By Amy Dockser Marcus
We’ve been following the efforts of one family, the Hempels, to draw attention to the potential of cyclodextrin, an experimental drug that may have potential against a fatal neurodegenerative disease called Niemann-Pick Type C.
Addison and Cassidy Hempel, twin six-year-old girls, have the disease, and their parents were part of a larger group that earlier this year successfully got orphan drug designation for the compound from the FDA, a key first step for drug development in rare diseases.
The FDA this week gave permission for cyclodextrin to be injected into the spines of the girls. They’ve been receiving the drug intravenously for over a year but new research indicated that it does not cross the blood-brain barrier.
Chris Hempel, the girls’ mother, tells the Health Blog that the family pursued the new approach for delivering the drug in the hopes it will get into the brain and stave off some of the neurological decline associated with NPC. She also hopes that the information they report to the FDA may help bring cyclodextrin one step closer to a trial.
A spokeswoman for the FDA says the agency can’t comment on drugs under agency review or on any individual’s private medical information.
It isn’t easy to get clinical trials going in children with brain disorders. The Children’s Neurobiological Solutions Foundation held a meeting last week with clinicians, researchers, advocates and others to discuss some of the barriers, including the potential risks to participants. (Those can leave drug companies and hospitals skittish.) The foundation is trying to create a national network of sites in the U.S. that will collaborate on setting up trials for pediatric brain disease treatments.
The NIH held a workshop on developing therapies for NPC disease earlier this year. Cyclodextrin played a prominent role there too. A working group of researchers and clinicians has been discussing what scientific questions need to get answered before a trial could be launched.
Hempel tells the HealthBlog that she hopes this latest FDA decision will help propel larger efforts to start a trial. “The biggest hurdle for a trial is showing something is safe,” she says. “If we show this works in the girls, we hope a broader trial can start for kids with NPC.”
Thank you to the Hempels for all their hard work and PERSEVERANCE!! We love you Addi and Cassi.