Disappointment, Encouragement and Uncertainty

Before I begin, I have to admit that I have never felt so much stress in the span of one week in my life. Everyone that reads the blog regularly knows that we have been waiting for some results the past few days in hopes that Parker has a chance of making the gene therapy work.

I had mentioned previously that Parker's metabolites had been increasing at a very high rate. The metabolites give his new cells an advantage over the old cells but for some reason we were not seeing the effects of the advantage. We were waiting for Parker's blood test results to arrive on Tuesday in hopes that his toxic metabolites either decreased or stayed relatively the same. What we got instead was our doctor telling us that he was not very optimistic anymore, that the toxic metabolites not only increased but they doubled and Parker had lost most of his engraftment when they looked in the peripheral blood. He was not ready at the point to say that Parker was the only patient who the gene therapy did not work for but he did say that he had the least number of engrafted cells and the highest number of toxic metabolites compared to any other patient so far.

Everyone was dumbfounded. No one could have predicted that Parker, the best patient to enter the study was going to be the only patient that would not display relative success with gene therapy. My wife cried. Me? I just could not understand what was happening.

The only good thing that came from our conversation was that for the first time the red blood cells had shown an increase in ADA activity. When Parker had his bone marrow aspiration he was given a red blood cell transfusion. Of course these are not his cells and display ADA activity. But as the days passed these cells died and the ADA activity from his red blood cells constantly decreased. What was surprising was the fact the Tuesday's results showed a 50% increase in ADA activity from these cells. The only way this could be would be for Parker's bone marrow to have produced them. The team was not convinced that this was the case and that it was not just an error in the test so they decided to repeat the test and put a rush on the results.

So, the past two days we have been miserable. We have been trying to figure out what we would do if the gene therapy did indeed fail for Parker. Yes, we were hoping for some miracle but being realistic we did not expect anything to get better when the results arrived later this week. No one here in Italy was very optimistic.

Last night, the phone rang close to nine in the evening. It was our doctor. He said that he did not want to wait to give us some good news. Parker's metabolites had decreased 30% in the span of one week. He also said that they now feel they can predict what is going to happen with his metabolites. They should slowly decrease over the next weeks. Does this mean that it is working? Does this mean that we are out of the woods? We don't know. We will probably meet with our doctor tomorrow and get a better understanding of what is going on. We do not really know what is happening in Parker's bone marrow. Did his cells proliferate and increase or were they all overstressed and die off. We don't know. We just hope that the metabolites do continue to decrease and his white blood cells begin to increase steadily.

We just want this to work so that Parker, his mother and father can live a normal life eventually.

Thank you to everyone who has been praying for Parker the past while we know that every prayer helps.