When Does Life Get Easy?

I know that it has been a while since the last time that I wrote but I find it very difficult to write when things are not going as smoothly as we like. That said, I am a little less stressed at the moment so I thought it would be a good time to write.

The latest bit of information that we have obtained is that Parker has the most toxic metabolites after transplant compared to the other 11 patients they have had here in the study. What does this mean? No one really knows yet. They toxic metabolites are removed by ADA and for that to happen there needs to be ADA in his system. It can be expressed either by his cells (which we want) or by giving to the body external using PEG-ADA. During the time in which the toxic metabolites are high his new cells have an advantage over the old cells and as a result, they can proliferate and increase in number. This is what we want. The new cells will produce white blood cells that go out into his blood stream, produce ADA and remove the toxic metabolites. For this reason, we remain optimistic. However, Parker's white blood cells did not increase yesterday. They decreased by 100. This is neither considered an increase or a decrease so it does not give us too much information. On Monday, we hope to obtain results from one of Parker's blood tests to see if the toxic metabolites have once again increased in numbers, stabilized or decreased. We are praying that they do not increase again.

As I had said, we do not know why his metabolites are so high. It could be due to the fact that Parker is taking longer to engraft, or maybe it is his particular mutation in his gene that causes him to produce metabolites at a higher rate than most children. It could also mean that the gene therapy did not work. We do not know. We just know that we want his white blood cells to begin to increase significantly to help remove the metabolites and for the result of the metabolite test to come back with a positive result early next week.

Parker wore a blood pressure monitor for 24 hours yesterday to get a better idea of his blood pressure. Apparently it has been quite high over the past weeks and they want to control it. Unfortunately, the monitor did not get very accurate readings over the 24 hours so the results were invalid. That said, Parker will begin taking medication to control his blood pressure while the toxic metabolites are causing his pressure to be elevated. When the metabolites so decrease (I am being optimistic) then his pressure should also decrease and he will not longer need therapy to control his pressure.

My wife's best friend arrived in Italy on Tuesday and we are very happy to have her here. Parker loves playing with her and it is nice to have an extra pair of hands around here to help. Thanks for all your help and support Auntie Natalie.

We did speak with Dr. Aiuti today and his still seemed optimistic with Parker and his progress. We just need to wait for more data to come in so they can have a better picture of what is going on with Parker. The fact that Parker is doing extremely well clinically definitely gives everyone more time before anything must be done to intervene in the natural process of the gene therapy.

As soon as we know more information I promise to update everyone. Parker gives blood on Tuesday of next week and we will be able to get a better idea of his white blood cells (including neutrophils) then. So, we hope to know more then.