Look what we are trying!!

PKU update:
In the fall of 2009 we were asked if we (Taylor and Shaylee) would like to participate in a clinical study for a new drug called KUVAN. First off KUVAN is a new prescription that is being study in people with PKU. KUVAN is the first and only prescription medication that helps some patients lower phe levels. Along with careful dietary management, KUVAN may help lower blood Phe levels more than the use of diet alone. However not all people with PKU respond to KUVAN, also KUVAN does not replace the diet it just helps lower phe levels allowing the persons taking it to eat more foods that contain phe.
When we were first asked to do this study I wasn’t sure how I felt about it for a lot of different reasons but mainly I was afraid that because not everybody responds to KUVAN and because I have two children with PKU that only one would be responsive. I know that I should have just been happy that there is finally something for people with PKU to use other than the diet but I couldn’t imagine not having both of my girls respond. I either wanted it to work for both of them or not work for either. I don’t really know how explain the way I felt but that was a big fear of mine, that and then just the fact that you are trying an experimental drug on your children is something very very scary!! However we decided that we needed to give it try!! Now when I first posted about “Our life with PKU” I told you I would be very honest with my feelings about living with this so……………….. I will be.
It was very exciting and nerve racking the first time we went up to University of Utah where the study was to be done. They explained that we would be expected to come up once (sometimes twice) a week until the study was over. They explained how we were to give the KUVAN and gave us a big book of papers and information. And then it began-

Our Study team (minus Dr. Longo)


There was a lot of pokes that the girls didn’t love but the nurses were so AWESOME so that helped a lot!!!!!
Here's Dr. Longo with Shaylee

I think I must have forgot to wash my hair that day :/ it looks awful!!!

Brooklyn was such a comfort for both of her sisters! I don’t know what they would do without her!!

So through out the trial they do not let the parents know to much (nothing really) about the phe levels because they want you to keep feeding them how you normally would instead of trying something to lower the phe levels. A little over half way through the trail Dr. Longo said something that I was really not wanting to hear. He said that as of that time it looked like only one of the girls was responding. I was so upset but I tried really hard to smile, I didn’t want the girls to think something was wrong. Like I said I tried to smile and I did a really good job of it until we got out of the hospital and to lunch and then I lost it, the poor waiter wasn’t sure if he should inturped my sobs to ask me what I wanted to eat or just come back later. hehe I wasn’t doing so good but after I had my melt down I pulled it back together and we finished the trial. After the month was over a girl named Krista called to let us know the results of the study. Sadly the girls weren't responsive to KUVAN. That was really hard to hear. I didn’t realize how much hope id put into KUVAN until she said it didn’t work for them.
I said I would be true about my feelings so here is what they were. I think that from the beging, meaning the first day I ever learned about PKU I was told “don’t plan on a cure. PKU is a rare disease there for the drug companies aren’t going to make a lot of money even if they found a cure so not much is being put into finding one.” In my mind that was that I really had no hope of cure and in a lot of ways that was easier for me. I wasn’t constantly hoping for some merical drug. Now I know that KUVAN isn't a cure for PKU, but when I was told the girls weren’t responsive to it I wasn't prepared for the let down of emotions I had. I though I was so much stronger than I was. I was sure I had build up my walls so noting could tear them down. Boy was I wrong! I think I was at my lowest point id had with PKU and to be honest the last two years have been the hardest ive had so far. Its really been a struggle for me and my family and im so thankful for my husband and our family and friends who help us stay on top of all the negative emotions!
Well that was part one. Here is part two! During the first study both of our girls got sick. When you get sick and have PKU it messes with your phe levels. Needless to say we never felt like we knew for sure how they responded to KUVAN because of the sicknesses. To stay in the trail (or be considered responsive to KUVAN) their levels needed to drop by 30% and although they didn’t drop that much they where close. so like I said, ive been having a very hard time dealing with PKU emotionally the last little while and one day after I had just got off the phone with Sharon (the girls dietitian) Mike found me crying in our bedroom. The past year the girls levels seem to be EVERYWHERE, high, low, and then high again and it doesn’t seem to matter how we try to bring them down….. or back up.. or down again, they just wont stabilize. Anyway Mike decided to call Sharon and they had a long talk and decided we needed to try KUVAN again!!! The company that makes KUVAN does what is called an in home study so basically we are doing just what we did before only this time no going back and forth to the hospital! And now they have decided that to be considered responsive the phe levels only need to drop by 20%. I feel like this time no matter the outcome i am much more prepared. Of course I hope they are responsive but if not I feel like we wont be wondering if its because they were sick, we will no for sure! So here are my girls giving it another try!!!

KUVAN comes in pill form but needs to be crushed and mixed with water or apple juice and taken with food. This is the first night they started!


Taylor is so good to make sure she gets all of the medicine at the bottom of the cup!


Shaylee is really good to get all of it too but you should see her face when she gets to the bottom, she sure doesn’t love it :(

And here's Brooklyn making sure everybody has a smile!!! She helps SO much!
We are about half way through with this in home study so I will keep you posted with the results!!