As 2016 is coming to a close I am very much looking forward to the very exciting things that the new year will bring our family. One of the big things we are looking forward to is Lucas doing a drug trail with a medication called KUVAN.
What is KUVAN & why should we try it?
(much of this is copied from the http://www.kuvan.com website)
KUVAN is currently not covered by any drug plans, especially the plan we have. The company that makes the medication has people in place to help us make it possible to keep Lucas on the drug if it is effective, however with the extremely high cost of this drug there is a very good chance we may have to stop the medication after the trial. We are hopeful that in a few years the drug will perhaps be covered by drug plans or that the cost of the medication will come down to something that we can make happen.
What is KUVAN & why should we try it?
(much of this is copied from the http://www.kuvan.com website)
This medication trial offers us the chance to see if Lucas is a responder to KUVAN.
KUVAN is a form of the cofactor (or helper) BH4 that already naturally exists in your body. In people who respond to treatment with KUVAN, it works in the body just like BH4 does to help stimulate residual PAH enzyme activity to convert Phe to tyrosine and lower blood Phe levels. KUVAN has been prescribed along with a low–Phe diet to lower blood Phe levels in many people with PKU of all ages and types
In many people with PKU, KUVAN increases the activity of the PAH enzyme that isn’t working properly. In other words, the PAH enzyme that isn’t working “wakes up” and starts to process the Phe. This helps to lower the amount of Phe in the blood.
A recent study compared children with PKU taking KUVAN along with a low-Phe diet with those who only followed the diet. The results show that those who responded to KUVAN not only reduced their blood Phe levels, but were also able to increase dietary Phe. KUVAN may allow children to add more natural protein to their diet to support critical growth and development while keeping blood Phe levels within the recommended range.
KUVAN is a form of the cofactor (or helper) BH4 that already naturally exists in your body. In people who respond to treatment with KUVAN, it works in the body just like BH4 does to help stimulate residual PAH enzyme activity to convert Phe to tyrosine and lower blood Phe levels. KUVAN has been prescribed along with a low–Phe diet to lower blood Phe levels in many people with PKU of all ages and types
In many people with PKU, KUVAN increases the activity of the PAH enzyme that isn’t working properly. In other words, the PAH enzyme that isn’t working “wakes up” and starts to process the Phe. This helps to lower the amount of Phe in the blood.
A recent study compared children with PKU taking KUVAN along with a low-Phe diet with those who only followed the diet. The results show that those who responded to KUVAN not only reduced their blood Phe levels, but were also able to increase dietary Phe. KUVAN may allow children to add more natural protein to their diet to support critical growth and development while keeping blood Phe levels within the recommended range.
What will this mean for Luke?
First we need to determine if he is a responder, because if he doesn't respond then that means this medication will not be an option for Luke.
First we need to determine if he is a responder, because if he doesn't respond then that means this medication will not be an option for Luke.
If he does respond then we have a bit of a road ahead of us. First, with Luke this would mean that he could eventually eat a significantly higher amount of natural protein. Even with this increase he will not be eating things like cheese, eggs or meat. However he would be able to eat things like regular waffles, have a cupcake at a party, maybe even have some pizza with no meat or cheese on it!
KUVAN is currently not covered by any drug plans, especially the plan we have. The company that makes the medication has people in place to help us make it possible to keep Lucas on the drug if it is effective, however with the extremely high cost of this drug there is a very good chance we may have to stop the medication after the trial. We are hopeful that in a few years the drug will perhaps be covered by drug plans or that the cost of the medication will come down to something that we can make happen.
As always, any questions are welcome :)
Hello....I stumbled across this blog site and just wanted to drop a comment to your post. My wife and I are fairly new to the PKU world as our son, Nicolas was diagnosed with PKU during his initial screening. He is 11 months, and we are preparing for his 1 year birthday.
ReplyDeleteThank you for sharing about Kuvan and I hope your son, Luke, has a positive take to it.
Thank you so much for taking the time to comment, it is always nice to hear from others. Hope you and your family are enjoying discovering his food tastes and all the other very exciting things that happen from 1 year old to 2 years old. Feel free to ask any questions or reach out via email.
DeleteYou already told me this I believe, but can you explain again why Kuvan is so expensive? It has to do with patenting, right?
ReplyDelete