Waiting has been hard.

Last September it seemed everything needed to start Gene Therapy for Ray would occur in record speed. I expected to see everything gear up beginning in January, but nothing happened.

NIH had to wait for new vectors from St  Jude and then test them to make sure they worked as expected. The bigger and heavier the patient the more vectors are needed. It is calculated based on how many kilos the patient weighs. So while Ray is small for his age, he weighs as much as probably 10 infants.

Apparently the newest batch of vectors from St. Jude were not performing with as much effect as the older batches. Vectors have to be grown, so I’ve compared the process to waiting for the perfect flower to bloom. No matter that you’ve watered it and feed it and given it sun, not every bloom turns out to be a blue ribbon winner at the State Fair. We want these vectors to be blue ribbon winners.

Since then Ray has been on 4 days per week of IV nutrition in addition to his regular feeding pump which pumps a liquid diet into his stomach. Part of the problem has been that his stomach and intestines are not handling his nutrition very well. We know he’s had a chronic virus in his GI tract which he cannot clear unless we help him to build a better working immune system, so we feel this can be improved through gene therapy. Meanwhile, I keep having to dilute the feeding pump nutrition with pedialyte to give extra fluids. This leaves him getting too little nutrition from this source, hence the need for more IV support. Ray can’t or won’t contribute to managing any of his IV care, which means that it has all fallen to me, making sure I’m available to access his port, hook up the tpn, unhook the tpn, and be available the entire time it runs in case of an occlusion or pump problem. Since Ray is hearing impaired he cannot even hear if the pump beeps so I have to sleep with a baby monitor by my head to hear if there’s a problem.

It’s been long and tiring waiting, but it seems the wait will soon be over. We got a call from Suk See DeRavin saying they could be ready at the beginning of June! Definitely exciting and and a little scary all at the same time. It looks like this week he’ll have to be at NIH for a couple of days for a bone marrow aspirate.

This is getting real!