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Witnessing a Treatment: Gulp


Shoots & Giggles Photography

Yesterday and today were two very different days. Both put us face to face with both the grim reality of the NPC diagnosis and also the exceptional people that NPC has brought into our lives, and those that were already there. The two days were in sharp contrast both to one another and within themselves. Today, representing hopefulness for the future and the vibrancy and sweetness of Marian, clutching her belly while throwing herself back on the bed in fits of giggles while having her picture taken. However, it also had a pull of feared foreseen nostalgia, wondering, is this going to be one of the good times - that we'll look back and say, she was so healthy and happy here.

Yesterday was on the surface much bleaker, I observed a treatment and it was rough for me. I kept visualizing Marian's little body confined to a hospital room on a regular basis for the foreseeable future. Our new reality. But it also is our gateway to a hopeful healthy future for her. So you can see, it's really quite a confusing mix of emotions. I'm going to write about the photo shoot tomorrow and share the beautiful pictures the golden hearted Briana from Shoots & Giggles took at no charge to capture this time. Today I'll write about yesterday, observing the treatment.

A wonderful NPC family invited me to go to their local hospital about an hour away to observe their son's treatment, their son is the only patient at this hospital receiving VTS-270 via compassionate use. This is what Marian needs if she will have any real chance. Naturally, since I am such a calm and centered adult, I asked my aunt to go with me. So the two of us hit the hospital, weaving through the crowded hallways to the pediatric unit.

Ok, so it's not like a million people are reading this blog, but this is a child and while I do have his family's permission to share what happens at treatments, I'm going to leave some things out, including his real name. Ted is a teenage boy and I was blown away immediately by how magnetic his personality is, he has a great smile and was clearly at home in his hospital room for the day. He's been doing this every other week for years, now the treatments take about five hours - an hour or so of prep, 10 minutes of treatment, four hours of observation. This is an improvement from the first year when he had to spend the night each time. He's obviously a favorite of the doctors and nurses and they moved through his room with tenderness and efficiency.

When we got there the nurses were taking his blood pressure and temperature. Next they came in and drew his blood, about four or five vials. I sat in a blue chair totally in the way and nervous. Next came the relaxing juice to prep for the actual treatment. For the treatment Ted lay on his side - not totally sedated due to his age. Marian will need to be completely under. The doctor cleaned his back and then applied a numbing lotion. After that the doctor injected more numbing medication with a syringe into his back muscle. Next she pierced his back with a long needle that remained protruding with various chemistry class looking vials being attached, removed and reattached for the rest of the procedure.

The needle is inserted between two vertebrae. My aunt slid a chair behind me, just in case I think. The doctor did the procedure with such ease and confidence, she was also incredibly kind, personable and calming, explaining what she was doing for my benefit. After the needle is inserted a long narrow vial is attached and a small amount of liquid is flushed into the spinal fluid. When completed after about a minute, it's removed and then about two teaspoons of spinal fluid are removed. However much cyclodextrin is put in needs to be removed in a corresponding amount in spinal fluid first. This was the majority of the procedure, collecting enough to match the amount of cyclodextrin - it took about five minutes. I felt 50% fascinated and 50% woozy.

The last step is then attaching a new vial to the needle and slowly injecting the VTS-270/cyclodextrin into the spinal fluid. When completed the needle is removed, the wound cleaned,and bandaid applied.Ted then rotated onto his stomach and the hospital bed was angled down about 45 degrees so gravity can help the VTS-270 reach his brain. He'll stay in that position for about forty minutes. Afterwards more blood drawn, a few hours of observation, snacks and then heading home. Frankly, I was exhausted and all I did was sit there. I cannot believe my baby is going to have to do this to stay alive. But, I also am so unbelievably lucky that there is a path for her to stay alive. We are definitely going to need some help at the hospital getting the hang of this.

The doctors at the hospital treated me like they had nothing else pressing them but to help us learn about the process - which certainly was not the case and I think a true testament to what incredible physicians they are. They were some of the nicest, most compassionate people I have ever met - not to mention that they are brilliant and highly important pediatric specialists. As unsettling as the treatment was to see, it was so important to take this in and prepare. I am so grateful that such a personal moment was opened to us. I don't think anyone would ever think - oh yes, that's a piece of cake, I can definitely do that every two weeks forever. But to see how this family has adapted and their love for each other gives me hope that we can do the same. To also see the partnership and bond between the family and their doctors was a true privilege. It seems that within this field, the doctors work together across hospital, city and state lines to support the best care possible for these kids and their families - which is truly incredible and comforting beyond words.

I'm not sure if this is translating well into written form, so I'll just say again. Despite the kindness, emotional handholding, and calmness of everyone around me, I was still rattled and sad to think of Marian laying vulnerable in a hospital bed over and over again. I don't know how we will make this work but I do know that we will. This is what can save her. It won't work forever but it could work for a really long time, years and years, enough time for the missing piece of the treatment to come along. Which it needs to. I also am praying that they'll be able to find a less invasive way to administer the treatment that is equally or more effective soon.

It's silly to say but one thing I love so much doing each week is taking Emily to her ballet class. She started in September right at the same time as the path where Marian didn't have anything serious going on was steadily sinking - or more like took a Titanic plunge. It's the one normal fun thing each week that we routinely do together that doesn't involve physical therapists, medical specialists, etc. The reason I bring this up is, I'm sure this is mis-placed anxiety, but I don't want to have to stop taking Emily to ballet. Of course I will if I need to - but it's the one thing we've been able to hold on to where she can see her friends, carefully twirling around the room with her ponytail whipping around her face. Marian can play with the other little siblings watching, and I can chat with the other moms and think about four year olds doing ballet - and not everything else. I don't want to have to stop doing ballet, our last voluntary activity, and replace it with this. I'd really prefer to not have this be happening at all though and there's no point in that. So, if ballet is on a Monday, maybe the hospital can do the treatment on a Tuesday. I guess that would make it all ok.


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