Fiona Midori King

Fiona Midori King

Hope 4 Fiona

Trip of Hope - Sunday February 26, 2012

 

 

 
Today we started a new journey for Fiona at the Fairview Children’s Hospital in Minneapolis. We traveled there for a consultation to discuss a potential treatment regimen, which includes the drug Migulstat, with Dr. Chester Whitley, a geneticist, and Dr. Jeanine Utz, the pharmacologist. The treatment consists of 4 different drugs: Miglustat, Ibuprofen, Minocycline, and Acetylcysteine. 
 
The new doctors in Minneapolis want Fiona in their treatment program. She would be the youngest to enter the program and is in the best condition compared to the other 6 patients. There are 4 GM2 patients and 2 GM1 patients. The GM2 patients don't seem to be responding as well to treatment compared to the GM1 kids. Both physicians were struck by how good Fiona looked and thought she must have a low level of undetectable enzyme activity – a comment we have now heard from 4 physicians. They commented that her type of genetic mutation was a splicing error which is a less severe form of mutation compared to other types.
 
Cost of treatment comes down to the cost of Miglustat/Zavesca each month. Fiona needs 100 mg, 3 times per day which is 300 mg daily. The cost of this drug at those levels is $18,000 per month, for the rest of her life. Miglustat is an FDA approved drug; approved for patients with Type I Gaucher disease. What U of Minn has found is that the drug catalyzes and inhibits a multitude of chemical reactions in the body. One of the things it does is prevents the formation of GM1 Ganglioside which is the specific lipid building up in Fiona's cells. If she has a low level of enzyme (beta galactosidase) and we prevent the formation of GM1 Ganglioside, over time her enzyme would break down the material already stored. Obviously time is of the essence.
 
At least we still have a little bit of hope to cling to.

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