinical trials.  And it is very difficult to raise the money to fund each trial.

Magic Water is very proud that every dollar raised goes directly toward some type of preclinical or clinical research that can help kids either immediately or within the next 6 to 12 months.

We are also very optimistic that there is hope for kids TODAY and so I start my 2010 blog by speaking about a paper published in 2006 about an FDA-approved drug that helped a multiply-relapsed neuroblastoma child live an additional 21-months. 

If you read the paper, you will see a very familiar story:  once a child with neuroblastoma relapses, non-toxic options start to dwindle.  The child had disease throughout his bones and bone marrow, his liver and his spleen, and was on intense levels of pain medication to manage the pain.  Prior therapies yielded short-lived responses and each response became more and more brief.

The drug used is for a very rare condition called tyrosinemia – about 100 children in the US have this every year.  What’s interesting about the thought process of the lead author, Dr. Nathan Kobrinsky MD, is that he had always thought of neuroblastoma as being a disease of tyrosine metabolism.  And he’s not alone in that thought – tyrosine kinase inhibitors have been in clinical trial for neuroblastoma for years and it is well-understood that over-expression of Tyrosine Kinase A means a good prognosis; over-expression of Tyrosine Kinase B means a much more bleak perspective.  So Dr. Kobrinsky made an educated guess and produced a remarkable case study. 

He also tried to help other kids - he approached a team of neuroblastoma specialists and asked them to open the trial.  He traveled with one of their leaders to Sweden to meet with the drug manufacturer.  But as of January 1st, 2010, no other child with neuroblastoma has tried this drug.

I have some anxiety as I write this column – I want to challenge the medical community and I am always worried that my comments will affect how the community would treat my child.  You get to a point where you can’t worry about it.

So I ask “when you have such a powerful case study that does the equivalent of stopping a speeding train using a drug that is FDA approved, used in pediatrics, and with known toxicities, why isn’t this in clinical trials for neuroblastoma TODAY?”

And for an answer, I turn to television.

One of my favorite shows is Pardon the Interruption on ESPN.  Two bald, middle-aged guys arguing over everything sports.  It’s intelligent, smart and funny.  And one of the hosts, Tony Kornheiser, always mentions that Don Ohlmeyer, one of the wisest men in television, told him that if you don’t know the answer to a question, the answer is money.

To answer the question of nitisinone, the drug I mention, or any of the other things I will write of as the year goes by, Magic Water will need money because only money gives you a seat at the table to help influence decisions.

So please help as you can, if you can.  I promise I will make this commentary insightful, honest, and any monies will help children.  And it may even help you or a loved one in their fight against cancer.

Sincerely, Neil

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