Typically compassionate use treatments carry far more risks and families asking for compassionate use approval have to find drugs that have many more years worth of safety data research.
"You got to try to move as fast as I can," said Hempel. "All of this is cholesterol-related; my kids have one of the worst cholesterol diseases on the planet. ? I'm trying to move as fast as cholesterol."
Despite Hempel's success with the FDA, the disease and the treatment will likely pose challenges.
According to Patterson, cyclodextrin has only proved beneficial in mice before they showed symptoms, not after. Mice studies rarely translate into human success, and, Patterson said, it is difficult for doctors to measure whether the drugs are working in neurological diseases like Niemann-Pick Type C.
But Hempel has already begun to try. The girls have been fitted with a chemo port that will allow their blood to be infused with the compound.
Working with doctors at Renown Regional Medical Center in Reno, Nev., the Hempels are now attempting to document the girl's condition before treatment.
"We want to see where we are today, because how else are we going to know if it takes an effect?" she said. Among other things, Hempel said the doctors are trying to measure the amount of cholesterol excreted in the stool.
"Now we have to collect diapers and we have to freeze them," Hempel said.
Patterson said whatever the outcome of the girls' treatment, he thinks families like the Hempels make a difference.
"This story exemplifies the extraordinary dedication that mothers like Chris Hempel and Cindy Parseghian [three of her four children died from Niemann-Pick Disease Type C and she raised millions of research dollars] bring to the pursuit of help for their children," said Patterson.
"There are countless stories of such parents but only those with access to the media become known to a wider public," he said. "The burden of suffering associated with these rare diseases is far in excess of their numbers."
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