The Answer is No

I have been very distracted the past several months.  My blog posts have been sporadic and maybe a bit less personal.  I had a lot on my mind, but I haven't wanted to say anything until we had some answers.

Shire Pharmaceuticals is conducting a first phase trial for Enzyme Replacement Therapy (ERT).  They are going to be placing a port into the backs of a sample group of children and administering the enzyme our kids are lacking into the CSF (cerebrospinal fluid).  The hope is some of the enzyme can pass through the Blood-Brain Barrier and begin to have an impact on the body.  They are not sure what effect the enzyme will have, but they are hoping it could either slow the disease down, stop the progression or even reverse some of the damage.  It is certainly not a cure, but it could potentially be a form of treatment for kids with Sanfilippo Type A.

The trial is being conducting in the UK and the Netherlands.  As soon as I caught wind of the study, we contacted the doctors in the UK regarding their sample group.  We wanted to be sure they knew about Waverly & Oliver.  We knew a move back to the UK would be difficult, but we were willing to do whatever possible to give our children a chance.  Matt spoke with people in the department and they were so supportive.  They were willing to transfer Matt back to the London office, so our kids would be able to participate.  Everything felt like it was coming together.

Our interactions with the doctors in the UK were not as positive.  I am sure they were being bombarded by families all over the world who wanted one of the coveted spots.  We thought we had a real shot - given the fact we had lived there perviously and Matt could easily transfer back.  Unfortunately, they decided to limit the sample to only those children residing in the UK and the Netherlands.

I have reduced this ordeal to a mere few paragraphs, but it was months of hoping.  I cannot even begin to explain the sadness.  It is something to do, instead of feeling hopeless.

Waverly & Oliver are participating in the Natural History study in MN.  That study is basically the control group for the ERT Trial.  Shire is hoping the US trial could begin as early as 2012, but given what I have learned watching medical research, that date could easily get moved back by months or even years.  We are hopeful that the European trials are going to show some kind of effectiveness, to help them push to get it to trial in the US.

For as disappointed as we are that our kids cannot participate, I am elated that Sanfilippo is getting some attention and people are actively working towards finding a cure.  Please keep those families who have children in the trial in your thoughts and prayers.  It is a huge sacrifice of time to participate, let alone the emotional toll and trust in the doctors to keep their kids safe.  They are helping future families in an incredible way.